Fighting an uphill battle to get payers to cover its $750,000 first-year price tag on Spinraza (nusinersen), Biogen is posting another round of stellar data to back its use among older children with rare cases of spinal muscular atrophy.
Researchers tested the drug in 126 SMA patients who were between the ages of 2 and 12 and generally suffered from Type 2 or Type 3 forms of the neuromuscular disease. As we’ve already seen with infants under 6 months suffering early-onset disease, there was a clear response, with a 3.9 point improvement at month 15 in the Hammersmith Functional Motor Scale Expanded (HFMSE) score, compared to a 1.0 point drop in patients in the placebo arm.
“In CHERISH, most children with later-onset SMA treated with Spinraza saw improvements in motor function and stabilization or slowing of disease progression,” said Dr. Richard Finkel, chief of neurology, Nemours Children’s Hospital, Orlando, Florida. “As a physician who has spent 37 years treating children with SMA, it’s incredibly encouraging to see some patients on Spinraza achieve milestones such as crawling and standing with assistance within the clinical trial. These kinds of clinically meaningful improvements are unprecedented and give new hope to individuals with SMA and their families.”
But it’s payers that Biogen most wants to impress now.
“We’re hopeful that when [health plans] see this data, they will update their policies” to cover the drug for older children, Biogen Chief Medical Officer Al Sandrock told the hometown newspaper Boston Globe.
Biogen, partnered with Ionis, stunned some analysts with its price, which drops to half that $750,000 in the second year, or $1.5 million for the first three years of treatment. Leerink’s Geoffrey Porges noted recently that the price could turn Spinraza into the “Sovaldi of rare disease drugs,” comparing it to the Gilead hep C cure that sparked an outcry over the $1,000-per-pill cost.
Biogen badly needs to make a success of Spinraza. Its flagship drug Tecfidera has been flagging as new competitors hit the MS market, and it has a thin pipeline of CNS drugs that most analysts would like to see fattened through new deals. Along those lines Biogen lined up a $300 million upfront deal a few days ago to grab an anti-tau drug from Bristol-Myers Squibb. And more deals may be on the way.
“We’re moving, and we’re back in terms of augmenting the pipeline,” research and development head Michael Ehlers told Bloomberg on Friday.
President Trump, meanwhile, has been castigating the industry for astronomical drug prices, though rare disease drugs have generally avoided the spotlight among all the price controversies that have erupted in the last couple of years.
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