Pharming has successfully secured reimbursement for its ultra-rare disease drug, Joenja, in England. This follows a reversal of the National Institute for Health and Care Excellence (NICE)’s initial rejection. The decision comes as the drug continues to undergo regulatory review in the European Union.
NICE’s change of heart came after Pharming presented additional data and adjusted the drug’s price. The list price for Joenja is £352,000 per patient annually.
This represents a significant development for patients with Activated PI3K-delta Syndrome (APDS), the condition Joenja treats.
