Pharming’s Portfolio Grows As Ultra-Rare Disease Drug Joenja Is Approved

Priced At $547,500 Per Year

The first ever approval for ultra-rare immunodeficiency disease APDS gives Pharming its second product on the market but patient diagnosis will be a major challenge.

Rare disease people
Pharming will provide free genetic testing to help diagnose patients with the ultra-rare condition. • Source: Shutterstocks

Pharming has gained US Food and Drug Administration approval for Joenja, a new disease-modifying treatment for the ultra-rare disease activated phosophoinositide 3-kinase delta syndrome (APDS).

The approval makes Joenja (leniolisib) the first drug approved to treat APDS and it is licensed in adult and pediatric...

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