Friday, January 21, 2011

European Medicines Agency recommends Gilenya (fingolimod), but rejects Fampyra (fampridine) and Movectro (cladribine)

Today was a big day for news of new Multiple Sclerosis medications in Europe.

While online publications have been excited about the recommendation by the American Academy of Neurology that plasmapheresis (plasma exchange) can be used to treat MS relapses which do not respond to high-dose steroids (something which I was under the impression neurologists already had in their tool belt), the European Medicines Agency's or EMA Committee for Medicinal Products for Human Use (CMPH) has made decisions regarding the newer pills designed for use in MS treatment (both disease-modifying and symptomatic).


The Committee adopted a positive opinion recommending the grant of marketing authority for Novartis' drug Gilenya (fingolimod).  Gilenya is a an oral (pill) disease-modifying drug which received FDA approval in September 2010.  Patients in the US have more recently been able to gain access to the drug.  In the summary of opinion, the Committee mentions that the benefits with Gilenya are its ability in reducing the number of relapses in patients with relapsing-remitting multiple sclerosis.

The most common side effects (seen in more than 1 patient in 10) are influenza viral infections, headache, diarrhoea, back pain, cough and elevated liver enzyme (known as ALT). Other common side effects (seen in between 1 and 10 patients in 100) that could be or could become serious are herpes virus infection (shingles or herpes zoster), lymphopenia, leucopenia (reduced white blood cells), bradycardia (slow heartbeat), atrioventricular block (irregular heart rhythm), bronchitis and gastroenteritis.

FAMPYRA/Biogen Idec

A negative opinion was delivered to Biogen Idec regarding Fampyra (fampridine) which is marketed in the United States as Ampyra (dalfampridine) by Acorda Therapeutics.  The Committee recommends that marketing authority should not be granted to Biogen for Fampyra, which is intended to be used to improve the walking ability of adult patients with multiple sclerosis.  The Committee was not convinced that Fampyra’s small effect on the walking speed was a meaningful benefit for patients. The effect on speed could not be linked to meaningful improvements such as better coordination, balance or stamina or increased range of action.

The Committee was of the view that the medicine’s uncertain benefits did not outweigh its side effects which included pain, dizziness, paraesthesia (unusual sensations like pins and needles) and problems with balance, as well as symptoms similar to those of multiple sclerosis that could impair the patient’s ability to walk. The Committee also noted the lack of adequate long-term data on the medicine’s benefits and safety as well as data on some groups of patients, such as the elderly and patients with epilepsy or heart problems.

As of September 30, 2010, Ampyra has been prescribed to 31,000 patients with MS in the US since it's FDA approval on January 22, 2010 and commercial launch in March 1, 2010.  Biogen Idec plans to appeal the Committee's decision.  Biogen owns the rights to market Fampyra/Ampyra outside of the United States.


Merck/Serono received greater bad news in that the Committee confirmed its previous negative opinion and adopted a final negative opinion regarding Movectro (cladribine).  Serono shall not be granted the authority to market Movectro (cladribine) as a disease-modifying therapy in relapsing remitting multiple sclerosis.

Previously, Merck/Serono had presented the results of one main study that compared Movectro with placebo (a dummy treatment) in 1,326 patients with relapsing-remitting MS.  The Committee was concerned that an increased number of patients with cancer were observed patients treated with Movectro compared to the control group in the clinical trial. The Committee also noted that the benefits and the most appropriate dosage for treatment had not been fully established in patients who were expected to use the medicine. Therefore, the Committee is of the opinion that the benefits of Movectro do not outweigh its risks.

According to Reuters, Merck said it remained committed to completing ongoing clinical trials with cladribine tablets. It can re-apply for approval of the tablets in 2012 at the earliest, when trial results are due.

I personally have no opinion regarding any of these medications for my own use.  I am highly unlikely to use any of them due to my history of methotrexate use (for fingolimod and cladribine) and due to insurance coverage for Ampyra (dalfampridine).

However, I am disappointed that these treatment options will not be made available to patients for whom they may benefit.  I know that many MS patients in Europe have been eager to try Fampyra as we have received many questions at HealthCentral asking when it will be made available.  Those patients will have to wait a while longer now.

More information about the Committee's decisions is available in separate question-and-answer documents on the Agency's website.  PDF's can be found in the right-hand sidebar of the website.

UPDATE: A good compilation of information and links to relevant press releases can be found at FiercePharma.  Read MS drugs get yeas, nays and price increases.

UPDATE #2: Biogen announces updated Tysabri/PML statistics.  The total number of cases is now at 85 patients with the addition of 6 new cases reported in January 2011.  The number of deaths holds at 16 patients.  Of the total PML cases, 36 were in the U.S., 44 were in the European Union and five in other areas.

Disclosure: The website for which I provide video and written blogs,, is sponsored by EMD Serono.


  1. This does give me, a bit of concern? if I were to try oral meds? Thanks for sharing this!

  2. Kim,

    It is always a concern when new drugs reach the market. There has been such build-up of excitement over the possibility of oral drugs for MS which 'might' replace injections. However, I know that my neuro is taking a conservative approach in waiting to see what the overall experience is for many more patients after the launch of Gilenya.

    Such a hard personal decision to make. These drugs are more effective but carry greater risks. Where does the balance tip one way or another? I don't know.

  3. I know my neuro told a friend that he was really upset with the side effect profile of the new oral med. At least they were in agreement on that!

    I have tried Ampyra, and it did NOTHING for me; my neuro said that while several other patients didn't show improvement on exam results, they "felt" better on the drug and wanted to keep taking it (at a cost of $1300/mo.).

    The PRICES of these drugs is just unreal. It makes me queasy just thinking about it. So I won't.

  4. Thanks for this excellent overview. I learned a great deal about the state of MS treatment from this post. I'll be sure to share this with my communities.

  5. Today my neurologist put me on a 3 day Solumedrol IV followed by an oral taper. I've had 39 Tysabri infusions so far with the most recent being 2 weeks ago.