ZURICH Roche's multiple sclerosis (MS) drug Ocrevus won U.S. approval, putting the potential blockbuster back on track after a brief delay and giving a lift to patients with a form of the disease that until now had no approved treatment.
The drug, also known as ocrelizumab and administered via infusion, becomes the first U.S. Food and Drug Administration-approved medicine for the primary progressive form of the neurological disease (PPMS).
Ocrevus, to have a list price of $65,000 annually, undercutting rival Merck's Rebif by a quarter, is also approved to treat the most common form of MS, known as relapsing-remitting multiple sclerosis (RRMS).
Until now, patients suffering from PPMS were often relegated to managing symptoms and working with rehabilitation specialists as drugs including Teva's Copaxone, the most popular MS drug, failed to make a difference.
In trials with Ocrevus, however, doctors found no evidence of disease progression in 47 percent more PPMS patients compared with a placebo.
"The FDA’s approval of Ocrevus is the beginning of a new era for the MS community and represents a significant scientific advance," said Sandra Horning, Roche's chief medical officer.
About 400,000 people have MS in the United States, according the MS Foundation, with the 15 percent suffering from PPMS largely without effective treatment options. PPMS is characterized by worsening neurologic function, while the more-common relapsing form comes and goes with attacks of new and increasing symptoms.
Among RRMS patients, Roche has said 75 percent more patients reached "no evidence of disease activity" status when taking Ocrevus compared with those using Rebif.
Ocrevus works by reducing the immune system's assault on the body's own neurons. Unlike some other MS drugs that target T cells, Roche's new agent tackles B cells' role in driving inflammation that is central to the neurological disease.
"This is a real game changer," said Cyndi Zagieboylo, president of the National MS Society.
Shares in Roche -- which is counting on Ocrevus to offset falling revenue from top-selling cancer drugs Rituxan, Avastin and Herceptin that face impending competition from cheaper biosimilar versions -- rose 0.24 percent to 253.60 francs.
While the FDA approval was largely expected, analysts said the FDA's broad support, for both forms of the disease, was a plus.
"The label is close to the best case," wrote Emmanuel Papadakis, a Barclays analyst, adding Ocrevus does not include health warnings accompanying other MS drugs including Biogen's Tysabri and Sanofi's Lemtrada.
"Likewise, the U.S. list pricing point is clearly intended to position Ocrevus competitively against both orals and antibody options," Papadakis said.
Analysts forecast annual sales exceeding $3 billion by 2021, according to Reuters data.
U.S.-based Biogen will also benefit, with Roche to pay the part-owner of Ocrevus a royalty under a 2010 agreement.
The drug's U.S. entry had been delayed by three months when the FDA in December pushed back its approval deadline, citing manufacturing concerns.
Roche rival Novartis AG is also developing an MS treatment, BAF312, for secondary progressive multiple sclerosis. It could file for regulatory approval later this year, Novartis has said, although the official target remains 2019.
Other companies also seeking new MS drugs include GeNeuro SA.
(Additonal reporting by Akankshita Mukhopadhyay in Bengaluru and Bill Berkrot in New York; Editing by Bill Rigby/Ruth Pitchford)