AstraZeneca Redeems Rare Pediatric Disease Priority Review Voucher for Myasthenia Gravis Drug

The FDA said that AstraZenca has redeemed a rare pediatric disease priority review voucher for Ultomiris (ravulizumab-cwvz).

The FDA approved Ultomiris in April for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80 percent of people living with the disease.

The rare, chronic autoimmune neuromuscular disease affects approximately 90,000 people in the U.S.

Ultomiris is also approved to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) in adults and children one month of age and older.

For priority reviews, the FDA aims to take action on an application within six months, compared to 10 months under standard reviews.