FDA Delays Decision on Rocket Pharmaceuticals’ Gene Therapy for Rare Disorder

In a recent announcement, Rocket Pharmaceuticals, Inc. has encountered a three-month postponement by the U.S. Food and Drug Administration (FDA) for its groundbreaking autologous gene therapy aimed at treating a rare white blood cell disorder. This decision shifts the expected date to June 30, allowing the FDA additional time to thoroughly review the therapy’s chemistry, manufacturing, and controls.

The therapy, known as KRESLADI™ (marnetegragene autotemcel), targets severe Leukocyte Adhesion Deficiency-I (LAD-I), a condition affecting approximately 800 to 1,000 individuals in the US and Europe. The delay, according to Rocket’s announcement, stems from the need for further clarification of Chemistry, Manufacturing, and Controls (CMC) information, in response to FDA requests, rather than any deficiencies in the application itself.

Market Reaction and Analyst Insights

Following the news, Rocket’s stock ($RCKT) saw a 4% decline in early trading on Tuesday. However, industry analysts, notably from William Blair and Stifel, remain optimistic. The delay is attributed more to FDA staffing challenges rather than concerns over the therapy’s efficacy or manufacturing processes. Historical precedents, such as the three-month delay experienced by Krystal Biotech’s Vyjuvek, serve as a reminder that such postponements, while impactful, are not uncommon and can still result in eventual approval.

William Blair analysts have also highlighted the potential for Rocket to receive a priority review voucher, which could significantly extend its financial runway and foster revenue growth. Stifel analysts echoed a sense of reassurance, noting that the FDA did not request additional clinical data, and the manufacturing facilities in question have been in use by Rocket since preclinical stages.

About KRESLADI™ and LAD-I

KRESLADI™ employs a lentivirus vector to deliver a functional copy of the ITGB2 gene to patients. This gene is crucial for the production of a protein that enables white blood cells to adhere together and efficiently exit blood vessels to combat infections. The treatment has shown promising results in a global Phase I/II trial, demonstrating a 100% survival rate at 12 months post-infusion for all nine patients treated, alongside significant improvements in infection rates and wound healing capabilities.

LAD-I is characterized by the body’s inability to properly direct leukocytes, or white blood cells, to sites of infection, resulting in an increased susceptibility to recurrent bacterial infections and a typically shortened life expectancy. The condition is due to mutations in the CD18 glycoprotein, leading to a deficiency in various adhesion molecules necessary for immune response.

Looking Forward

Despite the delay, Rocket Pharmaceuticals remains dedicated to advancing this vital therapy to market. Dr. Gaurav Shah, CEO of Rocket Pharma, expressed confidence in the ongoing collaboration with the FDA and the shared commitment to bringing novel, potentially curative gene therapies to patients in need. The positive outcomes from the Phase I/II trial underscore the potential of KRESLADI™ to significantly improve the lives of individuals with LAD-I, marking a significant step forward in the treatment of rare immunodeficiency disorders.

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