Pharma

The Peripheral and Central Nervous System Drugs Advisory Committee, appointed by the FDA to conduct an expert review of Novartis' new oral MS drug Gilenia (aka fingolimod or FTY720), met today and voted to recommend approval for this therapy. The panel discussed the side effects that have been observed in clinical studies (such as cardiac problems, an eye disorder called macular edema, and impaired lung function), but ultimately decided that the benefits of Gilenia outweigh these risks. The panel did recommend that a lower dosage of the drug be evaluated -- but agreed that this additional study could take place after approval of the current proposed dose (0.5 mg). The committee also voted in favor of making Gilenia available as a first-line treatment rather than only as a back-up if other treatments don't work.

The FDA is expected to decide whether to approve Gilenia in September. It usually follows the advice of its advisory committees, although it's not required to. Assuming Gilenia is approved, it will most likely be prescribed in conjunction with a safety monitoring plan to identify any possible new issues with the drug.

All of the background materials that the committee considered for today's meeting can be downloaded openly from the FDA website. These include information submitted by Novartis and reports prepared by FDA's scientific and medical experts. If you're considering trying Gilenia if and when it becomes available, you may want to read through this information, especially if you are concerned about particular side effects.

Here's some unhappy news for people with MS -- one of the few drugs being evaluated for helping with secondary progressive MS, dirucotide, has been pulled from further study following disappointing results from the phase III study MAESTRO-01. In this study, partners BioMS and Eli Lilly had hoped to show that the drug delayed how long it took subjects with MS to worsen. Unfortunately, this didn't happen, nor were the secondary goals of the study achieved. The companies also announced that the follow-on studies MAESTRO-02 and -03 will be discontinued. Participants in these trials are advised to contact their study investigators for more information on the discontinuation process.

In a small note of possible consolation, I have heard recently from different drug companies that they are interested in developing drugs for progressive MS. It will take time for these drugs to come to market, but the success of the first-line RRMS drugs has probably demonstrated that drugs for progressive stages of the disease would also find a receptive market. Hopefully some of these will meet with better luck than dirucotide.

The pharmaceutical company Merck KGaA has submitted an application to the European Medicines Agency (EMEA) (the European Union's FDA) asking them to approve oral cladribine as a therapy for patients with relapsing-remitting multiple sclerosis. Similar applications to the FDA and other countries' agencies will be following soon. The company hopes to be able to market this drug in 2010, which might make it the first oral drug officially authorized as a disease-modifying therapy in MS.

Cladribine is a chemotherapy drug that depletes white blood cells and is used to treat certain forms of leukemia. Recent results from the Phase III CLARITY trial showed that it reduced relapse rate and MRI disease activity in people with RRMS. The treatment schedule used in the trial involved taking the drug only 2 to 4 weeks per year. This drug would be easier to take than the current first-line therapies for MS, but would perhaps come with a greater risk of complications due to its strong effects on the immune system.

[Thanks for Stephanie S. for the pointer to this story!]

These market analysts are reporting that: The Multiple Sclerosis (MS) Market Has Total Global Revenues of Over $6bn in 2008. It Will Exceed $9bn During the Second Half of Next Decade.

I report this because the "good" side of this is that it means MS drugs can be very lucrative. Which means that pharma and biotech companies will consider MS as a target disease for their products. For the most part, more attention is better.

A cannabis-based medicine for MS from GW Pharmaceuticals has been filed for approval in Europe, paving the way for its potential approval at the end of 2009 or early in 2010.

Clinical trials have shown GW's drug Sativex, which is sprayed under the tongue, reduces spasticity in multiple sclerosis patients who do not respond adequately to existing therapies.

The subject basically says it all.

The FDA has granted priority review status to the experimental MS drug called Fampridine-SR.

Just received from Biogen Idec:

Please find below links to three Biogen Idec press releases that were distributed during AAN. The releases included data showing TYSABRI may have the potential to remyelinate brain lesions, patients reported a better quality of life after only three months of TYSABRI treatment, and AVONEX patients who started treatment shortly after their first attack fared better than those who delayed treatment.

• MRI DATA SHOWING TYSABRI® PROMOTED REMYELINATION PRESENTED AT THE 61ST ANNUAL MEETING OF THE AMERICAN ACADEMY OF NEUROLOGY
• New Tysabri(R) Data Demonstrate Significant Effects On Health-Related Quality Of Life Measures In Multiple Sclerosis Patients
  
• First Ten-Year Follow-Up Shows That Treatment with AVONEX® Leads to Long-Term Benefits in Early Multiple Sclerosis Patients

Check out this article talking about two new oral drugs being tested for MS.

In the Phase III study of 1,200 patients with the RRMS, approximately 80 percent of MS patients who took the drug cladribine remained relapse-free for two years compared to only 61 percent of those participants given a placebo.

In another successful phase III study of over 1,200 patients with RRMS, 80 to 84 percent of MS patients who were given daily dosages of the drug fingolimod remained relapse-free after one year in comparison to only 67 percent of study participants who received Avonex, a typical injectable drug used to treat MS.

An independent safety drug board recommended BioMS continue trials of its multiple sclerosis treatment.

The U.S. Data Safety Monitoring Board completed a safety analysis of its phase 3 Maestro-03 trial of dirucotide, used for the treatment of secondary progressive MS, (SPMS) and ruled that the company should continue.

This is just a review done to make sure that any adverse events from the trial are warranted given the results - it is not saying anything about the effectiveness (at this point), just the safety. Good news for those with SPMS, though.

A sixth patient taking Biogen Idec Inc's multiple sclerosis drug Tysabri has developed a potentially deadly brain infection since the drug was reintroduced onto the market in July 2006.

The article, per usual current-day journalism standards, gives no details on the person, how they are doing, what will be done for them, etc. It also indicates that Biogen will only announce future cases by posting to their web site, but gives no indication of *where* on their web site.

I contacted Biogen Idec and got a prompt reply - the updates will be posted weekly at this location. I made a stink about this appearing in their "Investor Relations" section of their web site and not in the patient information section. It is my opinion that this sends a clear signal that Biogen Idec sees PML as a financial issue, and not as a patient safety issue.

Biogen - if you disagree, then put an easily findable link on tysabri.com so people who are putting this stuff in their veins can be as fully informed as your investors.