New MS Drug Approved by FDA
March 29, 2017 — The FDA has approved a new medication that is the first to treat a rare form of multiple sclerosis.
The FDA approved Ocrevus (ocrelizumab) Tuesday for primary progressive MS (PPMS), as well as the most common form of MS.
There are already several treatments for the common form, relapsing-remitting MS (RRMS), which affects about 85% of MS patients. But the FDA gave Ocrevus a “breakthrough therapy designation” last year because it also treats PPMS, an especially debilitating form of MS where the disease steadily worsens rather than having periods of relapse or remission. The CDC estimates about 15% of patients with MS have PPMS.
Clyde E. Markowitz, MD, director of the Multiple Sclerosis Center at the University of Pennsylvania, says this fills a large unmet need for patients with progressive disease.
“This is a very exciting time for the treatment of MS,” he said. “We are thrilled to have another tool.”
Like other biologic drugs, however, Ocrevus’ price tag probably will be hefty.
It’s estimated that more than 2 million people worldwide have MS, a chronic condition where the immune system attacks the myelin sheath that surrounds and protects nerve cells in the brain, spinal cord, and optic nerves. Common symptoms include numbness, vision problems, weakness, pain, muscle cramping, fatigue, and impaired movement.
Here’s what you need to know about this new medication:
How does it work?
Ocrevus is a type of drug known as a monoclonal antibody, which targets a specific type of immune cell thought to be involved with nerve and myelin cell damage.
How well does it work?
In clinical trials, patients received 600 mg intravenously every 6 months.
“An infusion every 6 months makes it very convenient, so people don’t have to take it on a more regular basis like injectables or pills,” Markowitz says.
- Ocrevus helped slow the progression of PPMS by 24%, compared with a placebo. Progression is defined as difficulty walking and worsening problems with motor, sensory, and vision coordination. Patients on the drug were more stable.
- The relapsing-remitting MS trial showed a 46% drop in annual relapse rates, compared with an existing and commonly used MS drug, interferon beta-1a (Rebif). Doctors say patients on average relapse once a year without treatment.
- 48% of patients in the relapsing-remitting MS trial had no relapses, no worsening of neurological symptoms, and no new brain lesions seen on MRI scans.
Markowitz was the principal investigator on the primary progressive trial for Ocrelizumab.
“The results for the relapsing trial were more impressive, given that they were tested against an already approved therapy, but progressive disease is harder to study because it occurs so slowly,” he says. “To see a significant benefit would take years, and this was only approximately a 2-year trial.”
Who should and shouldn’t take it?
Christopher Lock, MD, an MS specialist at Stanford Health Care in Stanford, CA, says he expects to offer Ocrevus to all of his patients with the primary progressive form of the disease who are interested.
“There is a lot of anticipation in the MS community,” he says.
The FDA says Ocrevus should not be used by patients with hepatitis B or other active infections, and the agency warned it may increase the chance of malignancies, particularly breast cancer.
Lock says that as with other MS medications, doctors will screen patients to ensure they are good candidates before offering Ocrevus. He points out that in the clinical trials of ocrelizumab, subjects were screened for infections such as hepatitis B and C, HIV, syphilis, and tuberculosis.
Because the drug suppresses the immune system, it could reactivate those infections, Lock says. “A history of cancer will need to be taken into consideration on an individual basis. Subjects with a history of cancer are generally excluded from clinical trials, so we do not have information to guide us on this question,” he explains.
What are the side effects?
In clinical trials, side effects included infusion-related reactions like rash, throat irritation, and flushing that were more often seen in the first infusion. Other side effects included upper respiratory tract infections and oral herpes infections.
What is the price tag? Will insurance cover it?
The drug will cost about $65,000 a year, The New York Times reported Wednesday. Drug maker Genentech did not immediately respond to requests on price.
The cost would be in line with current MS drugs, which Markowitz says range from $60,000 to $70,000 a year before insurance. Since insurance companies often have a say in what medications you can try first, PPMS patients may have an easier time getting approval to take Ocrevus, since it is the only medication available for that form of the disease and there are many treatment options for relapsing-remitting MS.
When will it be available?
Genentech says Ocrevus will be available to people in the U.S. within 2 weeks.
Christopher Lock, MD, Stanford Health Care, Stanford, CA.
Clyde E. Markowitz, MD, University of Pennsylvania, Philadelphia.
G. Giovannoni, ECTRIMS Online Library, Sept. 15, 2016.
S.L. Hauser, The New England Journal of Medicine, Dec. 21, 2016.
X Montalban, The New England Journal of Medicine, Dec. 21, 2016.
Anthony Traboulsee, Neurology, April 5, 2016.
FDA: “FDA approves new drug to treat multiple sclerosis.”
Genentech: “FDA approves new medicine for relapsing and primary progressive forms of multiple sclerosis.”
National Multiple Sclerosis Society: “Relapsing-remitting MS (RRMS),” “Primary progressive MS (PPMS).”
Roche, “Investor Update,” Sept. 14, 2016; “Media Release,” Dec. 20, 2016.