RARE Daily

Bluebird Bio Sells Priority Review Voucher to Argenx for $102 Million

November 30, 2022

Bluebird Bio said that it has entered into a definitive agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) to Argenx for $102 million.

A PRV can shorten the review period of a marketing application from 10 months to six months. Argenx expects to redeem the PRV for a future marketing application for efgartigimod, its first-in-class neonatal Fc receptor (FcRn) blocker, which won marketing approval last December as Vyvgart for the treatment of adults with generalized myasthenia gravis.

“We have demonstrated proof-of-concept in four autoimmune diseases with our first-in-class FcRn blocker, efgartigimod, and are planning to be active in fifteen disease targets by 2025. With a priority review voucher available, we hope to expedite the approval process for one of our current or future indications to more quickly reach the patients who are in serious need of a new treatment option,” said Tim Van Hauwermeiren, CEO of Argenx.

Bluebird was granted two PRVs upon the U.S. Food and Drug Administration approvals of two gene therapies: Zynteglo for the treatment of beta-thalassemia in adult and pediatric patients requiring regular red blood cell transfusions on August 17, and Skysona for the treatment of early, active cerebral adrenoleukodystrophy on September 16.

“With the sale of our first priority review voucher, we have significantly strengthened our financial outlook,” said Andrew Obenshain, CEO of Bluebird Bio. “As momentum builds across our business, this non-dilutive capital further bolsters the ongoing launches of our two recently approved gene therapies and the execution of near-term, value-creating milestones, including the planned submission and subsequent FDA review of our biologics licensing application for lovo-cel for sickle cell disease.”

Bluebird will receive a payment of $102 million upon closing of the transaction, which remains subject to customary closing conditions, including anti-trust review. The company continues to explore additional financing opportunities, including the monetization of its second PRV, and anticipates providing full year 2023 guidance early next year.

The Rare Pediatric Disease Priority Review Voucher Program is intended to encourage development of new drug and biological products for the prevention and treatment of certain rare pediatric diseases. Under this program, upon approval, the FDA awards priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. The voucher can be redeemed to receive priority review of a subsequent marketing application for a different product. PRVs may be sold or transferred, and there is no limit on the number of times a PRV can be transferred.

Author: Rare Daily Staff

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