Drugs

Comments on FDA Rare Diseases Draft Guidance

The Biotechnology Innovation Organization (BIO) says an FDA draft guidance on early drug development for rare diseases and the role of pre-IND meetings “provides important information to drug developers regarding pre-IND meetings to support better the consistency, transparency, [and] efficiency of drug development, and the review of rare disease therapies. BIO included line-by-line edits in its comment letter.

Pharmaceutical Research and Manufacturers of America (PhRMA) commends FDA for its “continued acknowledgment of the need for regulatory flexibility for rare diseases, for example, when determining which clinical pharmacology studies are essential to inform the safe and effective use of a drug.” The association says it supports inclusion of modeling and simulation approaches to inform dosing and elements of the clinical trial design. “Model-informed approaches may help improve clinical trial efficiency, increase the probability of regulatory success, and optimize dosing in the absence of dedicated trials,” it says. The association also offers recommendations and requests for additional clarity on issues that would ensure the maximum benefit of the draft guidance to FDA, patients, sponsors, and other stakeholders.

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