BioNTech Buys Scholar Rock’s Priority Review Voucher for $205 Million

Rare Daily Staff

BioNTech, the German drugmaker best known for the COVID-19 vaccine it developed with Pfizer, has agreed to pay $205 million in cash to Scholar Rock for its priority review voucher.

BioNTech plans to use the voucher on an experimental cancer drug from its late-stage pipeline. It did not say which one.

The U.S. Food and Drug Administration awarded Scholar Rock the voucher on Sept. 11, when it approved Isembyld (apitegromab) for spinal muscular atrophy (SMA). SMA is a rare neuromuscular disease that destroys the nerve cells controlling muscles and can leave patients unable to walk, swallow or breathe on their own.

Existing SMA drugs work on the faulty genetics behind the disease. Isembyld is the first to target the muscle itself. It blocks myostatin, a protein that limits muscle growth. The drug is approved for patients 2 and older who are already taking one of those genetic treatments. In the main trial, patients on Isembyld improved their motor function, while patients on the genetic treatment alone lost ground.

Congress created the Rare Pediatric Disease Priority Review Voucher program in 2012 to give pharmaceutical companies an incentive to develop new treatments for rare pediatric diseases. A company that wins approval for a rare pediatric disease therapy receives a voucher. The voucher can be used to cut the FDA’s review of a later drug application to six months from the standard 10 months.

Because the vouchers can be sold, they are potentially lucrative. They have become an important source of nondilutive funding for rare disease drug developers, meaning money raised without issuing new shares.