Drugs

FDA Cites GMPs in Abeona Inspection

FDA has completed a pre-license inspection of Abeona’s Cleveland, OH manufacturing facility concerning the company’s BLA for pz-cel (prademagene zamikeracel) for treating recessive dystrophic epidermolysis bullosa (RDEB). The firm was issued a Form 483 that cited observations related to process controls.

“During the inspection, the FDA reviewed the facilities, systems, and processes at the Cleveland site,” the company says. “The FDA also observed the manufacturing process for pz-cel, as well as performance of in-process and release assays,” it says, adding that the two-week inspection, which was conducted by five FDA inspectors, concluded on 3/1. On 3/15, Abeona says it submitted a response to Form 483, “outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.”

Additionally, Abeona says the agency has completed the clinical study site inspections at Stanford University School of Medicine in Palo Alto, CA and University of Massachusets Medical School in Worcester, MA, both of which enrolled subjects in the pivotal Phase 3 VIITAL study. Both of the clinical study site inspections ended with no Form 483 observations noted, it says.

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.”

The user fee review action target date in 5/25.

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

Read more

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