Ultragenyx shares rise after agreeing to sell FDA priority review voucher for $210 million

Ultragenyx Pharmaceutical (NASDAQ:RARE) shares rose over 3.2% on Wednesday morning after the biopharmaceutical company agreed to sell a Rare Pediatric Disease Priority Review Voucher for $210 million.

The voucher was awarded to Ultragenyx after the US Food and Drug Administration approved GENGLYCOS, also known as DTX401, for glycogen storage disease (GSD) type Ia. A voucher can be redeemed by its holder to obtain priority review of a subsequent marketing application for a different product, and sponsors can sell or transfer the voucher to another company.

Ultragenyx said the transaction will provide significant non-dilutive capital to support the development of therapies for rare and ultra-rare diseases and help advance its path to profitability. The company said GENGLYCOS itself benefited from capital generated through a previous priority review voucher sale, highlighting the role of the FDA programme in funding rare disease drug development.

"Monetizing this PRV provides significant non-dilutive capital to advance our efforts to bring forward first-ever therapies for rare and ultra-rare diseases, and supports our path to profitability," said Howard Horn, chief financial officer and executive vice president, corporate strategy.

The sale remains subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.

GENGLYCOS is designed to address the underlying cause of GSDIa, a rare genetic disorder, and was the first treatment approved for the disease that targets its underlying cause. The FDA’s Rare Pediatric Disease Priority Review Voucher programme is intended to encourage development of drugs and biological products for certain rare childhood diseases.

Ultragenyx, which develops treatments for serious rare and ultra-rare genetic diseases, said the proceeds will strengthen its financial position as it advances its pipeline. The company appointed Jefferies as exclusive financial adviser and Gibson, Dunn and Crutcher as legal counsel for the transaction.

The agreement gives Ultragenyx a substantial source of non-dilutive funding while allowing the company to monetize an asset awarded through the FDA’s rare disease incentive programme.