Novartis AG (NVS) announced Wednesday that a two-year Phase III study involving more than 4,000 patients worldwide shows that a new drug significantly reduces relapses and disability progression of multiple sclerosis. The chronic, irreversible disease attacks the central nervous system and is characterized by symptoms ranging from numbness to blindness and paralysis. Eighty-five percent of MS patients have "relapsing-remitting MS," characterized by attacks -- relapses -- of worsening neurologic function.
Although MS has no cure, several strategies can modify the disease course, treat relapses, manage symptoms and improve function and safety. The Swiss drugmaker's FTY720 joins existing MS drugs including Biogen Idec (BIIB)'s Avonex, which reduces the risk of disability progression, exacerbations, and the number of lesions in the brain; Teva (TEVA)'s Copaxone, which also reduces relapse rate; and Elan (ELN)'s and Biogen Idec's Tysabri, which also reduces the risk of disability progression. The FDA recently amended the labeling of Tysabri to add information about incidences of a rare but sometimes fatal brain infection; on Tuesday, Elan received an SEC subpoena regarding its disclosure of two such cases.
Unlike most MS drugs, which are taken by injection or IV infusion, FTY720 is an oral drug. It reduced relapse rates in 54 percent to 60 percent of subjects, compared to a placebo, and disability progression in 30 percent to 32 percent. Detailed results are slated for presentation at a leading scientific congress next year, Novartis said. Merck KGaA and Biogen are also racing for first approval of an oral MS therapy. Merck already beat Novartis when it filed Wednesday for approval of its MS pill cladribine in the U.S.
Novartis's result builds on previous data showing superior efficacy to Avonex (interferon beta-1a), the current treatment standard, the company announced, and there was no significant difference in efficacy between high and low doses (1.25 mg and 0.5 mg doses), which could help with safety concerns. Novartis says it plans to file for approval of the lower dose.
FTY720 has been associated with such safety concerns as potentially fatal infections and skin cancer, which Novartis did not address. But Trevor Mundel, Novartis Pharma's global head of development, said in the release, "FTY720 0.5 mg therapy offers compelling efficacy on all relevant endpoints compared to both placebo and a standard of care, complemented by extensive safety data."
Novartis says it's on track to submit the drug for approval in the U.S. and Europe in December. The FDA may be willing to review the drug within six months, Mundel says, meaning an approval could come as early as next year. Novartis's drug is expected to reach blockbuster status -- over $1 billion in annual sales -- within a few years of its launch.
NVS shares closed up nearly 2 percent at $50.38.
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