Pharming Hit by FDA Setback on Joenja Pediatric Expansion but Adult Approval Unaffected

Pharming Group ( (NL:PHARM) ) has issued an update.

Pharming Group has received a Complete Response Letter from the U.S. FDA for its supplemental New Drug Application seeking to extend Joenja (leniolisib) to children aged 4 to 11 years with APDS, after regulators raised concerns about potential underexposure in lower-weight pediatric patients and requested additional pharmacokinetic and analytical testing data. While the setback delays potential label expansion into a younger, currently untreated patient population, Joenja’s existing U.S. approval for patients 12 and older remains intact, and Pharming plans to work closely with the FDA, including requesting a Type A meeting, to address the issues and resubmit, a process that could impact the timing of its pediatric growth strategy in the rare disease space.

The most recent analyst rating on (NL:PHARM) stock is a Hold with a EUR2.00 price target. To see the full list of analyst forecasts on Pharming Group stock, see the NL:PHARM Stock Forecast page.

More about Pharming Group

Pharming Group is a biopharmaceutical company focused on developing and commercializing rare disease therapies, notably Joenja (leniolisib), an oral PI3Kδ inhibitor that is the first targeted treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in patients aged 12 and older. The company is expanding its presence in the global rare immunology market through ongoing regulatory reviews in multiple regions and additional clinical trials in pediatric APDS and other primary immunodeficiencies with immune dysregulation.

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Average Trading Volume: 17,783,861

Technical Sentiment Signal: Buy

Current Market Cap: €1.2B

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