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

Priced At $547,500 Per Year

 
• By Andrew McConaghie

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).

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Pipeline Watch: Eight Approvals And Three Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.