What To Know About Zinbryta

Disease-modifying therapies (DMTs) are important tools in the fight against multiple sclerosis. To date, the U.S. Food and Drug Administration (FDA) has approved 14 DMTs: 13 therapies for the treatment of relapsing forms of MS, some of which are also approved for clinically isolated syndrome; and one therapy, Novantrone, for the treatment of “worsening MS;” however, Novantrone is no longer customarily used in the United States.

In May 2016, daclizumab (under the brand name, Zinbryta) was approved by the FDA as the fourteenth DMT option for people diagnosed with MS in the U.S. In July 2016, Zinbryta was approved for use in Europe. The FDA suggests that because of its safety profile, the use of Zinbryta should generally be reserved for patients who have had an inadequate response to two or more drugs indicated for the treatment of MS.


Read this post in its entirety:
What You Need to Know About the Latest MS Drug Zinbryta

Ocrevus Receives Breakthrough Therapy Designation

Genentech and Roche announced that the Food and Drug Administration has granted Breakthrough Therapy Designation for the investigational medicine ocrelizumab (Ocrevus™) for the treatment of people with PPMS. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening disease and for which preliminary clinical evidence suggests that the drug may demonstrate substantial improvement over existing therapies. Remember that PPMS has no approved therapies.

Breakthrough Therapy Designation was granted to ocrelizumab based on positive results form a pivotal Phase III study (called ORATORIO) which showed treatment with ocrelizumab significantly reduced disability progression and other markers of disease activity compared with placebo in patients with PPMS. Top-line results were of ORATORIO were presented at the 31th ECTRIMS conference in October 2015.

Read this post in its entirety:

FDA Grants Breakthrough Therapy Designation to Treatment for Primary Progressive MS

FDA Warns of PML in Patients Taking Gilenya (fingolimod)

The FDA warns that a case of definite progressive multifocal leukoencephalopathy (PML) and a case of probable PML have been reported in patients taking fingolimod (Gilenya, Novartis) for multiple sclerosis.

“These are the first cases of PML reported in patients taking Gilenya who had not been previously treated with an immunosuppressant drug for MS or any other medical condition,” said an FDA statement released yesterday. “As a result, information about these recent cases is being added to the drug label.”

Gilenya is an immunomodulator shown to benefit patients with relapsing forms of MS. Immunomodulators alter the immune system to reduce inflammation.

Two recent cases of PML in patients taking Gilenya WITHOUT prior exposure to immunosuppressant drugs:
Case #1: A 49-year-old patient with a five year history of MS developed probable PML after taking Gilenya for approximately four years. The patient had previously been treated with interferon beta-1a (Rebif) for 10 months in addition to short-term corticosteroids for relapse, before and during Gilenya treatment. During a routine MRI, new lesions considered atypical for MS and compatible with PML were detected. The patient with probable PML did not have clinical signs or symptoms suggestive of PML, and was diagnosed based on MRI findings and JC virus detected in the cerebrospinal fluid (CSF).

Case #2: A 54-year-old patient with a 13-14 year history of MS developed PML after taking Gilenya for approximately two and a half years. The patient had previously been treated with interferon beta-1b (Betaseron) for approximately 11 years. The patient had also been treated with mesalazine for ulcerative colitis for the last four years. The patient was hospitalized with suspected PML after developing new symptoms, including walking instability, clumsiness, inattention, somnolence and mental sluggishness. At that time, a brain MRI was suggestive of PML, and JC virus DNA was detected in the CSF. The patient was diagnosed with definite PML based on characteristic symptoms, MRI findings, and JC virus in the CSF.

Update: On August 17, 2015, Novartis was informed of a third MS patient taking Gilenya (who had not previously used Tysabri) who developed PML. This patient has a history of colorectal cancer treated with chemotherapy and radiation treatment, as well as Crohn's disease, and Novartis says they are currently in "active discussions with external advisors to review details of this case and the role of various risk factors contributing to the development of PML."

Two prior cases of PML in patients taking Gilenya WITH prior exposure to immunosuppressant drugs:
Case #1: In April 2012, Novartis reported a case of PML in a patient receiving Gilenya who had previously been treated for more than three years with natalizumab (Tysabri, Biogen) before switching to fingolimod. Tysabri exposure in patients who test positive for antibodies to the JC virus is a known risk factor for the development of PML.

Case #2: In August 2013, the FDA reported that a patient developed PML after taking Gilenya for eight months. However, PML could not be conclusively linked to Gilenya because the patient had been treated with: 1) an immunosuppressant drug (azathioprine) prior to starting Gilenya, and 2) multiple courses of intravenous corticosteroids, which can weaken the immune system, before and during Gilenya treatment.

What is PML?
Progressive multifocal leukoencephalopathy (PML) is a rare and serious brain infection caused by the John Cunningham (JC) virus. The JC virus is a common virus that is harmless in most people but can cause PML in some patients who have weakened immune systems, including those taking immunosuppressant drugs. Symptoms of PML are diverse and may include progressive weakness on one side of the body; clumsiness; vision problems; confusion, and changes in thinking, personality, memory and orientation. The progression of deficits can lead to severe disability or death. A magnetic resonance imaging (MRI) scan may find lesions in the brain before these symptoms develop.

What should I do about PML if I take Gilenya?

• Seek medical attention immediately if you experience symptoms that concern you, such as:
- new or worsening weakness
- trouble using your arms or legs
- changes in thinking, eyesight, strength, or balance
• Do not stop taking Gilenya without first talking to your health care professional.
• Read the Medication Guide you receive with your Gilenya prescription.
• Discuss any questions or concerns about Gilenya and the risk of PML with your health care professional.

Healthcare professionals and patients are also encouraged to report adverse events or side effects related to the use of these products to the FDA's MedWatch Safety Information and Adverse Event Reporting Program.

Source:
FDA Drug Safety Communication: FDA warns about cases of rare brain infection with MS drug Gilenya (fingolimod) in two patients with no prior exposure to immunosuppressant drugs [August 4, 2015].

Glatopa: Generic Copaxone Approved by FDA

Before lengthy legal battles between Teva Pharmaceuticals and drug manufacturers eager to claim a portion of the lucrative MS drug market have been completely resolved, the FDA announced late on Thursday, April 16, 2015, approval of the first generic version of Copaxone (glatiramer acetate injection).

Sandoz, a subsidiary of Novartis, has received FDA approval to market Glatopa, a generic version of glatiramer acetate in a 20 mg/ml daily injection. Developed in collaboration with Momenta Pharmaceuticals and produced entirely in the US, Glatopa is indicated for the treatment of patients with relapsing forms of MS, including patients with clinically isolated syndrome.

Mylan Inc, in collaboration with Natco Pharma Ltd, is also working on generic versions of Copaxone. I say versions, plural, because last August 2014, Mylan, Momenta and Sandoz announced that the FDA had accepted their ANDAs (abbreviated new drug application) for three-times-a-week generic Copaxone.

“Health care professionals and patients can be assured that FDA-approved generic drugs have met the same rigorous standards of quality as the brand-name drug,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “Before approving [Glatopa], given its complexity, we reviewed additional information to make sure that the generic product is as safe and effective as the brand name product.”

Read this post in its entirety:

FDA Approves Glatopa (aka generic Copaxone) for Relapsing MS

12 FDA-Approved Treatments for Relapsing Multiple Sclerosis

As of February 2015, members of the relapsing MS community in the US may choose from twelve FDA-approved disease-modifying therapies (DMTs) to slow down the long-term progression of the disease by reducing relapses, number of lesions, and accumulation of physical disability.

Many of the DMTs detailed below are prescribed for people with relapsing forms of MS, including relapsing-remitting MS, as well as secondary-progressive MS and progressive-relapsing MS in those people still having relapses. Select DMTs have also been approved to delay a second exacerbation in people who have been diagnosed with clinically isolated syndrome (CIS).

So far, no DMTs have proven to be effective in progressive forms of MS without relapses.

Oral therapies:

• Aubagio (teriflunomide, 7 mg and 14 mg; pyrimidine synthesis inhibitor) is taken once daily by mouth for relapsing forms of MS; approved in 2012. Sanofi-Genzyme: aubagio.com, msonetoone.com
• Gilenya (fingolimod, 0.5 mg; sphingosine 1-phosphate receptor modulator) is taken once every day by mouth for relapsing forms of MS; approved in 2010. Novartis: gilenya.com
• Tecfidera (dimethyl fumarate, 240 mg; Nrf2 activator) is taken twice daily by mouth for relapsing forms of MS; approved in 2013. Biogen Idec: tecfidera.com, msactivesource.com

Injectable therapies:

• Copaxone (glatiramer acetate, 20 mg/mL and 40 mg/mL; synthetic polypeptide) is taken by subcutaneous injection every day (20 mg dose) or three days each week (40 mg dose) for CIS and relapsing forms of MS; approved in 1996; auto-injector available. (Generic options may become available soon.) Teva Neuroscience: copaxone.com, sharedsolutions.com
• Avonex (interferon beta-1a, 30 mcg/.5 mL) is taken once weekly by intramuscular injection for CIS and relapsing forms of MS; approved in 1996; auto-injector and dose titration available. Biogen Idec: avonex.com, msactivesource.com
• Betaseron (interferon beta-1b, 0.25 mg/mL) is taken every other day by subcutaneous injection for CIS and relapsing forms of MS; approved in 1993. Solution must be mixed before injection. Bayer HealthCare: betaseron.com
• Extavia (interferon beta-1b, 0.25 mg/mL) is taken every other day by subcutaneous injection for CIS and relapsing forms of MS; approved in 2009. Solution must be mixed before injection; dose titration available. Novartis: extavia.com
• Plegridy (peginterferon beta-1a, 0.125 mg/.5 mL) is taken every 14 days by subcutaneous injection for relapsing forms of MS; approved in 2014; auto-injector available. Biogen Idec: plegridy.com, msactivesource.com
• Rebif (interferon beta-1a, 22 mcg/.5 mL and 44 mcg/.5 mL) is taken three days each week by subcutaneous injection for relapsing forms of MS: approved in 2002; auto-injector and dose titration pack available. EMD Serono/Pfizer: rebif.com, mslifelines.com

Infusion therapies:

• Lemtrada (alemtuzumab, 12 mg; CD52 monoclonal antibody) is delivered by intravenous infusion on five consecutive days, followed by another three consecutive days one year later, for relapsing forms of MS and generally reserved for people with MS who have failed other treatments; approved in 2014. Sanofi-Genzyme: lemtrada.com
• Novantrone (mitoxantrone, 140 mg; antineoplastic anthracenedione) is delivered by intravenous infusion once every 3 months with a lifetime maximum limit of 8-12 doses over 2-3 years for worsening relapsing-remitting MS, progressive-relapsing MS, or secondary-progressive MS; approved in 2000. Available as generic drug since 2006. EMD Serono/Immunex Corp.
• Tysabri (natalizumab, 300 mg; α4β1-integrin monoclonal antibody) is delivered by intravenous infusion once every four weeks at a registered infusion center for relapsing forms of MS; approved in 2006. Must not be combined with other disease-modifying therapies. Biogen Idec: tysabri.com, msactivesource.com

If one DMT doesn’t work for you, or is intolerable, discuss other options with your neurologist.

Republished from:
MS Patients Have 12 Disease-Modifying Therapeutic Choices

November 2014 Round-Up of MS News and Research

• Details of PML Death to be Added to Tecfidera Prescription Label
• FDA Reverses Previous Rejection and Approves MS Drug Lemtrada
• New Italian Study: Azathioprine is Not Inferior to Beta Interferon in Treating RRMS
• Other Studies of Interest

Read this post in its entirety:
Hot Topics and MS Research News for November 2014

Lemtrada (alemtuzumab) Approved by the FDA for Relapsing-Remitting MS in the US

The FDA approves Lemtrada for the treatment of relapsing-remitting MS. Read the press release below. The drug will be priced at $158,000 for two courses of treatment over two years, reports The Boston Globe. Lemtrada is administered by infusion over 5 consecutive days followed by infusions given over 3 consecutive days 12 months later. The Boston Globe continues, "Rebif, a competing drug compared with Lemtrada in clinical studies, costs $134,600 for a similar treatment regimen, or 17 percent less. But the Genzyme executives pointed out that patients on Lemtrada suffered 50 percent fewer relapses than those taking the other drug."

Genzyme’s Lemtrada Approved by the FDA

November 14, 2014

- Approval Establishes Genzyme’s MS Franchise in the U.S. with Two Approved Products; Follows Global Approvals -

Genzyme, a Sanofi company, announced today that the U.S. Food and Drug Administration (FDA) has approved Lemtrada^TM (alemtuzumab) for the treatment of patients with relapsing forms of multiple sclerosis (MS). Because of its safety profile, the use of Lemtrada should generally be reserved for patients who have had an inadequate response to two or more drugs indicated for the treatment of MS.

“Today’s approval is the culmination of more than a decade of work by Genzyme to develop Lemtrada,” said Genzyme President and CEO, David Meeker. “Lemtrada demonstrated superior efficacy over Rebif on annualized relapse rates in the two studies which were the basis for approval. A comprehensive risk evaluation and mitigation strategy (REMS) will be instituted in order to help detect and manage the serious risks identified with treatment.”

The FDA approval of Lemtrada is based on two pivotal randomized Phase III open-label rater-blinded studies comparing treatment with Lemtrada to Rebif^®(high-dose subcutaneous interferon beta-1a) in patients with relapsing remitting MS who were either new to treatment (CARE-MS I) or who had relapsed while on prior therapy (CARE-MS II).

In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates; the difference observed in slowing disability progression did not reach statistical significance. In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates, and accumulation of disability was significantly slowed in patients given Lemtrada vs. interferon beta-1a. The clinical development program for Lemtrada involved nearly 1,500 patients with more than 6,400 patient-years of safety follow-up.

“The unmet need in MS remains high,” said Edward Fox, M.D., Ph.D., Director of the Multiple Sclerosis Clinic of Central Texas. “It is a great day for people living with relapsing forms of MS in the United States, who will now have access to this new meaningful treatment”.

The Lemtrada label includes a boxed warning noting a risk of serious, sometimes fatal autoimmune conditions, serious and life-threatening infusion reactions and also noting Lemtrada may cause an increased risk of malignancies including thyroid cancer, melanoma and lymphoproliferative disorders.

Lemtrada is only available through a restricted distribution program, the Lemtrada REMS (Risk Evaluation and Mitigation Strategy). This program has been developed to ensure that access to Lemtrada in the U.S. is only through certified prescribers, healthcare facilities and specialty pharmacies and to also ensure that patients are enrolled in the REMS program. The program is intended to help educate healthcare providers and patients on the serious risks associated with Lemtrada and the appropriate periodic monitoring required to support the detection of these risks for 48 months after the last infusion. The REMS is based on a developmental risk management program that was successfully implemented in the Phase 2 and Phase 3 trials and allowed for early detection and management of some of the serious risks associated with Lemtrada.

“The FDA approval of Lemtrada is a significant milestone for people living with relapsing MS in the United States,” said Dr. Timothy Coetzee, Chief Advocacy, Services and Research Officer at the National MS Society. “We are pleased that the voices of the MS community have been recognized and that people with relapsing MS will now have access to a new, needed treatment option.”

Lemtrada has a unique dosing and administration schedule of two annual treatment courses. The first treatment course is administered via intravenous infusion on five consecutive days, and the second course is administered on three consecutive days, 12 months later.

The most common side effects of Lemtrada are rash, headache, pyrexia, nasopharyngitis, nausea, urinary tract infection, fatigue, insomnia, upper respiratory tract infection, herpes viral infection, urticaria, pruritus, thyroid gland disorders, fungal infection, arthralgia, pain in extremity, back pain, diarrhea, sinusitis, oropharyngeal pain, paresthesia, dizziness, abdominal pain, flushing, and vomiting. Other serious side effects associated with Lemtrada include autoimmune thyroid disease, autoimmune cytopenias, infections and pneumonitis.

First approved in September 2013 in the European Union, Lemtrada is approved in more than 40 countries. Additional marketing applications for Lemtrada are under review by regulatory agencies around the world.

The FDA approval of Lemtrada marks Genzyme’s second MS treatment approval in the United States. Genzyme received FDA approval of its once-daily, oral Aubagio^® (teriflunomide) for the treatment of relapsing forms of MS in September 2012. Aubagio is approved in more than 50 countries, and is under review by additional regulatory agencies. Between clinical trials and commercial use, approximately 30,000 patients have been treated with Aubagio.

Multiple sclerosis is estimated to affect more than 2.3 million people globally. There are approximately 400,000 people living with MS in the United States.

Important Safety Information About Lemtrada for U.S. Patients

Serious and life-threatening autoimmune conditions such as immune thrombocytopenia (ITP) and anti-glomerular basement membrane disease can occur in patients receiving Lemtrada. Monitor complete blood counts with differential, serum creatinine levels, and urinalysis with urine cell counts at periodic intervals in patients who receive Lemtrada. Lemtrada is associated with serious and life-threatening infusion reactions. Lemtrada can only be administered in certified healthcare settings that have on-site access to equipment and personnel trained to manage anaphylaxis and serious infusion reactions. Lemtrada may be associated with an increased risk of malignancy, including thyroid cancer, melanoma and lymphoproliferative disorders. The Lemtrada REMS Program, a comprehensive risk management program with frequent monitoring, is being implemented to help mitigate these serious risks.

The Lemtrada label includes a boxed warning noting a risk of serious, sometimes fatal autoimmune conditions, serious and life-threatening infusion reactions and also noting Lemtrada may cause an increased risk of malignancies including thyroid cancer, melanoma and lymphoproliferative disorders. Lemtrada is contraindicated in patients with Human Immunodeficiency Virus (HIV) infection.

U.S. Indication and Usage

Lemtrada is indicated for the treatment of patients with relapsing forms of multiple sclerosis (MS). Because of its safety profile, the use of Lemtrada should generally be reserved for patients who have had an inadequate response to two or more drugs indicated for the treatment of MS.

Please click here for full U.S. Prescribing Information for Lemtrada, including boxed warning and contraindications.

As part of its continued commitment to MS patients, Genzyme’s MS One to One® program will provide information about multiple sclerosis, Lemtrada and other relevant resources. MS One to One is available and staffed by dedicated MS nurses and highly trained representatives who can provide support for individuals living with MS, their health care providers, family and loved ones. For more information about these support services, call the MS One to One line at 1-855-MSOne2One (1-855-676-6326) Monday through Friday, from 8:30am – 8:00pm ET. Information and support are also available atwww.MSOnetoOne.com.

About Lemtrada™ (alemtuzumab)

Alemtuzumab is a monoclonal antibody that targets CD52, a protein abundant on T and B cells. Circulating T and B cells are thought to be responsible for the damaging inflammatory process in MS. Alemtuzumab depletes circulating T and B lymphocytes after each treatment course. Lymphocyte counts then increase over time with a reconstitution of the lymphocyte population that varies for the different lymphocyte subtypes.

In CARE-MS I, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rate (0.18 for Lemtrada and 0.39 for interferon beta-1a (p<0 -6.5="" .0001="" 1-a="" 11="" 30="" 55="" 59="" 78="" a="" and="" at="" baseline="" beta-1a="" beta="" change="" did="" difference="" disability="" for="" from="" in="" interferon="" lemtrada="" lesion="" not="" observed="" of="" p="0.31).<u" patients="" percent.="" percent="" progression="" proportion="" reach="" reduction.="" reduction="" relapse-free="" relative="" remaining="" risk="" significance="" statistical="" t2="" the="" two="" volume="" vs.="" was="" with="" year="">

In CARE-MS II, Lemtrada was significantly more effective than interferon beta-1a at reducing annualized relapse rates (0.26 for Lemtrada and 0.52 for interferon beta 1-a, p<0 -1.2="" .0001="" 1-a="" 21="" 42="" 47="" 49="" 65="" a="" and="" at="" baseline="" beta-1a="" beta="" change="" confirmed="" did="" disability="" for="" from="" in="" interferon="" lemtrada="" lesion="" lower="" not="" of="" p="0.14).<u" patients="" percent="" progression="" proportion="" reach="" reduction.="" reduction="" relapse-free="" relative="" remaining="" risk="" significance="" significantly="" six-month="" statistical="" t2="" the="" two="" volume="" vs.="" was="" with="" year="">

Genzyme holds the worldwide rights to alemtuzumab and has responsibility for its development and commercialization in multiple sclerosis. Bayer Healthcare receives contingent payments based on global sales revenue.

About Aubagio^® (teriflunomide)

Aubagio is an immunomodulator with anti-inflammatory properties. Although the exact mechanism of action for Aubagio is not fully understood, it may involve a reduction in the number of activated lymphocytes in the central nervous system (CNS). Aubagio is supported by one of the largest clinical programs of any MS therapy, with more than 5,000 trial participants in 36 countries. Some patients in extension trials have been treated for up to 10 years.

U.S. Indication and Usage

Aubagio (teriflunomide) is a once-daily, oral therapy indicated for the treatment of adult patients with relapsing forms of multiple sclerosis. The recommended dose of Aubagio is 7 mg or 14 mg orally once-daily.

Important Safety Information About Aubagio for U.S. Patients

The Aubagio label includes the risk of hepatotoxicity and, teratogenicity (based on animal data). In the United States, this information can be found in the boxed warning.

In MS clinical studies with Aubagio, the incidence of serious adverse events were similar among Aubagio and placebo-treated patients. Serious events may include decreased white blood cell count, peripheral neuropathy, hyperkalemia, skin reactions and increased blood pressure. The most common adverse events associated with Aubagio in MS patients included increased ALT levels, alopecia, diarrhea, influenza, nausea and paresthesia.

Teriflunomide is the principal active metabolite of leflunomide, which is indicated in the U.S. for the treatment of rheumatoid arthritis. Severe liver injury including fatal liver failure has been reported in patients treated with leflunomide. ALT should be monitored monthly for at least 6 months in patients who start treatment with Aubagio.

Aubagio is contraindicated in patients with severe hepatic impairment, pregnant women and women of childbearing potential who are not using reliable contraception and in patients who are taking leflunomide. Aubagio is not recommended for breast-feeding women, patients with immunodeficiency states, patients with significantly impaired bone marrow function or significant anemia, leucopenia, neutropenia or thrombocytopenia, patients with severe active infection until resolution, patients with severe renal impairment undergoing dialysis and patients with hypoproteinaemia.

Please click here for full U.S. Prescribing Information for Aubagio, including boxed warning and contraindications.

About Genzyme, a Sanofi Company

Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years. We accomplish our goals through world-class research and with the compassion and commitment of our employees. With a focus on rare diseases and multiple sclerosis, we are dedicated to making a positive impact on the lives of the patients and families we serve. That goal guides and inspires us every day. Genzyme’s portfolio of transformative therapies, which are marketed in countries around the world, represents groundbreaking and life-saving advances in medicine. As a Sanofi company, Genzyme benefits from the reach and resources of one of the world’s largest pharmaceutical companies, with a shared commitment to improving the lives of patients. Learn more atwww.genzyme.com.

About Sanofi

Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients’ needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and the new Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).

Genzyme^®, Aubagio^® and MS One to One^® are registered trademarks, and Lemtrada^TM is a trademark of Genzyme Corporation. Rebif^® is a registered trademark of EMD Serono, Inc. All rights reserved.

[Press Release]

Tecfidera (BG12) Approved by FDA for Relapsing MS

The long anticipated oral MS drug known as BG12 has been approved by the FDA for use in relapsing forms of multiple sclerosis.  The new drug will be sold by Biogen Idec under the brand name Tecfidera (dimethyl fumarate).

Tec-fi-dera is an oral drug which is taken twice daily with or without food.  Take Tecfidera exactly as your doctor instructs you to take it.  Biogen states that the recommended starting dose is one 120mg capsule taken by mouth 2 times a day for 7 days.  The recommended dose after 7 days is one 240 mg capsule taken by mouth 2 times a day.  Swallow Tecfidera whole, without crushing, crewing, or sprinkling capsule contents on food.

What are the possible side effects of Tecfidera?

Read this post in its entirety:
Tecfidera (BG12), A New Oral Medication for Relapsing Forms of MS

New FDA Warnings for Xenical and Alli

Last week the U.S. Food and Drug Administration (FDA) announced the approval of revised labeling for the prescription weight-loss drug Xenical (orlistat 120 mg) and its OTC version Alli (orlistat 60 mg). 

The revised label carries new safety information regarding cases of severe liver injury that have been reported with the use of orlistat.  It is important to note that these cases have occurred only rarely, estimated at 13 cases out of the approximate 40 million people worldwide who have used this medication since its approval in 1999 (Xenical) and 2007 (Alli).

Of the 13 cases of severe livery injury, 12 were foreign reports associated with the use of Xenical and one was a U.S. report associated with Alli use.  Two patients died from liver failure and three patients required liver transplants.  A causal relationship has not been established as other factors or drugs may have contributed to the development of severe liver injury.

Read this post in its entirety:

Weight Loss Drugs, Xenical and Alli, Receive New FDA Warning Label

From the FDA

Reporting Adverse Events to FDA's MedWatch Program:


Health Fraud Awareness (Consumer Update):

Report Adverse Events to MedWatch (FDA)

The FDA Safety Information and Adverse Event Reporting Program

“Your FDA gateway for finding clinically important safety information and reporting serious problems with human medical products.”

Consumers and the Food and Drug Administration (FDA)

Consumers play an important public health role by reporting to the FDA any adverse events (unexpected side effects) after using a medical product, or other problems with any products that the agency regulates. Timely reporting allows the agency to take prompt action. There are a number of ways you can report problems to the agency, depending on the type of problem and product.

What is MedWatch?

MedWatch is the Food and Drug Administration's (FDA) program for reporting serious reactions, product quality problems, therapeutic inequivalence/failure, and product use errors with human medical products, such as drugs and medical devices.

Read this post in its entirety:
Report Adverse Events Directly to the FDA MedWatch Program

FDA, Social Media, and Pharma Ads

I’m looking for your thoughts and opinions on a matter which may directly impact us as patients and the manner in which we get information. This Thursday and Friday (November 12-13, 2009), the Food and Drug Administration (FDA) is holding a public hearing “on how pharmaceutical companies use the web and social-media tools to market their products - the first step to providing guidelines around this type of marketing. Sixty-two speakers from pharmaceutical companies, media companies, agencies, and others have been asked to give their viewpoints.”

I’ve been following the lead-up to this event fairly closely as the CEO of HealthCentral, Chris Schroeder, will be speaking during two sessions. HealthCentral is even hosting cocktails after the first day of hearings. (invitation seen here) Have I ever mentioned that I live only about 4 miles from HealthCentral's Arlington, VA, offices? I do, although I've still not been over there to check things out in person.

Read this post in its entirety and please comment:
FDA, Social Media, and Pharmaceutical Advertising

FDA Accepts Fampridine-SR NDA For Review

FOR IMMEDIATE RELEASE:

Acorda Therapeutics Announces FDA Acceptance of Fampridine-SR New Drug Application for Filing

• FDA Assigns Priority Review and PDUFA Date of October 22, 2009
• No Current Therapies Indicated to Improve Walking Ability in People with MS

HAWTHORNE, N.Y., May 6, 2009 – Acorda Therapeutics, Inc. (Nasdaq: ACOR) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Fampridine-SR New Drug Application (NDA) for filing, assigning Priority Review and a Prescription Drug User Fee Act (PDUFA) date of October 22, 2009. The PDUFA date is the target date for the FDA to complete its review of the Fampridine-SR NDA.

“I am pleased that we were able to work quickly to address the comments from the FDA and resubmit our NDA approximately three weeks from having received the Refuse to File letter on our initial NDA submission, and that the FDA accepted the filing less than two weeks later,” said Ron Cohen, M.D., Acorda Therapeutics’ President and CEO. “We are also encouraged that the FDA has elected to assign Priority Review status to the Fampridine-SR NDA.”

About Fampridine-SR
Fampridine-SR is a sustained-release tablet formulation of the investigational drug fampridine (4-aminopyridine or 4-AP). In laboratory studies, fampridine has been found to improve impulse conduction in nerve fibers in which the insulating layer, called myelin, has been damaged. Fampridine-SR is being developed by Acorda Therapeutics and manufactured by Elan Corporation plc.

About Acorda Therapeutics
Acorda Therapeutics is a biotechnology company developing therapies for spinal cord injury, multiple sclerosis and related nervous system disorders. The Company's marketed products include Zanaflex Capsules® (tizanidine hydrochloride), a short-acting drug for the management of spasticity. The Company's pipeline includes a number of products in development for the treatment, regeneration and repair of the spinal cord and brain.

About Elan Drug Technologies
Elan Drug Technologies (EDT) is the world’s leading drug delivery company and is a business unit of Elan (NYSE:ELN). EDT developed Fampridine-SR, using one of their proprietary Oral Controlled Release Technologies, the MXDAS® (MatriX Drug Absorption System) Technology. Products developed by EDT aim to deliver clinically meaningful benefits to patients by using their extensive experience and proprietary delivery technologies in partnership with pharmaceutical companies. More information is available at www.elandrugtechnologies.com

Generic Biotech Drugs -- The House Energy and Commerce Committee meeting to consider establishing a Regulatory Pathway for Follow-On Biologic Drugs

"The Access to Life-Saving Medicine Act — introduced by Congressman Henry Waxman (CA) — would provide a statutory pathway for the Food and Drug Administration (FDA) to review and approve generic biologic therapies."

The cost of biological multiple sclerosis treatments — $16,500 to $29,000 each year — can unfortunately force many people to stop their prescribed therapy because they just cannot afford it. More affordable, generic options for this fast-growing and expensive category of drugs are not yet available. Many people living with MS and other diseases depend on biological drugs to sustain or improve their quality of life. For MS, those therapies include Avonex, Betaseron, Copaxone, Novantrone, Rebif, and Tysabri.

It is the case too often that prescription assistance programs are insufficiently funded, have extremely strict eligibility requirements, or simply do not offer assistance for these biologic drugs due to the extreme cost. Listen to a recent NPR broadcast on this issue (transcript provided below). It includes the story of Donna Gosbee of Wyoming who lives with MS and her struggle to afford therapy.

Biologic (also known as biological or biotech) drugs are produced from living cell cultures rather than synthesized chemically. The generic drugs that are currently available are synthetically exact copies of the brand name original, based on a precise chemical composition. Generic versions of biologic drugs, on the other hand, would need to allow for nuances in the cell cultures while meeting certain parameters that are strict enough to ensure they are just as safe and effective as the originals.

The U.S. Senate has passed legislation that would establish a course for approving safe, effective, affordable, and comparable versions of biologic therapies for MS and other diseases. Unfortunately, the House still has not yet taken up a similar bill. Members of the House of Representatives will be meeting this month to talk about creating a pathway for the approval of follow-on biological drugs. Reports indicate that members of the House Energy and Commerce Committee will meet on October 31 to discuss the issue.

It is imperative that the House Energy and Commerce Committee take action on this bill, where Congressman John Dingell (MI) and the rest of the committee will help shape the final legislation. The time for Congress to take action is now. The Access to Life-Saving Medicine Act — introduced by Congressman Henry Waxman (CA) — would provide a statutory pathway for the Food and Drug Administration (FDA) to review and approve generic biologic therapies.

Take a minute to write your Representative today. Ask them to move forward with the Access to Life-Saving Medicine Act (H.R. 1038). Congress can provide the FDA with a pathway for approving safe, effective, and lower-cost versions of biologic drugs. And give people living with MS and other diseases more affordable options for therapy.

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History of the Access to Life-Saving Medicine Act (H.R.1038)

On February 14, 2007, Rep. Waxman, Rep. Jo Ann Emerson, and Rep. Frank Pallone, Jr., along with Senators Charles E. Schumer and Hillary Rodham Clinton introduced H.R. 1038, the “Access to Life-Saving Medicine Act,” which will establish a process through which the FDA will be able to approve lower cost copies of biotech drugs, also known as biologics or biopharmaceuticals. Other original co-sponsors are Reps. Rahm Emanuel and Mazie Hirono, and Senators David Vitter, Susan M. Collins, Patrick J. Leahy, and Debbie Stabenow. Biotech drugs, which are produced from living cell cultures rather than synthesized chemically, are among the fastest growing and most expensive components of the nation’s drug bill. Currently there is no statutory pathway for biotech drugs, and manufacturers of biotech drugs can charge monopoly prices, indefinitely.

Generic drugs (first made possible under the 1984 Hatch-Waxman Amendments) have been extremely successful in bringing down the high cost of prescription drugs. Generic drugs save patients and payers $10 billion a year. But there is no generic competition for one of the fastest growing and most expensive category of drugs: so-called biotech drugs, also known as biological drugs or biopharmaceuticals. It is common for these drugs to cost tens of thousands of dollars a year, even after patent expiration. Many patients are now denied access to these important drugs because even the co-payments can reach thousands of dollars a year. And the sky-rocketing cost of biotech medicines is imposing increasing burdens on employers, insurers, and the federal government. Introducing fair competition for biotech drugs is essential to keep these life-saving treatments affordable.
Press Release - Rep. Waxman's Statement - Background on Biologics - Bill Summary - Quick Summary - Bill Text - Letters of Support

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NPR Health & Science: All Things Considered, August 9, 2007

Are Generic Biotech Drugs Coming Soon?
by Joanne Silberner

Correction: At the time this story aired neither the House nor Senate had voted on legislation. There have been two hearings in subcommittees of the House of Representatives, and a Senate committee has approved legislation.

All Things Considered, August 9, 2007 · While Congress is away this month, House and Senate staffers are working on a bill aimed at helping patients who rely on innovative biotech drugs that can cost as much as several thousand dollars per month.

Generic versions of the drugs would surely be cheaper, but opponents of generic versions say that making sure the knockoffs are safe and effective may be trickier than it sounds.

Donna Gosbee, 51, came from Wyoming to Washington, D.C., to attend a meeting of the Multiple Sclerosis Society. She is using a walker.

"I never know from one day to the next until I put my feet down on the floor whether I'm going to be able to walk," Gosbee says.

Gosbee was diagnosed with multiple sclerosis four years ago.

"The doctors put me on one of the biologics, Betaseron," she says. "I've been on it for three years now and this drug costs $1,500 a month."

The government helps Gosbee with some of the cost, but she's worried the help won't always be there. So she came to Washington to lobby Congress.

Lobbying for Change

"I'm going to talk to Wyoming legislators to try to convince them that medications are just out of reach of the normal person," Gosbee says.

A generic could significantly cut her medical bill. At www.drugstore.com, for example, the generic version of the statin drug, Mevacor, is only one-third of Mevacor's price.

But Gosbee's drug is manufactured very differently — by living, bioengineered cells.

They make molecules that are much bigger than most conventional drugs, and a lot more complex, says Roger Williams, CEO of U.S. Pharmacopeia, a nonprofit organization that sets standards for drug manufacturers.

"If I showed you on a page the molecular structure of a biologic, it might cover two pages just of carbons and nitrogens and hydrogens and oxygens," Williams says.

In contrast, he says the structure of many conventional drugs fill less than half a page.

Williams says making conventional drugs is like snapping together Tinker Toys. You add one chemical after another, and the manufacturer is very much in control.

Making a biotech drug is more like farming. You start with a living cell, then keep the temperature, the nutrients and other growing conditions just right. Just as wheat grown from a different seed stock or under different conditions will produce slightly different grain, different versions of biotech drugs can vary — even when made by the same company.

The Process of Biotech

In Gaithersburg, Md., a company called MedImmune makes the biotech drug Synagis. The drug fights a virus called RSV that can kill premature babies.

Just getting into MedImmune takes work — you have to put on gloves, booties and a double set of what look like surgical scrubs.

You must also go through multiple airlocks. Sterility is important for conventional drugs, too, but it's especially important here. If bacteria or viruses get into the cell cultures, they could disrupt the manufacturing process and wind up in the final drug.

Workers take large beakers of living cells in culture from a refrigerator. The cells go into gleaming metal vats the size of small cars.

"This is the start of our bioreactor train," explains Tony Luttrell, MedImmune's vice president.

There are enough pressure monitors and temperature gauges and pipes to make a science-fiction movie proud.

Conventional manufacturers use temperature gauges too, as well as vats filled with buckets of chemicals.

But these biotech vats hold mouse cells, each one loaded up with human DNA. The human DNA is directing the cells to produce a particular protein — an antibody that can fight RSV.

If you want to make a copy of MedImmune's drug, you would have to insert some human DNA into mouse cells in a precise way — a way that will produce that protein. It's a real challenge.

"One little genetic change, one little change in the way the protein is configured or the way the proteins fold onto each other may have an effect on how it actually acts in the clinic," Luttrell says.

Then you have to be really careful about how you grow the cells, Williams says. For example, temperature can make a big difference. Look at albumen — the protein in egg whites.

"If you take egg white and cook it, that's OK to eat, but it's no good to the chicken anymore," Williams says.

Generic Outlook

Williams thinks some biotech drugs could be made generically. But the only way to know for sure that a different version would be just as safe and effective is to perform lengthy and expensive testing in animals or people — something that's not required for conventional generics.

There have been two hearings in subcommittees of the House of Represenatives, and a Senate committee has approved legislation. Staffers are now working to develop legislation that can win approval.

But don't hold your breath — companies that make brand name drugs are still adamant that testing in people should be required.

Patients and generic companies waiting for less expensive drugs aren't happy either. Both bills would make generic companies wait 12 years before they could market a cheaper drug, not much different from conventional generics, but a long time when you're spending several thousand dollars a month to fill a prescription.

Copyright 2007 NPR

Questcor uses the Orphan Drug Act as a strategy tool to boost marketing and development

"Abusing the orphan drug law to rip off customers" by David Williams, Health Business Blog. When I read Williams' article, I became furious, not because I am familiar with Infantile Spasms (IS), but because the families affected by IS will be facing an outrageous financial battle. On August 30, 2007, the Epilepsy Foundation announced "Dramatic Increase Seen in Cost of H.P.Acthar Gel."

Acthar was developed in the 1950's for the treatment of exacerbations in Multiple Sclerosis. In 1997, the FDA recognized the need to continue manufacturing of H.P.Acthar Gel by Centour for use in the treatment of Infantile Spasms. In August 2006, Questcor Pharmaceuticals, Inc. submitted sNDA for FDA approval to include treatment of IS on the label of H.P.Acthar Gel which had been acquired from Aventis in 2001. On May 14, 2007, Questcor announced that the FDA did not approve the sNDA. One week later, Questcor CEO James Fares resigns.

On August 27, 2007, Questcor board announces New Strategy and Business Model for H.P. Acthar Gel.
Questcor will initiate a new pricing model, create an expanded safety net for patients using Acthar, and provide a group of Medical Science Liaisons to work with health care providers who are administering Acthar.
"...the goal of Questcor's new strategy is to make manufacturing and distribution of Acthar economically viable on a stand-alone basis, so that Questcor can continue to ensure the availability of Acthar for those patients who need it most and fund projects which can contribute to the growth of the company."
Previously, the FDA granted Orphan Designation to H.P.Acthar Gel for the treatment of IS. As a result of this Orphan Designation, if Questcor is successful in obtaining FDA approval for the IS indication, Questcor will also qualify for a seven year exclusivity period...
The cost for a course of treatment could approach $80,000-$100,000.

The term "orphan drug" refers to a product that treats a rare disease affecting fewer than 200,000 Americans. The Orphan Drug Act was signed into law on January 4, 1983. The intent of the Orphan Drug Act is to stimulate the research, development, and approval of products that treat rare diseases.

Now the issue of "rare diseases" is one which I do have personal knowledge. I have MS and I use an expensive self-injectible drug. The safety net for that drug is administered by National Organization for Rare Disorders (NORD), which is the same safety net Questcor now uses for H.P.Acthar Gel. However, this 'safety net' has big holes and many families will likely fall right through. If you've read my other posts, you are aware that NORD finally awarded full assistance in receiving that $21,000 drug after my earnings sunk below $20,000.

So this is what I suspect happened sometime before 2001 (of course I have no proof):

• Investor A looks at the drug industry and thinks to himself, "where is there a better opportunity to make some money?"
• Investor B - "hey, the neurological med field is booming...just look at the success of the self-injectible MS meds. They are industry blockbusters."
• Investor C - "yeah, those MS drugs are working so well that the old standby exacerbation drugs, like Aventis' H.P.Acthar Gel, aren't doing as well."
• Investor B - "you're right, but the H.P.Acthar Gel is working wonders for Infantile Spasms."
• Investor C - "you know, it's the specialty pharms which are raking in the money. We could do that with the Acthar Gel, if we owned it."

In 2001, Questcor acquires H.P.Acthar Gel from Aventis. In 2002, the FDA uses Questcor's Acthar Gel as an example of how they respond to drug shortages which affect seriously ill patients. In Oct 2003, the FDA grants 'orphan drug' status to Acthar Gel for the treatment of Infantile Spasms. In November 2003, Questcor and IDIS enter into an exclusive agreement for distribution outside of the U.S. Between 2001 and 2005, Questcor makes several strategic moves to consolidate Acthar manufacturing and distribution.

In February 2005, Questcor names James L. Fares President and CEO, followed by the announcement in April of a New Business Strategy -- Company to Focus on Neurology Products." Between May 2005 and September 2005, Questcor hires Craig Chambliss as Vice President of Sales & Marketing, Gregg Lapointe to the Board of Directors, and George Stuart as Chief Financial Officer. In October 2005, Questcor sells three of their products for $28.3 Million earning them enough money to pay off much debt and focus on realizing their grand vision. In May 2006, Questcor acquires Doral(R) giving them a Second Product for National Neurology Sales Force. In December 2006, Questcor presents data on H.P.Acthar Gel at American Epilepsy Society annual meeting. In May 2007, FDA rejects sNDA application and James Fares resigns.

So in August 2007, Questcor finally announces their new strategy which will effectively drain as much profit as possible out of A.P.Acthar Gel. Whether from private insurance, Medicare, SCHIP, Medicaid, or the patient...it doesn't matter as long as Questcor is able to...

..."fund projects which can contribute to the growth of the company."

Job well done clever investors.

So sorry patients.

Aargh!!!!

http://www.acthar.com/