Investing.com -- Pharming Group NV (NASDAQ:PHAR) stock plunged 16.8% in premarket trading Monday after the company announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its supplemental New Drug Application (sNDA) for Joenja.
The FDA raised concerns about potential underexposure in lower weight pediatric patients for the oral treatment, which targets activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. Regulators requested additional pediatric pharmacokinetic data to reassess proposed doses for children aged 4 to 11 years. The agency also identified issues with an analytical method used for production batch testing.
Pharming said it believes it can address the issues outlined in the letter and plans to work closely with the FDA to determine next steps for resubmission. The company intends to request a Type A meeting with the FDA to discuss the path forward.
"While we are disappointed in the FDA’s response, we remain dedicated to making Joenja available to pediatric patients aged 4-11 with APDS," said Fabrice Chouraqui, Chief Executive Officer of Pharming.
The sNDA submission was based on data from an open-label Phase III study in children aged 4 to 11 years, which showed improvements in lymphadenopathy and increased naïve B cells over 12 weeks. The FDA had previously granted the application Priority Review in October 2023.
Importantly, Joenja’s existing FDA approval for treating APDS in patients 12 years of age and older remains unaffected by this regulatory action. The drug received this approval in March 2023 and currently represents the only approved treatment for APDS in the U.S. for patients in this age group.
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