Rare Pediatric Disease Priority Review Voucher Sells for $130 Million

Ultragenyx will present voucher to Novartis upon closing of transaction

Ultragenyx Pharmaceutical, Inc., has agreed to sell its rare pediatric disease priority review voucher (PRV) to Novartis for $130 million. Ultragenyx was awarded the voucher under an FDA program intended to encourage the development of treatments for rare pediatric diseases. The company received the PRV in November when vestronidase alfa (Mepsevii) was approved by the FDA for the treatment of children and adults with mucopolysaccharidosis VII (also called Sly syndrome).

"The sale of the PRV provides us with an important source of nondilutive capital to help advance our pipeline of rare and ultra-rare therapies, and accelerates the availability of these potential therapies to patients," Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx, said in a press release.

Under the agreement, Ultragenyx will receive a lump sum payment of $130 million upon the closing of the transaction, which is subject to customary closing conditions including antitrust review.

This FDA 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, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a PRV that can be redeemed to receive a priority review of a subsequent marketing application for a different product. The sponsor receives the PRV upon approval of the rare pediatric disease product application, and it can be sold or transferred.

Source: Ultragenyx; December 18, 2017.