FDA Again Delays Approval of Rocket Pharmaceuticals' Gene Therapy Treatment

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Bill McColl
Bill McColl
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Bill McColl has 25+ years of experience as a senior producer and writer for TV, radio, and digital media leading teams of anchors, reporters, and editors in creating news broadcasts, covering some of the most notable news stories of the time.

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Published June 28, 2024
The U.S. Food and Drug Administration headquarters in White Oak, Maryland, on Aug. 25, 2020

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Key Takeaways

  • Rocket Pharmaceuticals' experimental treatment for a rare immune disorder in children was delayed again by the Food and Drug Administration (FDA).
  • Regulators sent a Complete Response Letter to the company, asking for more information on Kresladi.
  • The FDA was initially expected to grant approval in March, but delayed it for three months to complete its review.

Rocket Pharmaceuticals (RCKT) shares declined in intraday trading Friday after the biopharmaceutical firm announced that the Food and Drug Administration (FDA) wants more data about its gene therapy to treat a rare immune disorder in children before granting approval.

The company reported that the FDA sent it a Complete Response Letter (CRL) asking for “limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review” of the drug, called Kresladi.

Rocket noted that it has met with senior leadership of the FDA’s Center for Biologics Evaluation and Research (CBER) to determine what is needed. Chief Executive Officer (CEO) Dr. Gaurav Shah said he was hopeful of a quick resolution because of “CBER leadership’s direct involvement and commitment to working expeditiously to deliver this therapy to patients.”

FDA Initially Delayed Kresladi Decision Earlier This Year

This is the second time the FDA has delayed a decision on Kresladi, which is designed to treat severe leukocyte adhesion deficiency-I (LAD-I). Rocket had anticipated action by the end of March, but officials extended that by three months, also because of questions about CMC.

Analysts at William Blair noted that while they were disappointed at the issuing of the CRL, they were confident that the company will quickly meet the request and resubmit Kresladi “in a timely manner.” Blair has an "outperform" rating on the stock. 

Shares of Rocket Pharmaceuticals were down 1.3% to $21.15 as of 12:30 p.m. ET Friday, and have lost nearly 30% of their value year-to-date.

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  1. Rocket Pharmaceuticals. "Rocket Pharmaceuticals Provides Regulatory Update on KRESLADI™ (marnetegragene autotemcel; marne-cel)."

  2. Rocket Pharmaceuticals. "Rocket Pharmaceuticals Announces Update on FDA Review Timeline of KRESLADI™ (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)."

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