Drugs

Abeona Therapeutics, FDA Agree on Cell Therapy Endpoints

Abeona Therapeutics says it has completed a Type B meeting with FDA on co-primary endpoints for its planned pivotal Phase 3 VIITAL study of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB), which 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. Those with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen to anchor the skin’s dermal and epidermal layers, according to the company. EB-101 is described as an autologous, gene-corrected cell therapy.

In the trial, Abeona says it will enroll between 10 to 15 patients with RDEB with about 35 large chronic wound sites treated in total. The co-primary endpoints of the study are: (1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 as determined by an investigator assessment; and (2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24.

Earlier Phase 1/2a clinical trial data showed wound healing of 50% or greater following EB-101 treatment in patients, the company says. RDEB was also associated with no pain at treated sites at three, four and five years post-treatment, compared with the presence of pain in 53% of wound sites at baseline, it adds.

EB-101 is described as an autologous, gene-corrected cell therapy.

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