Biogen’s spinal muscular atrophy med Spinraza hit the market running after its launch, helping fuel growth at the drugmaker as its MS franchise faced a slowdown. Now, the company is touting an interim analysis showing that infants with SMA can benefit "dramatically" from the drug if they’re treated before symptoms develop.
The Nurture study is testing Spinraza in 25 patients genetically diagnosed with the disease and treated before symptoms developed. At an interim analysis in May 2018, all patients were alive, and none required tracheostomy or permanent ventilation. The patients were 14 months or older at the time of the analysis.
Plus, 88% percent of the patients were able to walk, either with assistance or independently, and all were able to sit without support. In a note, Evercore ISI analyst Josh Schimmer said the results were "pretty amazing."
Wildon Farwell, M.D., Biogen senior medical director of clinical development, said in a statement the data show that “early diagnosis and treatment with Spinraza has the potential to dramatically change the course of SMA."
“This is the longest available span of data on infants with SMA who began treatment in a presymptomatic period and indicates that children treated early with Spinraza can achieve motor milestones they would likely not attain without treatment,” Farwell added.
Biogen launched Spinraza after an approval in late 2016, and the drug quickly captured headlines for its price of $750,000 for the first year and $375,000 annually after that. Despite some concern about the price, the drug has far surpassed early expectations, leaping out to $884 million in sales during its first full year on the market.
As of this summer, the drug has won approval in at least 24 countries. More than 5,000 patients were taking Spinraza as of June, including those in an expanded access program, clinical trials and a postmarketing study, according to Biogen.
Biogen presented the new Spinraza data at the World Muscle Society’s annual congress in Mendoza, Argentina.