Rare Daily Staff
Liminal BioSciences said its subsidiary Prometic Biotherapeutics has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher for $105 million to an undisclosed buyer.
The U.S. Food and Drug Administration granted the PRV to Prometic with the approval of Ryplazim, its treatment for patients with plasminogen deficiency type 1. Individuals with plasminogen deficiency type 1 lack a protein called plasminogen, which is responsible for the ability of the body to break down fibrin clots. Plasminogen deficiency leads to an accumulation of fibrin, causing the development of lesions that can impair normal tissue and organ function and may lead to blindness when these lesions affect the eyes.
The active ingredient in Ryplazim is plasminogen, purified from human plasma. Treatment with Ryplazim helps to increase the plasma level of plasminogen – enabling a temporary correction of the plasminogen deficiency and reduction or resolution of the lesions.
The Rare Pediatric Disease designation program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases.
The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. If a biologics license application for a therapy granted Rare Pediatric Disease designation, it may be eligible to receive a priority review voucher that may be sold or transferred.
The voucher, which reduces the standard review time for a new drug application by four months, is potentially lucrative because it is transferable. The latest sale is a slight increase over the previous sale of a priority review voucher that Rhythm Pharmaceuticals made to Alexion in January for $100 million.
“We are pleased to announce the sale of the PRV, which will provide an important source of non-dilutive capital to advance the continued development of our small molecule pipeline,” said Bruce Pritchard, CEO of Liminal BioSciences. “This transaction is also another key milestone in completing the divestiture of our remaining plasma-derived therapeutics business to Kedrion.”
Photo: Bruce Pritchard, CEO of Liminal BioSciences