FDA delays review of Rocket's gene therapy for rare immune disorder

2024-02-14

优先审批临床2期基因疗法临床结果

Rocket Pharmaceuticals will have to wait another three months for an FDA decision on its investigational gene therapy for severe leukocyte adhesion deficiency-I (LAD-I). The US regulator is now expected to determine whether to approve Kresladi (marnetegragene autotemcel) by June 30 to allow more time to review "clarifying" information about the company's chemistry, manufacturing, and controls (CMC) process.

"We remain confident and focused on making this therapy available for patients as quickly as possible," stated CEO Gaurav Shah. According to Rocket, the FDA has also confirmed that it does not intend to convene an advisory committee meeting to discuss the filing, which was granted a priority review last October.

The gene therapy, also known as RP-L201, contains patient-derived haematopoietic stem cells modified with a lentiviral vector to deliver a functional ITGB2 gene, which encodes human CD18 receptor to facilitate leukocyte adhesion and ability to move out of blood vessels to fight infection.

In September, Rocket completed a global Phase I/II trial of the treatment in nine paediatric patients with severe LAD-I, according to ClinicalTrials.gov. The company says all primary and secondary endpoints were met with Kresladi demonstrating 100% overall survival at one-year post-infusion, as well as for the entire 12 to 24 months of follow-up.

Compared with pre-treatment history, it said Kresladi also showed "large decreases" in the incidence of significant infections, and there was evidence of resolution of LAD-I-related skin lesions and restoration of wound repair capabilities. The gene therapy was "very well tolerated" in all patients, none of whom suffered serious adverse events related to treatment.

Company shares soared last September announced plans to raise $175 million in an upsized public offering. Around the same time, it initiated a pivotal Phase II to support accelerated approval of its RP-A501 gene therapy for Danon disease, a rare X-linked inherited disorder caused by mutations in the LAMP-2 gene.

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