Ultragenyx Pharmaceutical has agreed to sell a rare pediatric disease priority review voucher for $210 million, providing capital to support development of its rare disease therapies and its efforts to reach profitability.
The company received the voucher following FDA approval of Genglycos, or pariglasgene brecaparvovec-opnr, for glycogen storage disease type Ia. Also known as DTX401, Genglycos is the first treatment designed to address the disease’s underlying cause, according to Ultragenyx.
The sale is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.
Howard Horn, chief financial officer and executive vice president of corporate strategy, said proceeds would provide non-dilutive capital to advance therapies for rare and ultra-rare diseases. He said development of Genglycos had itself benefited from funds generated by an earlier priority review voucher sale.
The FDA’s rare pediatric disease voucher program is intended to encourage development of drugs and biological products for the prevention and treatment of certain rare diseases affecting children.
Under the program, a sponsor receiving approval for a qualifying rare pediatric disease product may receive a voucher. The voucher can be used to obtain priority review of a subsequent marketing application for a different product.
Sponsors receive the voucher upon approval of the qualifying product application and may sell or transfer it. Ultragenyx’s agreement converts the voucher awarded for Genglycos into funding for its broader development efforts.
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