FDA approves 1st drug for aggressive multiple sclerosis
- Author: Joanne Flowers Mar 29, 2017,
Mar 29, 2017, 20:18
The first treatment for the most severe form of multiple sclerosis-primary progressive MS-has been approved by the FDA.
Multiple sclerosis comes in different forms characterized by the severity and progression of the disease.
All forms of MS medication now on the market are meant to treat the relapsing/remitting form of the disease, making Ocrevus the first medication approved to treat the rarer, primary progressive form. Of the more than 2.3 million people affected by MS worldwide, approximately 15 percent are diagnosed with primary progressive form of the disease, according to the National Multiple Sclerosis Society.
Roche rival Novartis AG is also developing its own MS treatment, BAF312, for secondary progressive multiple sclerosis, and expects regulatory approval in the first half of 2017, depending on the outcome of talks with regulators. Ocrevus was also approved for relapsing forms of MS, which progress more slowly. Earlier MS drugs try to mitigate this attack by targeting the body's T cells.
The FDA's approval announcement noted that the drug "may increase the risk for malignancies, particularly breast cancer", but without special emphasis such as a boxed warning. In the two trials testing the drug in patients with relapsing-remitting MS, ocrelizumab led to lower relapse rates than treatment with standard of care interferon beta-1a. In both studies, the patients receiving Ocrevus had reduced relapse rates and reduced worsening of disability compared to Rebif.
The drug's USA entry had been delayed by three months when the FDA in December pushed back its approval deadline, citing manufacturing concerns. But those concerns weren't about the data. Patients with PPMS who received ocrelizumab achieved a 24% relative risk reduction in CDP sustained for at least 12 weeks compared with placebo; a 0.39 cm mean change in volume of brain hyperintense T2 lesions compared with a 0.70 cm mean change in volume of placebo-treated patients over 120 weeks; and a 25% relative risk reduction in the proportion of patients with 20% worsening of the timed 25-foot walk confirmed at 12 weeks. That made the trial "a landmark study in the field", the Journal wrote in an accompanying editorial. The patients with this severe type have watched for years as more than a dozen other treatments have emerged for other MS patients. The most common side effects in the study of PPMS were upper respiratory tract infection, skin infection, and lower respiratory tract infection. Patients were administered either 600 mg of ocrelizumab by intravenous infusion every 6 months or 44 mcg of interferon beta-1a administered by subcutaneous injection 3 times per week. But Porges cautioned that if a patient were to get a risky infection with ocrelizumab, it would be hard to reverse the drug's effect and fight it off.
Ocrevus will be available within the next two weeks and is supplied as a 300mg/10mL single-dose vial.