Promising Multiple Sclerosis Drug Approved By F.D.A.

On Tuesday the Food and Drug Administration approved the first drug to treat a severe form of multiple sclerosis which offers hope to patients who previously had no other options to combat the disease that leads to paralysis and cognitive decline.

The drug has also been cleared by the federal agency to treat people with the more common, relapsing form of the disease.

Promising drug:

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, said “I think that this is a very big deal. The magnitude of the benefits that we’ve seen with ocrelizumab in all forms of M.S. are really quite stunning.”

The drug will be sold under the brand name Ocrevus by Genentech. It showed the most notable results in patients with relapsing multiple sclerosis, appearing to stop development of the disease with few serious side effects. Patients with the more severe form, primary progressive multiple sclerosis, the drug only modestly slowed patients’ decline, however, medical experts described it as an important first step.

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, said “This sort of opens the door for us. Once we open that door, then we do better and better and better. It’s a very encouraging result.”

More than a dozen treatment options are available for people with the relapsing form of the disease, however, the most effective drugs also come with significant side effects, meaning that doctors usually don’t prescribe them until a patient’s disease has advanced significantly.

Ocrevus side effects are considered mild: reactions at the injection site (the drug is infused every six months), and more upper respiratory infections and cold sores.

Which means it represents a new treatment option that has the potential to be used earlier.

In the United States, around 400,000 people have multiple sclerosis, and about 15% have the primary progressive form of the disease.

Trial results:

The trials that studied the relapsing form of the disease involved 1,656 patients, those taking Ocrevus had a 47% reduction in their relapses rate compared to patients who were taking the existing treatment, Rebif.

As for the clinical trial for people with primary progressive multiple sclerosis, it involved 732 patients. Those who were on the drug had 24% less risk of their disability progressing compared with patients who were taking a placebo.

Ocrevus depletes a specific type of a patient’s B cells. These cells are part of the immune system, which normally help the body fight off infections, however, they are believed to malfunction and contribute to central nervous system damage in people with multiple sclerosis.

Dr. Steven L. Galetta, the chairman of the department of neurology at NYU Langone Medical Center, who is an expert in multiple sclerosis but who wasn’t involved in the clinical trials said “I think if the safety holds up, it will become the leading M.S. therapy.”

One of the patients who received Ocrevus in the clinical trial, Jerrie Gullick, said that since she began taking the drug around three and a half years ago, the progression of her primary progressive multiple sclerosis has significantly slowed.