The Food and Drug Administration approved on Tuesday the first drug to treat a severe form of multiple sclerosis, offering hope to patients who previously had no other options to combat a relentless disease that leads to paralysis and cognitive decline.
The federal agency also cleared the drug to treat people with the more common, relapsing form of the disease.
“I think that this is a very big deal,” said Dr. Stephen Hauser, the chairman of the neurology department at the University of California, San Francisco, and leader of the steering committee that oversaw the late-stage clinical trials of the drug, ocrelizumab. “The magnitude of the benefits that we’ve seen with ocrelizumab in all forms of M.S. are really quite stunning.”
The drug, which will be sold under the brand name Ocrevus by Genentech, showed the most notable results in patients with relapsing multiple sclerosis, appearing to halt progression of the disease with few serious side effects. In patients with the more severe form, primary progressive multiple sclerosis, the drug only modestly slowed patients’ decline, but medical experts described it as an important first step.
“This sort of opens the door for us,” said Dr. Fred D. Lublin, who was a crucial investigator for the clinical trial and is director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis at Mount Sinai Hospital in New York. “Once we open that door, then we do better and better and better. It’s a very encouraging result.”
Genentech, which is owned by the Swiss pharmaceutical giant Roche, said Tuesday that it would charge a list price of $65,000 a year, which — though expensive — is 25 percent less than an existing drug, Rebif, that was shown to be clinically inferior to Ocrevus in the two clinical trials that led to Ocrevus’s approval.
Rebif, sold in the United States by the German company EMD Serono, carries a list price of about $86,000. In a statement, Genentech noted that the price of drugs to treat multiple sclerosis had risen sharply in recent years.
“We feel that the industry needs to start to reverse this trend, and believe that pricing Ocrevus 25 percent less than the comparator in our trials is an important first step,” the company said.
Although people with the relapsing form of the disease have more than a dozen treatment options, many of the most effective drugs also come with significant side effects, which means that doctors often wait to prescribe them until a patient’s disease has advanced significantly. Ocrevus is viewed as relatively safe: Side effects included reactions at the injection site (the drug is infused every six months), and more upper respiratory infections and cold sores.
As a result, “it represents a new treatment option that has the potential to be used earlier,” said Dr. Peter Chin, the group medical director of neuroscience at Genentech, who was closely involved in developing the drug.