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FDA oks Pharming rare immune disorder drug

The U.S. FDA has approved Pharming Group’s PI3K? inhibitor for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adult and pediatric patients 12 years of age and older.

APDS is a rare genetic disorder that leads to a weakened immune system. The drug, branded Joenja, is the first and only treatment approved in the U.S. for APDS.

The FDA evaluated the Dutch biotech’s New Drug Application (NDA) for Joenja under priority review and approved the drug based on findings from a multinational, placebo-controlled, randomized phase 2/3 clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older. 

Pharming licensed the late-phase treatment from Novartis for $20 million upfront back in August 2019. Now, pursuant to the agreement, Pharming will make milestone payments to Novartis and another unnamed party totaling $10.5 million, as well as additional milestone payments to Novartis in an aggregate amount of up to $190 million if sales milestones are achieved.

Also part of that deal: Novartis has the right to purchase the priority review voucher that Pharming earned through Joenja’s approval as a treatment for a rare pediatric disease. According to Pharming, Novartis can purchase the voucher for “a small minority share of the value.”

Pharming is expecting to launch Joenja in the U.S. in early April.