- Biogen and partner Ionis Pharmaceuticals picked up approval for Spinraza (nusinersen) for the treatment of all subtypes of spinal muscular atrophy (SMA) on Dec. 23, making it the first licensed drug for the condition.
- Biogen announced shortly after approval that Spinraza will be $125,000 per vial — making the cost $750,000 in the first year of treatment and $375,000 for each following year for a drug that will need to be taken for a lifetime.
- Launch is expected in early January, impacted by front-loading of frequent dosing in new patients, but potentially delayed by physician training and other set-up requirements.
Just before the Christmas break, Biogen and Ionis Pharmaceuticals gained approval for Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA), well before its PDUFA date of May 26, 2017. This is based on Phase 3 trials with positive read-outs for the drug's use in the muscle-weakening disorder, including reduced mortality in Type 1 disease.
Biogen exercised its option to snag the rights to nusinersen from developer Ionis in August 2016 for $75 million; Ionis will also be eligible for an estimated 13-16% royalties worldwide and up to $150 million in additional milestones. At first glance, the price and the fact that this is the first to market for SMA makes it look like the company would recoup its investment in no time, even for an ultra-orphan disease. Jefferies, for example, predicts worldwide sales of $1.6 billion by 2021, with net royalties to Ionis of $218 million.
However, the challenge for Biogen will be that the payers and the public are currently very sensitive to high drug prices. Stories about the costs of potentially curative hepatitis C drugs like Gilead's Solvadi (sofosbuvir) or about price gouging by specialty pharma companies have put the entire industry on edge.
Jefferies analysts noted that the price tag for Spinraza was at the high end of expectations.
A potential competitor is also on the horizon. AveXis' AVXS-101 gene therapy for SMA is currently in clinical trials: a pivotal trial for Type 1 and a trial for Type 2 is planned for the first half of 2017, with possible expansion into Type 3.
The AveXis drug has breakthrough therapy designation and could be launched as early as 2018. While Spinraza has the advantage of a broad approval, including adult and pediatric approval, AVXS-101 as a gene therapy has potential of duration of effect, which is ongoing at 2+ years in some patients according to trials. This length of efficacy could mean prices as high as $1.1 million for AVXS-101, Jefferies analysts added in a recent note to investors.