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Stock Markets February 2, 2026

Pharming Shares Fall After FDA Sends Complete Response Letter on Pediatric sNDA for Joenja

Regulatory letter flags potential underexposure in younger children and analytical testing issues; company plans to seek a Type A meeting with FDA

By Avery Klein PHAR
Pharming Shares Fall After FDA Sends Complete Response Letter on Pediatric sNDA for Joenja
PHAR

Pharming Group NV shares dropped sharply in premarket trading after the U.S. Food and Drug Administration issued a Complete Response Letter for the supplemental New Drug Application for Joenja, citing concerns about possible underexposure in lower weight pediatric patients and problems with an analytical method used in production batch testing. The company said it will work with the FDA and pursue a Type A meeting to discuss next steps toward resubmission.

Key Points

  • Pharming Group stock fell 16.8% in premarket trading after the FDA issued a Complete Response Letter for the Joenja sNDA.
  • The FDA requested additional pediatric pharmacokinetic data to reassess dosing for children aged 4 to 11 and identified problems with an analytical method used in production batch testing.
  • Joenja's approval for patients 12 and older remains in place; the sNDA was supported by an open-label Phase III study showing improvements over 12 weeks and had received Priority Review in October 2023.

Pharming Group NV (NASDAQ:PHAR) saw its stock decline 16.8% in premarket trading Monday following the release of a Complete Response Letter from the U.S. Food and Drug Administration related to the supplemental New Drug Application for Joenja.

The FDA identified two primary areas of concern in the CRL. First, regulators flagged potential underexposure to the oral treatment in lower weight pediatric patients and asked for additional pediatric pharmacokinetic data to reassess the proposed dosing for children aged 4 to 11 years. Second, the agency raised issues with an analytical method that had been used for production batch testing.

Pharming said it believes the topics raised in the letter can be addressed and that it will engage with the FDA to determine the path forward for resubmitting the application. The company indicated it intends to request a Type A meeting with the agency to discuss next steps.

Company statement

Fabrice Chouraqui, Pharming's chief executive officer, said the company is disappointed in the FDA's response but remains committed to making Joenja available to pediatric patients aged 4 to 11 with activated phosphoinositide 3-kinase delta syndrome, or APDS.

Basis of the sNDA

The supplemental application was supported by data from an open-label Phase III study in children aged 4 to 11 years. According to the submission, the trial showed improvements in lymphadenopathy and an increase in naïve B cells over a 12-week period. The FDA had previously granted Priority Review to this sNDA in October 2023.

Existing approval and market status

Pharming emphasized that Joenja's existing FDA approval for the treatment of APDS in patients 12 years of age and older remains unaffected by the CRL. That approval was granted in March 2023, and Joenja currently represents the only approved U.S. treatment for APDS in that older pediatric and adult age group.

Outlook

Pharming will coordinate with the FDA to address the specified concerns and pursue regulatory dialogue via a Type A meeting to clarify the requirements for resubmission. The company has not provided a timeline for resolving the issues outlined in the CRL.

Risks

  • Regulatory uncertainty - The FDA's request for more pediatric pharmacokinetic data and issues with analytical testing create uncertainty about the timing and outcome of any resubmission, affecting the company's regulatory pathway and near-term commercial prospects in the pediatric 4-11 age group.
  • Manufacturing and quality testing - Identified problems with an analytical method used for production batch testing could require technical remediation and additional validation work, potentially delaying product availability and increasing costs.
  • Market reaction - The stock's sharp premarket decline reflects near-term market sensitivity to regulatory setbacks in the pharmaceutical and biotech sectors, which may influence investor sentiment and access to capital.

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