FDA Approval Delay for Rocket Pharma's Pediatric Gene Therapy

Rocket Pharma disclosed that the US health regulatory agency did not approve its investigational gene therapy designed to treat a rare pediatric disease that severely compromises immune system functionality. This announcement resulted in a significant drop of over 12% in the company's stock price during early trading hours.

The US Food and Drug Administration (FDA) issued a detailed letter to Rocket, requesting that the company supply additional information on specific processes related to its gene therapy candidate, Kresladi, to facilitate a thorough review. Rocket is aiming to achieve the FDA's first approval for Kresladi, which is intended to treat severe Leukocyte Adhesion Deficiency-I (LAD-I) patients. This rare disorder affects approximately 800 to 1,000 individuals in the US and Europe.

LAD-I patients suffer from a genetic mutation in the ITGB2 gene, which impairs the ability of white blood cells to adhere properly to blood vessel walls at infection sites. This defect can cause severe immune dysfunction and recurrent infections. Rocket Pharma has engaged in discussions with the FDA to address the information needed to accelerate the approval process.

The marketing application for Kresladi is supported by early to mid-stage clinical trial data, which showed that all nine patients treated survived for at least 12 months post-treatment. Yet, in February, the FDA extended the review period for some process-related data concerning this gene therapy.

Tyler Van Buren, an analyst from TD Cowen, commented on the situation, suggesting that the delay seems to be due to internal bandwidth issues within the FDA rather than any specific concerns about Rocket's therapy. He further pointed out that while Kresladi represents a relatively small market opportunity for Rocket Pharma, its successful approval would significantly validate the company's lentiviral platform. This platform has the potential to offer treatment options to broader patient populations.

At present, the only available treatment for LAD-I is bone marrow transplantation, a procedure that is often difficult to secure and carries substantial risks.

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