On March 1, 1999, the U.S. Food and Drug Administration (FDA) ruled that it would uphold the Orphan Drug Act (ODA), an important federal law which provides incentives to companies which develop novel therapies to treat diseases or conditions that affect relatively small numbers of people. This is good news for people with multiple sclerosis (MS), as this disease is one of the conditions covered by the ODA. By upholding the ODA, the FDA has solidified incentives that will hopefully generate truly new therapies for MS in the coming years.

The ODA provides seven years of marketing exclusivity for a given product, when approved to treat a rare disease or condition. Both AVONEXÒ (Interferon beta-1a) and BetaseronÒ (Interferon beta-1b) both have orphan drug protection for relapsing-remitting MS. As a result, the FDA ruled that it would not permit a third beta interferon product to enter the US marketplace for the treatment of Multiple Sclerosis (MS). In addition to upholding the ODA, the FDA also had questions about the data filed in the marketing application for this product, called Rebif^® (Interferon beta-1a), that prevented granting even tentative approval (which would have enabled the drug to enter the U.S. market after the exclusivity period ends in 2003).

While it is possible for the FDA to overturn an orphan drug’s market exclusivity, it may do so only when it deems that a product offers "a significant therapeutic advantage over and above that provided by an approved orphan drug." In this case, the FDA found that Rebif did not provide a significant therapeutic advantage over existing products. People with MS should feel confident that effective therapies are already available on the market and that they are not missing out on a product with any proven advantages over those existing therapies. Rebif has not been proven any more effective than existing therapies, and, in fact, unlike AVONEXÒ, is associated with skin necrosis (tissue damage) and frequent injection site reactions. AVONEXÒ remains the standard of care; it offers unsurpassed safety and effectiveness, and its once-weekly dose makes therapy convenient.

Jim Vincent, Biogen’s Chairman and Chief Executive Officer, said, "The Agency’s decision reinforces our confidence that the FDA makes its decisions on the merits of good science and clinical results. People with MS and their physicians in the U.S. currently have three safe and effective therapies available to treat multiple sclerosis. These include AVONEXÒ, which is approved for all relapsing forms of MS, and is the drug of choice among people with MS and their physicians. As the FDA ruling makes clear, Rebif does not offer greater efficacy or safety and does not provide a significant addition to patient care."

It’s important to understand that the FDA’s decision to uphold the ODA in this case is in the best interest of people with MS. The ODA has provided incentives which have led to many of the recent breakthroughs in MS therapy. A strong ODA will continue to encourage new research into developing new treatments for MS. Ultimately, it is more important for people with MS to have entirely new therapeutics available in the upcoming years, than to have another beta interferon product which offers no proven advantages, available now.

The incentive of Orphan Drug exclusivity has enabled Biogen to fund important new research on AVONEXÒ and other products. Biogen reinvests about 30% of revenues into research for new compounds, many of which have potential to provide new treatment options for people with MS. This level of investment is substantially greater than the average for the pharmaceutical industry. Hopefully, Biogen’s research will generate new products that provide significant advances for people with MS and other diseases.

AVONEX website

This material is provided as general medical information and is not intended as advice for individual patients; please contact your physician for specific recommendations.

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Email Jean ©1996-2002 International MS Support Foundation.  All rights reserved. Disclaimer: This material is provided as general medical information only and may not include all side effects or details relevant to a particular individual's treatment. Answers are not intended as advice for individual patients; please contact your own physician/neurologist  for specific recommendations.