Rare Daily Staff

Rare disease drugmaker Ultragenyx Pharmaceutical said it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher to Novartis for $130 million.

The U.S. Food and Drug Administration awarded Ultragenyx the voucher in November when it won approval for Mepsevii, its enzyme replacement therapy to treat patients with MPS VII, or Sly syndrome. The FDA grants priority review vouchers as a incentive to encourage the development of treatments for rare pediatric diseases.

The priority review voucher, which is transferable, can be used to shorten the FDA review time for an application to market a new drug to six months from ten months.

“The sale of the PRV provides us with an important source of non-dilutive capital to help advance our pipeline of rare and ultra-rare therapies, and accelerates the availability of these potential therapies to patients,” said Emil Kakkis, president and CEO of Ultragenyx.

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.

December 18, 2017

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