Drugs

MyoKardia Gets Breakthrough Status for Mavacamten

FDA has granted MyoKardia a breakthrough therapy designation for mavacamten, an oral, allosteric modulator of cardiac myosin for treating symptomatic, obstructive hypertrophic cardiomyopathy. In May, MyoKardia announced topline results from the pivotal Phase 3 EXPLORER clinical trial that showed mavacamten was well tolerated and has a safety profile comparable to placebo, the company says. The data “demonstrated a robust treatment effect, with patients experiencing clinically meaningful responses to treatment, including reductions in symptoms, improvements in cardiac function and reduction or elimination of the obstruction of the left ventricle,” it says. MyoKardia is currently preparing an NDA that will be submitted in the first quarter of 2021.

Hypertrophic cardiomyopathy is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to debilitating symptoms and cardiac dysfunction, according to the company. The most frequent cause is mutations in the heart muscle proteins of the sarcomere.

Mavacamten works by “reducing cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance,” MyoKardia says. “In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility and increased diastolic compliance.”

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