Biologics

FDA Grants Accelerated Approval to Rocket’s Gene Therapy

FDA has granted Rocket Pharmaceuticals accelerated approval for its gene therapy Kresladi (marnetegragene autotemcel) for children with severe Leukocyte Adhesion Deficiency-I (LAD-1), marking the first approved treatment targeting the underlying genetic cause of the disease. The therapy’s approval covers pediatric patients with biallelic mutations in the ITGB2 gene who lack a matched sibling donor for stem cell transplant — a group with few viable treatment options and high early mortality risk.

Kresladi is described as an autologous hematopoietic stem cell gene therapy designed to restore expression of CD18, a key protein required for immune cells to migrate to sites of infection. Infants with severe LAD-I suffer from recurrent, life-threatening bacterial and fungal infections that often require repeated hospitalizations.

The approval is based on biomarker data showing increased expression of CD18 and CD11a on neutrophils, the company says. Rocket will need to confirm clinical benefit through long-term follow-up and a post-marketing registry.

Alongside the approval, FDA awarded Rocket a Rare Pediatric Disease Priority Review Voucher — a potentially lucrative asset that companies often sell to other drugmakers to expedite future reviews.

The BLA resubmission was filed in October and addressed 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.

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