Pharming Group (PHAR) announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to its supplemental New Drug Application (sNDA) for Joenja (leniolisib). Joenja is an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor being developed as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
The FDA raised concerns regarding the potential for underexposure in lower-weight pediatric patients. To address this, the Agency has requested additional pediatric pharmacokinetic data to reassess the proposed dosing regimen and confirm that children in the lower weight groups can achieve exposure levels comparable to those observed in approved adult and adolescent regimens. The letter also highlighted an issue with one of the analytical methods used for production batch testing, requesting further data and clarification.
Pharming Group stated that it believes the issues outlined in the CRL can be addressed. The company plans to work closely with the FDA to meet the Agency's requirements and determine next steps for resubmission. As part of this process, Pharming intends to request a Type A meeting with the FDA.
Joenja's U.S. FDA approval for the treatment of APDS in patients aged 12 years of age and older is unaffected by this regulatory action.
PHAR closed trading on January 30 at $20.47, down $0.40 or 1.92%. In after-hours trading, the stock rebounded to $20.87, gaining $0.40 or 1.95%.
For More Such Health News, visit rttnews.com
For comments and feedback contact: editorial@rttnews.com
Business News
May 22, 2026 14:46 ET Minutes of the latest Fed policy session was the highlight of the week along with survey data on the U.S. housing market. In Europe, survey data signaled the trends in the euro area private sector. Further, consumer price inflation data from the U.K. was in focus. In Asia, various economic indicators from China drew attention to the health of the economy.