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earlier speculation from Leerink Partners analyst Geoffrey Porges and others polled by Xconomy that insurers might push back on nusinersen because of its high price tag. The drug, a chronic treatment, costs $750,000 for the first year and $375,000 each year thereafter. Those numbers continued to push the envelope higher for the price of drugs for rare or “orphan” diseases, which affect 200,000 people or less. Historically, insurers haven’t resisted coverage of orphan drugs because of the small total patient numbers and lack of other options. But they began pushing back last year upon the controversial approval of the Duchenne muscular dystrophy drug eteplirsen (Exondys 51) from Sarepta Therapeutics (NASDAQ: SRPT). The drug was priced at $300,000 per patient per year but approved without evidence of a clinical benefit.
Anthem was one of the early insurers to draw a hard line on eteplirsen, deeming it “investigational and not medically necessary.” Sarepta CEO Ed Kaye reported at the J.P. Morgan Healthcare Conference earlier this month that though the company has been making progress, other insurers have been slow to reimburse as well.
Unlike eteplirsen, nusinersen showed a clear benefit in placebo-controlled trials, and Biogen recently published data showing that the drug reduced the risk of death or permanent need of a ventilator in type 1 SMA patients.
Nonetheless, many expected a fight. Porges recently wrote in a research note that nusinersen’s price was “likely to invite a storm of criticism, up to and including Presidential tweets” and may lead to backlash from insurers. “At the very least… the price is going to force payers to closely scrutinize which patients receive access and limit the overall access provided,” Porges wrote, adding that it “seems certain” that older patients with type 3 or type 4 SMA and milder symptoms will “find it difficult to obtain treatment.” (Earlier this month, Xconomy wrote about the difficulty one such Type 3 SMA patient, Mikhail Memedovich, is having obtaining treatment.). Roger Longman, CEO of drug reimbursement specialist Real Endpoints, said at the time that in conversations, payers indicated they were “beginning to plan how to restrict things.”
Jefferies biotech analyst Brian Abrahams wrote in a note Tuesday it was “slightly surprising” that Anthem “omit[ted] commentary” on nusinersen’s use in Type 2 and Type 3 patients despite the drug’s broad label from the FDA and may “somewhat slow” the drug’s launch. RBC Capital Markets’ Michael Yee added in a different note that nusinersen “was a fast approval based on strong data so over time Anthem is likely to open up coverage more broadly.”