FDA Policy/General

Alnylam Gets Additional Approval for Amvuttra

FDA has approved an Alnylam Pharmaceuticals supplemental NDA for Amvuttra (vutrisiran), an RNAi therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). Vutrisiran was previously approved for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. “By rapidly knocking down both mutant and wild‑type transthyretin (TTR), vutrisiran addresses the underlying cause of transthyretin amyloidosis (ATTR),” the company says.

The submission was based on data from HELIOS-B, a placebo-controlled multicenter global Phase 3 study in patients with ATTR-CM. “The study demonstrated favorable effects of vutrisiran on outcomes of death and cardiovascular events, functional capacity and quality of life in patients with ATTR-CM,” the company says.

Alnylam says vutrisiran’s safety and tolerability are well-established, as demonstrated in the positive HELIOS-A clinical trial in hATTR-PN which resulted in FDA approval in 2022. In the study, the most common adverse reactions in patients treated with vutrisiran were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreases (7%). No new safety concerns were identified in the HELIOS-B clinical trial of patients with ATTR-CM, the company adds.

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