The US Food and Drug Administration (FDA) has approved ofatumumab injection (Kesimpta, Novartis) for the treatment of adults with relapsing forms of multiple sclerosis, including relapsing-remitting MS, active secondary progressive MS, and clinically isolated syndrome, the manufacturer announced in a press release.
This is the first FDA approval of a self-administered, targeted B-cell therapy for these conditions, and is delivered via an autoinjector pen.
“This approval is wonderful news for patients with relapsing multiple sclerosis,” Stephen Hauser, MD, director of the Weill Institute for Neurosciences at the University of California San Francisco, said in the same release.
“Through its favorable safety profile and well-tolerated monthly injection regimen, patients can self-administer the treatment at home, avoiding visits to the infusion center,” he noted.
Hauser is also co-chair of the steering committee for the phase 3 ASCLEPIOS I and II studies that were part of the basis for the FDA’s approval.
Bruce Bebo, PhD, executive vice president of research at the National MS Society, said because response to disease-modifying treatments varies among individuals with MS, it’s important to have a range of treatment options available with differing mechanisms of action.
“We are pleased to have an additional option approved for the treatment of relapsing forms of MS,” he said.
Formerly known as OMB157, ofatumumab is a precisely-dosed anti-CD20 monoclonal antibody administered subcutaneously via once-monthly injection.
However, Novartis noted that initial doses are given at weeks 0, 1, and 2 — with the first injection occurring with a healthcare professional present.
The drug “is thought to work by binding to a distinct epitope on the CD20 molecule inducing potent B-cell lysis and depletion,” the manufacturer noted.
As reported at the time by Medscape Medical News, results for the ACLEPIOS I and II studies were presented at the 2019 Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), with additional results presented at the 2020 Virtual Annual Meeting of the Consortium of Multiple Sclerosis Centers.
In addition, the findings were published earlier this month in the New England Journal of Medicine.
The twin, identically designed phase 3 studies assessed the safety and efficacy of the drug at a monthly subcutaneous dose of 20 mg vs once daily teriflunomide 14 mg oral tablets. Together, the studies included 1882 adult patients at more than 350 sites in 37 countries.
Results showed that the study drug reduced the annualized relapse rate (ARR) by 51% in the first study and by 59% in the second vs teriflunomide (P < .001 in both studies), meeting the primary endpoint.
Both studies also showed significant reductions of gadolinium-enhancing (Gd+) T1 lesions (by 98% and 94%, respectively) and new or enlarging T2 lesions (by 82% and 85%).
Although the FDA first approved ofatumumab in 2009 for treating chronic lymphocytic leukemia (CLL), it was administered as a high-dose intravenous infusion by a healthcare provider.
“This is a different dosing regimen and route of administration than was previously approved for the CLL indication,” the company noted.
The drug is expected to be available in the United States early next month.