Drugs

Phase 3 Study on Disc Medicine’s Bitopertin

FDA has agreed on a Phase 3 study (APOLLO) design for Disc Medicine’s bitopertin and its use in treating erythropoietic protoporphyria (EPP), a rare and debilitating condition. The study design was recently discussed during an end-of-Phase 2 meeting that provided a “clear path forward for bitopertin,” the company says. “The agency’s agreement on our study design and the potential for an accelerated approval pathway reflect the high unmet need in EPP and the promise of our data.”

Bitopertin could be eligible for the accelerated approval program based on PPIX reduction as a surrogate endpoint, Disc Medicine says. Accumulation of PPIX in human porphyrias can cause skin photosensitivity. The accelerated pathway would allow Disc to potentially submit an NDA using existing data while conducting APOLLO as a confirmatory trial, it says, adding that the study’s primary endpoint would be average monthly total time in sunlight without pain during the last month following six months of treatment.

EPP and its subtype, X-linked protoporphyria, are rare genetic disorders characterized by extreme sensitivity to sunlight, leading to painful and potentially disfiguring skin reactions.

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