Drugs

FDA Lifts Clinical Hold on Intellia’s Phase 3 Trial

FDA has lifted a clinical hold on Intellia Therapeutics’ MAGNITUDE-2 Phase 3 trial evaluating nexiguran ziclumeran, or nex-z, for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), clearing the way for the company to resume patient enrollment and dosing. The gene editing company says FDA removed the hold on the IND application for MAGNITUDE-2 following a review of additional data and agreed-upon study modifications, including enhanced monitoring of liver safety laboratory tests.

The agency placed clinical holds on the MAGNITUDE and MAGNITUDE-2 trials in late October after a patient in the MAGNITUDE Phase 3 trial for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) experienced Grade 4 elevations in liver transaminases along with increased total bilirubin that met protocol-defined pausing criteria, according to the company.

While the hold on MAGNITUDE-2 has been lifted, Intellia says discussions with FDA are continuing regarding the MAGNITUDE Phase 3 trial. The company said it will provide an update once there is alignment on a path forward for that program.

MAGNITUDE-2 is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of a single infusion of nex-z in patients with ATTRv-PN. As part of a protocol amendment agreed to with regulators, Intellia increased the trial’s target enrollment to about 60 patients from approximately 50. Participants are randomized 1:1 to receive either a single 55-mg infusion of nex-z or placebo. The study’s primary endpoints include changes in modified neuropathy impairment score and serum transthyretin (TTR) levels.

Nex-z is described as an in vivo CRISPR/Cas9 gene editing therapy designed to inactivate the TTR gene, which encodes the transthyretin protein that misfolds and accumulates in ATTR amyloidosis.

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