Spinraza regulatory update

FDA approved an NDA from Biogen for Spinraza nusinersen to treat children and adults with spinal muscular atrophy (SMA). It is the first drug approved for the indication. On Dec. 23, 2016, Biogen said it would launch Spinraza in about a week. The company said it priced the product at a wholesale acquisition cost (WAC) of $125,000 per vial. Spinraza is dosed 6 times in the first year and every 4 months in subsequent years, which translates to a WAC of $750,000 in the first year and $375,000 in each subsequent year. The company will offer undisclosed financial assistance through its SMA360 program.

Biogen received a Priority Review voucher under FDA’s rare pediatric disease voucher program upon Spinraza’s approval. The company declined to disclose its plans for the voucher. The approval also triggered a $60 million milestone payment to Ionis, which said it remains eligible for up to $90 million in additional milestones based on regulatory approvals in Europe and Japan, plus tiered royalties up to the mid-teens. In August, Biogen exercised its option under a 2012 deal for worldwide rights to Spinraza after the partners said the therapy met a co-primary endpoint in the Phase III ENDEAR trial to treat infantile-onset SMA (see BioCentury, Jan. 9, 2012 & Aug. 8, 2016). Spinraza also met the primary endpoint of the Phase III CHERISH trial to treat later-onset SMA (see BioCentury, Nov. 14, 2016)...