Rocket Pharmaceuticals’ Kresladi approval faces another delay

USA —Rocket Pharmaceuticals, a biotech company based in New Jersey, has announced a delay in Kresladi’s approval due to a recent FDA communication.

The FDA issued a Complete Response Letter (CRL) requesting additional information on the manufacturing process.

Kresladi, also known as marnetegragene autotemcel or marne-cel, is a gene therapy for severe leukocyte adhesion deficiency-I (LAD-I), a rare and fatal childhood immune disorder.

 It utilizes genetically modified patient stem cells to deliver a functional copy of the ITGB2 gene.

The FDA initially targeted a decision by March 2024. However, in February, they extended the review to June to assess additional Chemistry, Manufacturing, and Controls (CMC) information provided by Rocket.

According to Rocket, the new CRL seeks “limited additional CMC information” to finalize the review.

The company has already met with FDA’s Center for Biologics Evaluation and Research (CBER) to determine the specific requirements for approval.

Commenting on the new development, Rocket’s CEO, Gaurav Shah, M.D., reassured that they are collaborating closely with the FDA, acknowledging the high unmet medical need, clear clinical benefit, and importance of timely patient access.

 Shah further expressed optimism, stating that CBER leadership’s direct involvement and commitment to working expeditiously to deliver this therapy to patients gives them great hope on behalf of the primary immunodeficiency community.

Leukocyte adhesion deficiency-I is a rare genetic immune disorder that predisposes patients to recurrent infections and is almost always fatal in childhood without an allogeneic hematopoietic stem cell transplant.

Rocket’s approval application for Kresladi was based on a phase 1/2 study of nine patients, all of whom were alive 12 months after their infusions.

 This delay is a setback for Rocket, whose stock price fell over 13% to US$18.51 premarket following the isuarance of the CRL.

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