Rocket's LAD-I gene therapy Kresladi faces FDA setback over manufacturing
28 Jun 2024
Gene TherapyClinical Result
Following a prolonged review period, the FDA issued a complete response letter (CRL) for Rocket Pharmaceuticals' Kresladi (marnetegragene autotemcel), a lentiviral vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I).
In a regulatory update, the company said the FDA's decision hinges on a request for additional chemistry, manufacturing and controls (CMC) information to complete its review. This latest hurdle comes after the agency had extended its review period for Kresladi by three months back in February, citing the need to assess "clarifying" information about the company's CMC process.
Despite the setback, CEO Gaurav Shah remains optimistic, with Rocket having already met with FDA senior leaders from Center for Biologics Evaluation and Research (CBER) to align on the "limited scope of additional CMC information" needed to support Kresladi's approval.
Investor sentiment seemed to reflect Shah’s optimism. While Rocket’s shares were down 13% during midmorning trading, they rallied in the afternoon to close essentially flat on Friday.
"It is reassuring to have the FDA as a close collaborator who understands the high unmet medical need, clear clinical benefit and importance of timely patient access. CBER leadership's direct involvement and commitment to working expeditiously to deliver this therapy to patients gives us great hope on behalf of the primary immunodeficiency community," Shah added.
Results from a Phase I/II study of the gene therapy demonstrated 100% overall survival at 12 months post-infusion for all nine paediatric LAD-I patients, with follow-up ranging from 18 to 42 months. The therapy also showed significant reductions in infection rates compared with pre-treatment history, as well as improvements in LAD-I-related skin lesions and wound repair capabilities.
The regulatory hurdle for Rocket comes in contrast to recent positive news in the gene therapy space. Earlier in the week, the FDA okayed an expanded label for Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec-rokl), despite mixed clinical data. CBER director Peter Marks essentially overruled other dissenting members of the agency, raising questions about the FDA's approach to gene therapy approvals.
See – Physician Views Results: Expanded approval of Sarepta's Elevidys will drive further adoption of gene therapy.
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Indications
Targets
-Hot reports
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.