Pharming Stock Drops After FDA Rejects Joenja sNDA

Key Summary of FDA's Response

Pharming Group NV (NASDAQ:PHAR) stock experienced a sharp decline after the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL), effectively rejecting the supplemental New Drug Application (sNDA) for its drug Joenja for use in younger pediatric patients.

Overview of the Regulatory Concerns

The FDA expressed concerns regarding potential underexposure to the treatment in children aged 4 to 11 with lower body weights. The agency has requested additional pediatric pharmacokinetic data before it can reassess the proposed doses. Furthermore, the CRL identified issues with an analytical method used for testing production batches. Pharming stated it believes the issues are addressable and intends to work closely with the FDA.

Resulting Impact on Pharming's Stock

The announcement led to a significant negative reaction in the market, with Pharming's stock plunging 16.8% in premarket trading. This development delays the potential market expansion for Joenja. It is important to note that this decision does not impact the drug's existing approval for patients aged 12 and older, which remains the only approved treatment for APDS in that age group in the U.S.

Summary and Outlook

Despite the setback, Pharming Group's leadership has affirmed its dedication to making Joenja available to the younger pediatric population. The company plans to request a Type A meeting with the FDA to clarify the path to resubmission. The outcome of this meeting and the timeline for gathering new data will be key factors for investors to watch moving forward.

FAQ

Q: Why did Pharming Group's stock fall?
A: The stock fell after the U.S. FDA issued a Complete Response Letter for its drug Joenja's application for pediatric use, citing data concerns and requesting more information before approval.

Q: What is a Complete Response Letter (CRL)?
A: A CRL from the FDA indicates that a new drug application cannot be approved in its present form. It details the specific deficiencies the applicant must address.

Q: Does this affect the current approval of Joenja?
A: No, the existing FDA approval for Joenja to treat patients 12 years of age and older is not affected by this regulatory action.

Source: Investing.com