Rare Daily Staff

BioMarin Pharmaceutical said that it has entered into a definitive agreement to sell the Rare Pediatric Disease Priority Review Voucher it obtained in April for $125 million.

The company, which did not disclose the buyer, obtained the voucher when it gained U.S. Food and Drug Administration approval for Brineura, an enzyme replacement therapy to treat patients with late infantile neuronal ceroid lipofuscinosis type 2, a form of Batten disease. BioMarin CEO Jean-Jacques Bienaimé said the company will use the proceeds from the sale to fund development of its pipeline.

FDA grants Rare Pediatric Disease Priority Review Vouchers as a way to encourage the development of treatments for rare pediatric diseases. The vouchers are transferable and can be used to accelerate the review of large market therapeutics. The priority review voucher entitles the holder to priority review of a single New Drug Application or Biologics License Application. 

BioMarin was the first company to receive and sell a Rare Pediatric Disease Priority Review Voucher. It obtained that voucher for Vimizim, its enzyme replacement therapy for MPS IVA, also known as Morquio A syndrome. In 2014 it sold the voucher to Regeneron Ireland, a wholly-owned subsidiary or Regeneron, for $67.5 million.

The company said it will record the sale as a $125 million gain on sale of an intangible asset and it will also be associated with approximately $25 million of income tax expense.  As a result of the sale, the company will update its GAAP Net Loss guidance by the $100 million net after tax gain, and for full-year 2017, the GAAP Net Loss guidance will be reduced to between a $10 million loss and $30 million loss. The sale of the voucher will be excluded from non-GAAP income and consequently the non-GAAP income guidance for the full-year 2017 is unchanged at $60 million to $80 million.  

November 27, 2017

Photo: BioMarin CEO Jean-Jacques Bienaimé