The company expressed confidence that it can resolve the FDA’s inquiries.
- FDA feedback focused on dosing and testing questions, not safety or effectiveness concerns.
- Joenja’s existing approval and sales in patients aged 12 and older remain unchanged.
- Pharming said it plans to work with regulators and pursue a resubmission path.
Pharming Group shares drew heightened retail chatter on Monday after the company said it received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for its application to expand the use of Joenja in younger pediatric patients, while signaling confidence it can address the agency’s concerns.
FDA Requests Additional Pediatric Data
Dutch drug developer Pharming announced that it had received a Complete Response Letter from the FDA on its filing for the approval of Joenja in children aged 4-11 with activated phosphoinositide 3-kinase delta syndrome (APDS).
APDS is a rare primary immunodeficiency disorder in which the immune system does not function properly, resulting in recurrent infections and other complications associated with immune dysfunction. Pharming’s Joenja is an oral medication indicated to treat APDS.
The company said the FDA was concerned that younger children may not be receiving sufficient doses at the proposed levels and requested additional data to confirm appropriate dosing in younger patients. The agency also flagged a technical issue with a manufacturing test and requested additional information.
Pharming expressed confidence that it can resolve both inquiries. The company added that it plans to meet with the FDA to discuss next steps for resubmission, including a Type A meeting.
Approval For Older Patients Unaffected
The company emphasized that the Complete Response Letter does not impact Joenja’s existing FDA approval for adult and pediatric patients aged 12 years and older, which has been in place since March 2023.
CEO Fabrice Chouraqui said Pharming remains focused on making Joenja available to younger children with APDS, noting that there are currently no approved targeted treatments globally for patients under the age of 12.
Trial Results Backing The Application
The application was backed by data from a late-stage clinical study in children aged 4 to 11. Pharming said that over 12 weeks of treatment, children showed reduced lymph node swelling and healthier immune cell levels, signs that the drug was helping correct the immune problem caused by APDS.
Safety data from up to eight months of treatment were also included in the submission. The company said all treatment-emergent adverse events were mild to moderate, no drug-related serious adverse events were reported, and all patients completed the 12-week treatment period.
In October 2025, the FDA granted the application Priority Review, citing the potential for significant improvements in the treatment of a serious condition.
How Did Stocktwits Users React?
On Stocktwits, retail sentiment for Pharming was ‘bullish’ amid ‘high’ message volume ahead of the company’s Virtual Investor Day on Tuesday.
One user called the FDA response a “soft” CRL, noting that it focused on dosing and testing issues rather than safety, and that Joenja remains approved and generating revenue for patients aged 12 and older.
Another user said, “will buy the dip. Hopefully it’s a large drop”
U.S.-listed shares of Pharming have surged over 130% over the past 12 months.
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