FDA approves Kesimpta® (ofatumumab) for patients with relapsing multiple sclerosis

Kesimpta was approved based on trial results showing the monthly injection significantly reduced the relapse rate and disability progression of MS patients compared to a currently approved treatment.

close up image of a man's arm pushing the wheel of his wheelchair

The US Food and Drug Administration (FDA) has approved Kesimpta® (ofatumumab) as a once-monthly subcutaneous injection for the treatment of relapsing forms of multiple sclerosis (RMS) in adults. The indication includes use in clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease.

Kesimpta is a targeted, precisely dosed and delivered B-cell therapy that can be self-administered once monthly at home via the Sensoready® autoinjector pen. The drug has shown superior efficacy with a similar safety profile compared with teriflunomide, a clinically available treatment for RMS.

“This approval is wonderful news for patients with relapsing multiple sclerosis. In the key clinical studies, this breakthrough treatment produced a profound reduction in new brain lesions, reducing relapses and slowing underlying disease progression,” said Professor Stephen Hauser, Director of the UCSF (University of California – San Francisco) Weill Institute for Neurosciences and co-chair of the steering committee for the ASCLEPIOS I and II studies which evaluated the safety and efficacy of Kesimpta 20 mg monthly subcutaneous injections versus teriflunomide 14 mg oral tablets taken once daily in adults with RMS. “Through its favourable safety profile and well-tolerated monthly injection regimen, patients can self-administer the treatment at home, avoiding visits to the infusion center.”

One of the goals when managing RMS is to preserve neurological function and slow the worsening of disability. Despite the availability of several disease-modifying therapies (DMTs) for the treatment of RMS, the majority of individuals with RMS continue to experience disease activity. However, evidence suggests early initiation of high-efficacy treatment could improve long-term outcomes for patients with RMS.

“Multiple sclerosis (MS) is a complex disease, and response to disease modifying treatment will vary among individuals,” said Dr Bruce Bebo, Executive Vice President of Research at the US National MS Society. “This makes it important to have a range of treatments available with different mechanisms of action and routes of administration. We are pleased to have an additional option approved for the treatment of relapsing forms of MS.”

Traditionally, B-cell treatments, which bind to and deplete B-cells associated with disease activity in MS, have predominantly been administered in hospitals or infusion treatment centres, adding cost to the healthcare system and a lifestyle burden for patients. Novartis stated, Kesimpta provides patients the flexibility of self-administering via once-monthly subcutaneous dosing requiring no premedication and eliminating the need to travel to an infusion centre. The positive results from the APLIOS study – an open-label Phase II study to determine the bioequivalence of subcutaneous delivery of Kesimpta via a prefilled syringe and a Sensoready pen in patients with RMS – and the ASCLEPIOS studies show Kesimpta to be a highly effective B-cell therapy that can be easily self-administered at home.

Marie-France Tschudin, President of Novartis Pharmaceuticals said: “When treating patients with RMS, Kesimpta is a meaningful treatment option that delivers both high efficacy and safety with the ability for patients to have more freedom in managing their disease. The development of Kesimpta is a great example of our commitment, knowledge and understanding of multiple sclerosis, which enabled us to identify a targeted treatment that can significantly improve patient outcomes and experience.”

Ofatumumab was first approved by the FDA in 2009 for the treatment of chronic lymphocytic leukemia (CLL) as an intravenous infusion with a high dose, administered by a healthcare provider. The drug was then investigated in an entirely new development program in RMS, due to the role of B-cells in the development of autoimmune diseases, such as MS. The clinical development program for ofatumumab in RMS took 10 years and has involved studies in more than 2,300 patients around the world. Kesimpta was found to work through a distinct mode of action, and the treatment regimen – specifically designed for RMS – plays a critical role in the outcome. This is a different dosing regimen and route of administration than was previously approved for the CLL indication.

The approval of Kesimpta is based on results from the Phase III ASCLEPIOS I and II studies, in which Kesimpta demonstrated superiority versus teriflunomide in significantly reducing the annualised relapse rate (ARR, primary endpoint), 3-month confirmed disability progression (CDP) and the number of gadolinium-enhancing (Gd+) T1 and new or enlarging T2 lesions. Results from these two studies were recently published in The New England Journal of Medicine.

Kesimpta is expected to be available in the US in early September. Additional regulatory filings are currently underway across the world and regulatory approval for the drug in Europe is expected by Quarter two of 2021.