Biologics

Abeona Skin Disorder BLA Resubmission Accepted

FDA has accepted for review an Abeona Therapeutics BLA resubmission for prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). The resubmission follows an April complete response letter that requested additional information to satisfy chemistry manufacturing and controls requirements (see earlier story). The agency has set a user fee review action target date of 4/29/2025.

According to the company, the resubmission is based on data from the pivotal Phase 3 VIITAL study (NCT04227106) and a Phase 1/2a study (NCT01263379). “Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction,” it adds.

RDEB is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life, Abeona says. “People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin,” it says. Pz-cel is designed to “incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome,” it adds.

If approved, the company says it may be eligible for a priority review voucher.

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