- The Food and Drug Administration has approved the first new drug in more than 15 years for a later-stage form of multiple sclerosis, clearing on Tuesday a medicine from Swiss pharma Novartis for use in a patient population that's estimated to number roughly 250,000 in the U.S.
- Mayzent, as the drug will be sold, is OK'd for secondary progressive MS, which is characterized by steady advances in neurological and physical disability. As many as eight in 10 patients with relapsing/remitting MS — the most commonly diagnosed type of the chronic neurological disorder — eventually progress to develop secondary progressive MS.
- Mayzent will enter a therapeutic market that's become one of the most competitive in the pharmaceutical industry. As one of the few options for secondary progressive MS, Novartis' drug could still play an important option in treatment of the disease — something the drugmaker is counting on in predicting future blockbuster sales. The drug's label also covers relapsing/remitting MS.
For many MS patients, the disease is defined by acute relapses or attacks, between which their symptoms lessen or remain stable. Known as relapsing/remitting MS, this form accounts for roughly 85% of all MS diagnoses.
Over time, however, the disease can evolve into secondary progressive MS, resulting in steady disease progression without intervening moderation between relapses.
MS, which across all forms affects more than 2 million people globally, interferes with normal brain and nerve function. Individuals affected by the disease experience deteriorating physical abilities, as well as cognitive decline.
Many drugs, including top-sellers like Biogen's Tecfidera (dimethyl fumarate) and Novartis' own Gilenya (fingolimod), are approved to treat relapsing/remitting MS, but not secondary progressive disease.
In the Phase 3 study supporting the approval of Mayzent (siponimod), secondary progressive MS patients treated with Novartis' drug had a 21% lower risk of confirmed disease progression at three months than those given placebo. Mayzent also showed a significant reduction in the risk of progression at six months, and reduced annualized relapse rates by 55%, according to results from the trial.
Novartis claims Mayzent is the only disease-modifying therapy that's demonstrated efficacy in a typical secondary progressive MS patient population. Yet the drug isn't a panacea, and data showed no difference between patients on treatment and those taking placebo on a MS walking scale and on a timed 25-foot walk test.
In granting its OK, the FDA cleared Mayzent with a broader label than expected, approving the drug for relapsing forms of MS broadly, including relapsing/remitting MS as well as active secondary progressive disease.
Novartis indicated it would focus primarily on those relapsing/remitting patients in transition, as well as the smaller group of individuals with secondary progressive disease.
"This is a highly competitive area, increasingly dominated by Ocrevus, and where Mayzent has limited differentiation and only modest clinical data," wrote Credit Suisse's team of European pharmaceutical analysts in client note, referring to a competing medicine from Roche.
Novartis priced Mayzent at a list price of $88,500 per year, a cost the company said was "in-line" with other MS medications. Gilenya, which last year earned Novartis $3.3 billion, costs $98,500 per year while Biogen's Tecfidera is priced at $93,700.
"We are confident we have appropriately priced Mayzent to enable affordable access for people with MS," Novartis said in an emailed statement.
Mayzent is one of a slate of 10 medicines Novartis has pegged for future sales above $1 billion a year. Commercial success with Mayzent could help cushion a hit to revenues from Gilenya, which will lose protection from a key patent in August.
Novartis has a patent on the dosage regimen for the drug that runs through 2027, however — potentially enough to keep generic rivals at bay. The big pharma has filed a lawsuit against 23 generic companies seeking to enforce that patent, and hopes to secure a preliminary injunction on any at-risk launches of copycats to Gilenya.
Mayzent will be available in the U.S. in approximately one week, Novartis said.