Novartis's Multiple Sclerosis Pill, a Likely Blockbuster, Gets FDA Approval

It took nine months since Novartis (NVS) published results from a late-stage study of its multiple sclerosis pill, but the Swiss pharmaceutical finally announced Wednesday that the U.S. Food and Drug Administration approved the oral treatment Gilenya (fingolimod). The FDA approved Gilenya as a first-line treatment for relapsing forms of multiple sclerosis -- the most common form (85% of patients) of the disease.

Multiple sclerosis is a chronic, irreversible illness in which the body's own immune system attacks the central nervous system. It's characterized by symptoms ranging from numbness to blindness and paralysis. In relapsing MS, patients experience recurrences or flare-ups of worsening neurological function, followed by periods in which the symptoms abate. MS affects over 400,000 Americans and more than 2.1 million people worldwide.

Although no cure has been found for MS, there are several strategies to treat symptoms or try slowing the disease's progression. But most of those involve cumbersome injections and infusions, so an oral therapy would have blockbuster potential for its manufacturer. Analysts estimate Gilenya would quickly reach sales of $1 billion a year.

Better Than Avonex, With Caveats

"A new treatment option that offers significant efficacy in the convenience of a capsule is a welcome alternative to frequent injections for individuals living with this chronic disease," says Nicholas LaRocca, vice president of Healthcare Delivery and Policy Research at the National Multiple Sclerosis Society.

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Novartis's Gilenya reduces the frequency of relapses and slows the disease's progression. In trials, Gilenya beat an established interferon therapy (Biogen Idec's (BIIB) Avonex) in 12 months of a nonoral treatment. However, since the pill works by suppressing the immune system (and thus preventing it from attacking a nerve covering called myelin that insulates and helps transmit impulses), patients who took the drugs in the study had higher rates of infections, including herpes and shingles, as well as other adverse events such as cancer.

And because Gilenya can cause a patient's heart rate to slow down, especially after the first dose, a doctor will have to watch patients for the first six hours after they take the first dose to see if they have any serious side effects. The FDA also requested that Novartis perform a five-year worldwide post-authorization study to monitor selected safety-related outcomes.

Direct Competitors?

Also, with other drugs on the market such as Teva's (TEVA) Copaxone, which also reduces relapse rate, and Elan's (ELN) and Biogen Idec's Tysabri, which has been linked to a deadly brain infection, more head-to-head trials may be needed to compare the efficacy of Gilenya to drugs other than Avonex.

Gilenya may also have direct competition soon if the FDA also approves a rival oral drug from Germany-based Merck KGaA. Another oral drug to improve walking in patients with MS from Acorda (ACOR) was approved in January.

NVS shares gained half a percent in premarket trading.