RARE Daily

Pharming Sells Priority Review Voucher for Pre-Set Price of $22.1 Million

June 1, 2023

Rare Daily Staff

Pharming Group said it has entered into a definitive agreement with Novartis Pharma to sell its Rare Pediatric Disease Priority Review Voucher for a pre-set price of $21.1 million.

The vouchers, which shorten the FDA review time for a new drug to six month from 10 months drug, have been consistently selling for around $100 million. Most recently, Bluebird Bio sold a priority review voucher to Bristol Myers Squibb for $95 million in January.

Pharming was granted the PRV by the U.S. Food and Drug Administration in March 2023 in connection with the approval of Joenja (leniolisib), an oral, selective PI3Kδ inhibitor, for activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare and progressive primary immunodeficiency, in adult and pediatric patients 12 years of age and older.

Pharming and Novartis set the sale price for the PRV in an August 2019 exclusive license agreement for leniolisib.

The Rare Pediatric Disease Priority Review Voucher program is intended to encourage development of new drug and biological products for prevention and treatment of certain rare pediatric diseases. A PRV may be issued to the sponsor of a rare pediatric disease product application and entitles the holder to priority review of a single New Drug Application or Biologics License Application, which reduces the target review time and could lead to an expedited approval.

The sponsor receives the PRV upon approval of the rare pediatric disease product application and it can be sold without limitation, subject to applicable FDA requirements for filing and use.

“The sale of the PRV will further strengthen Pharming’s financial position and help fund pipeline developments, including new indications for leniolisib,” said Jeroen Wakkerman, CFO of Pharming.


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