Biologics

FDA Accepts Rocket’s Gene Therapy BLA Resubmission

FDA has accepted for review a Rocket Pharmaceuticals BLA resubmission for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector–based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). The agency has set a 3/28/2026 user fee review action target date.

The resubmission addresses issues raised in a 6/2024 complete response letter (CRL), which the company said at the time requested “limited additional” chemistry, manufacturing, and controls (CMC) information. The letter caught Rocket by surprise and followed an earlier extension of the review period from March to June 2024, also due to the need for more CMC information, according to the company.

Severe LAD-I is an ultra-rare, autosomal recessive immune disorder caused by mutations in the ITGB2 gene, resulting in defective leukocyte adhesion, according to the company. It says children with severe LAD-I are highly susceptible to recurrent, life-threatening infections and often do not survive beyond early childhood without a bone marrow transplant.

The original BLA is supported by Phase 1-2 trial data, which showed 100% overall survival at 12 months post-infusion and across the full follow-up period for all enrolled patients, according to Rocket. All primary and secondary endpoints were met, with no treatment-related serious adverse events reported, it says, adding that patients also experienced substantial reductions in serious infections, improvements in skin lesions, and restored wound-healing capabilities.

Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher if Kresladi is approved.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome