Shares of Pharming Group NV (NASDAQ:PHAR) dropped around 16.8% in premarket trading on Monday after the company disclosed that it received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration for its supplemental New Drug Application covering Joenja.
The FDA said it has concerns about possible underdosing in lower-weight pediatric patients for the oral therapy, which is designed to treat activated phosphoinositide 3-kinase delta syndrome (APDS), a rare immune disorder. Regulators asked for additional pediatric pharmacokinetic data to reassess the proposed dosing for children aged 4 to 11, and also flagged issues related to an analytical method used in production batch testing.
Pharming said it believes the matters raised are addressable and that it plans to engage closely with the FDA to define the next steps toward resubmission. The company intends to request a Type A meeting with the agency to clarify requirements and timelines.
“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 was supported by results from an open-label Phase III trial in children aged 4 to 11, which showed reductions in lymphadenopathy and increases in naïve B cells over a 12-week treatment period. The FDA had previously granted Priority Review to the application in October 2023.
Pharming emphasized that the existing FDA approval for Joenja in patients aged 12 and older is unchanged. The drug received approval for that population in March 2023 and remains the only FDA-approved treatment for APDS in the U.S. for patients in that age group.