National organizations that represent patients with multiple sclerosis (MS) welcome the U.S. Food and Drug Administration’s March 26 approval of Novartis’ oral therapy Mayzent (siponimod) — but they complain that, at $88,500 per year, the treatment is overpriced.
The Multiple Sclerosis Society of America (MSSA) is clearly upbeat about the new medication. The organization’s chief medical consultant, Jack Burks, MD, noted that Mayzent is the only disease-modifying therapy to be approved in recent years for active secondary progressive MS, or SPMS. It also treats clinically isolated syndrome (CIS) and relapsing-remitting MS (RRMS).
According to Novartis, patients taking Mayzent do not require a first-dose observation to monitor for cardiac events, except in patients with certain pre-existing heart conditions. The company also expects to have the therapy available in the U.S. in early April.
“The FDA approval of Mayzent for wide-ranging forms of MS — including active SPMS, RRMS, and CIS — is very good news for members of the MS community,” Burks said in a press release. “The lack of first-dose cardiac events and the fact that it will soon be available both add to the good news. MSAA, the medical community and those affected by MS are greatly pleased to hear of this exciting new approval.”
Kathy Costello, associate vice president of healthcare access at the National Multiple Sclerosis Society (NMSS), said Mayzent is significant for several reasons.
“It crosses a number of disease phenotypes of MS, including clinically isolated syndrome, RRMS, and active SPMS,” she said in a phone interview with Multiple Sclerosis News Today. “While this does not get at the type of SPMS that’s not active, it’s certainly a move in the right direction.”
She added: “Most people would prefer an oral medication over an injectable, although adherence to medication can be difficult no matter what the route of administration is. It’s a challenge for anybody over a long period of time.”
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Costello noted that the last medication the FDA approved for such conditions was Novantrone (mitoxantrone) back in 2000. “However, it’s rarely used — if at all — because it has significant long-term risks including heart issues and secondary leukemia. Therefore, in terms of SPMS, this becomes an important approval and will hopefully stimulate important research so that additional therapies come to market.”
Novartis therapy ‘long overdue’
Asked what percentage of the nearly 1 million Americans now believed to have MS could benefit from Mayzent’s approval, Costello said “it’s difficult to pin down the exact time someone with SPMS is no longer exhibiting activity.”
“Time has to elapse to understand if they’re getting worse and having relapses, or just progressing but not having new activity,” she explained. “Probably, after 20 years with MS, the majority of people will transition from RRMS to SPMS. But much of that number is based on data prior to having disease-modifying therapies.”
Ed Tobias, a senior patient columnist for Multiple Sclerosis News Today, said Mayzent is long overdue.
“From my point of view, this is wonderful,” said the 70-year-old, who has lived with MS since 1980. “There are a significant number of people with MS in the secondary progressive stages. That’s where you wind up sooner or later with this disease.”
Tobias, who needs two canes to walk even short distances — and uses an electric scooter for longer trips — said he constantly surfs MS-related social media websites.
“One of the things I see regularly is patients complaining about not having a drug available to them for SPMS, at least in the United States,” said Tobias, a resident of Ocean City, Maryland. “The focus of the pharmaceutical companies was, and seems to still be, the RRMS form of the disease.”
Tobias now takes Lemtrada (alemtuzumab), developed by Sanofi Genzyme, after years on various other medications. He explained that very few therapies have been available for either form of progressive MS. The only therapy on the market for primary progressive MS (PPMS) is Genentech’s Ocrevus (ocrelizumab), which is also approved for relapsing MS forms.
Patient sees ‘untapped market’ for Mayzent
Following the FDA’s approval of Novantrone, the rights for that medication were sold to another company, Tobias said, “and that company, for whatever reason, decided not to distribute it in the U.S.,” though it’s still available overseas under its generic name, mitoxantrone.
“Any medication that’s in pill form taken once a day that has any kind of decent efficacy is something patients will be interested in speaking with their neurologists about,” he said.
One MS patient eager to try Mayzent is Jenn Powell, a patient columnist for Multiple Sclerosis News Today who lives in Orange County, California.
“What’s exciting is that this helps patients with secondary progressive MS, which has really been an untapped market,” she said.
Powell, 50, was diagnosed with MS just over 10 years ago. She’s already written to her doctor asking to switch to the new therapy.
“Ever since I have progressed, there’s never been an oral drug focused on active disease modification for SPMS. Right now I’m on chemo, and people who have been on immunosuppressant therapies have a greater risk of developing some other side effects such as PML [progressive multifocal leukoencephalopathy].”
Powell said Mayzent carries “a very high price tag,” but that her managed healthcare plan pays for therapies that other plans often don’t cover.
ICER says therapy way overpriced
Indeed, Costello said the NMSS has “long called for a reversal in the upward trajectory” of expensive MS medications, and criticized Novartis for pricing Mayzent at $88,500 a year — especially after the Boston-based Institute for Clinical and Economic Review (ICER) determined that to hit a cost-effectiveness threshold of $150,000 per added year of health, the therapy should cost no more than $12,000 a year, according to a March 27 report by Bloomberg.
That’s almost 87 percent cheaper than the price Novartis has established.
“We believe the company missed an opportunity to continue the downward trend started by the last new entry to the market,” Costello said. “Price is an important factor in determining access to a medication. So while Mayzent is not priced at the top of the MS drug list, we believe the price is still too high.”
Yet Novartis itself insists its new therapy is priced in line with other MS treatments, and that it’s confident that Mayzent’s $88,500 annual price tag “enables affordable access for people” with the disease.
“Novartis considered many factors in determining the price of Mayzent,” the company said in a statement sent to NMSS. “As the first oral drug to delay disability progression in RRMS, CIS, and active SPMS, Mayzent is poised to substantially reduce the burden of the condition on patients, caregivers, employers, insurers, and the healthcare system and society as a whole.”
Costello said the NMSS, along with other members of the MS Coalition, recently published guidelines on the use of disease-modifying therapies. Among other things, the coalition recommends that patients continue with the therapies they’re already taking, unless they have side effects or a better therapy comes along.
“We hope the reasons for making a change in therapy would be medically based,” she said, adding that insurance companies should also be educated about Mayzent.
“They need to know about the drug,” Costello said. “We hope patients will have access to the full range of therapies, but those decisions are made by each individual insurer.”