Drugs

Patient Injured in Intellia’s Phase 3 nex-z Trial Dies

Intellia Therapeutics is reporting that a patient who experienced liver toxicity after being administered the company’s CRISPR-based therapy nexiguran ziclumeran (nex-z) has died. Late last month, FDA issued a clinical hold on Intellia’s MAGNITUDE and MAGNITUDE-2 Phase 3 trials for nex-z after the company disclosed Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed in the MAGNITUDE trial.

The hold follows a 10/24 report about the patient’s liver monitoring levels, the company says, adding that the patient was hospitalized and was receiving medical care. The company says it is consulting with external experts, exploring potential risk mitigation strategies, and engaging with regulatory authorities globally to determine a safe path to resume enrollment.

The MAGNITUDE trial currently has over 650 patients enrolled, and MAGNITUDE-2 has 47 patients enrolled, with more than 450 patients estimated to have received nex-z.

Nex-z, based on CRISPR/Cas9 gene editing, is designed to inactivate the TTR gene to reduce the production of transthyretin protein, according to Intellia. Interim Phase 1 data previously showed consistent, deep, and long-lasting TTR reduction, it says.

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