Scholar Rock books $205M voucher sale as SMA drug reaches US market

Scholar Rock has moved from developer to commercial-stage company in the US. In its third-quarter results presentation on 10 November 2026, it said it has started selling Isembyld (apitegromab-mstn), cleared by the FDA for spinal muscular atrophy patients aged two and over who are already on treatment aimed at the SMN protein. The facts provided give no price, sales forecast or launch-uptake data.

The company also said it has signed a definitive agreement to sell its priority review voucher for $205M. These FDA-issued vouchers can be sold on and redeemed to shorten the review of a later application. The facts do not name the buyer or say when the deal closes or how Scholar Rock plans to use the proceeds.

Outside the US, two reviews are under way. Scholar Rock expects a CHMP opinion on its resubmitted European marketing authorisation application in the first half of 2027. In Japan, the PMDA is assessing the company's new drug application under priority review. No decision dates were given for Japan.

On the pipeline, Scholar Rock has begun dosing in FORGE, a Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD). The FDA has granted the programme Fast Track and Orphan Drug designations. The facts supplied do not include trial size, endpoints or timelines for data.