Novartis AG won FDA clearance for the anti-CD20 monoclonal antibody Kesimpta (ofatumumab) in relapsing forms of multiple sclerosis (MS) in adults, with a label that includes clinically isolated syndrome and relapsing-remitting as well as active secondary progressive disease.

A targeted B-cell therapy that has shown superior efficacy with a similar safety profile compared with oral Aubagio (teriflunomide), the dihydroorotate dehydrogenase inhibitor from Sanofi SA, Kesimpta is a first-choice treatment option for relapsing MS patients, Basel, Switzerland-based Novartis said. It’s also the first B-cell therapy that can be self-administered once monthly at home using an autoinjector pen.

The approval came earlier than expected, after what regulators signaled would be a three-month delay of the PDUFA date, pushing the sBLA decision to September. Ofatumumab was first given the go-ahead in October 2009 as Arzerra for chronic lymphocytic leukemia (CLL). The clinical development program for Kesimpta took 10 years and has involved more than 2,300 patients around the world. Partnered with Genmab A/S of Copenhagen, Denmark, Kesimpta involves a different dosing regimen and route of administration than for CLL with Arzerra.

Bolstering the case for relapsing MS approval were the phase III Asclepios I and II studies. They were twin, identical-design, flexible-duration (up to 30 months), double-blind, randomized, multicenter experiments to measure the safety and efficacy of 20-mg monthly subcutaneous injections vs. Aubagio 14-mg tablets taken once daily. Enrolled were 1,882 patients between the ages of 18 and 55 years, with an Expanded Disability Status Scale score between 0 and 5.51,2. The studies were conducted at more than 350 sites in 37 countries. The primary endpoint was to prove Kesimpta superior to Aubagio in reducing the frequency of confirmed relapses (as evaluated by the annualized rates) in patients treated up to 30 months. Secondary endpoints included time to disability progression confirmed at three and six months, confirmed disability improvement at six months, gadolinium enhancing T1 lesions, number of new or enlarging T2 lesions, serum levels of neurofilament light chain, and rate of brain volume loss.

In multiple sclerosis, the protective myelin sheath becomes damaged and myelin is lost, a process known as demyelination, leaving the nerve fiber exposed. This causes the axon to malfunction as electrical impulses between nerve fibers are interrupted. © 2017 Clarivate Analytics. All rights reserved.

The go-ahead for Kesimpta holds meaning not only for Paris-based Sanofi’s Aubagio, approved in September 2012, but also for Ocrevus (ocrelizumab), the anti-CD20 B cell therapy from Roche Holding AG, of Basel, Switzerland, and Cambridge, Mass.-based Biogen Inc., OK’d for marketing in the U.S. in March 2017.

Piper Sandler analyst Christopher Raymond said in a June report on his firm’s survey of neurologists that Kesimpta was “gearing up as a major challenger” to Ocrevus. The latest good news for Novartis follows word this month that the U.S. District Court of Delaware upheld the validity of the Gilenya (fingolimod) dosage-regimen patent. Gilenya, a sphingosine 1-phosphate receptor modulator, was cleared in September 2010 for relapsing MS. The patent battled pitted Novartis against Hec Pharm Co. Ltd., of Guangdong, China.

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