The specialty pharmaceutical company, Knight Therapeutics Inc., through one of its wholly owned subsidiaries, has sold its Neglected Tropical Disease Priority Review Voucher (PRV) to Gilead Sciences for $125 million. The PRV was granted to Knight in March of this year with the approval by the US Food and Drug Administration (FDA) of Impavido (miltefosine) for treatment of patients with visceral, mucosa,l and cutaneous leishmaniasis.

Developed by the FDA in 2007 under the Food and Drug Administration Amendments Act, a this type of PRV is an incentive for companies to invest in new drugs and vaccines for neglected tropical diseases. Leishmaniasis is considered a neglected tropical disease and is one of 16 tropical diseases that qualify for a PRV. A PRV is a transferrable asset that entitles the holder to a priority review for a drug of its choice. Under Prescription Drug User Fee Act, the FDA sets a target of 10 months from the date the FDA accepts the application for a standard review of a new drug application (NDA) or a biologic license application (BLA). A priority review means that the FDA reduces its target review time for a NDA or BLA to six months from the date the FDA accepts the application, thereby speeding up the initial review of the drug.

This is a second example of a sale of a PRV this year. In June 2014, Sanofi and Regeneron Pharmaceuticals acquired from BioMarin Pharmaceuticals a rare pediatric disease PRV in connection with their BLA submission in the US for alirocumab, a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor for treating high cholesterol. BioMarin had received the PRV through FDA's Rare Pediatric Disease Priority Review Voucher Program. The Pediatric PRV was created by the 2012 Food and Drug Administration Safety and Innovation Act and is intended to encourage the development of treatments for rare pediatric diseases. Companies that receive a voucher may use it or transfer the voucher, including by sale, to other organizations. BioMarin had received the voucher in connection with FDA approval of its orphan drug, Vimizin (elosulfase alfa), an enzyme-replacement therapy for treating Mucopolysaccharidosis Type IVA (Morquio A).

Source: Knight Therapeutics