Drugs

Italfarmaco Wins Approval for Duchenne Drug

FDA has approved Italfarmaco’s Duvyzat (givinostat), an oral drug for treating Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. “Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD,” an FDA release says. “It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.”

Approval was based on data from a randomized, double-blind, placebo-controlled 18-month Phase 3 study with a primary endpoint of change from baseline to Month 18 using a four stair climb to measure muscle function, the agency says. Patients treated with Duvyzat showed “statistically significant less decline in the time it took to climb four stairs compared to placebo,” it says, adding that mean change from baseline to Month 18 was 1.25 seconds for patients receiving Duvyzat compared to 3.03 seconds for those on placebo.

Regarding safety, the most common side effects in those taking Duvyzat are diarrhea, abdominal pain, a decrease in platelets (increased bleeding potential), nausea/vomiting, an increase in triglycerides, and fever, FDA says. Additionally, Duvyzat’s label includes warnings for health care providers to evaluate patients’ platelet counts and triglycerides before prescribing. “Duvyzat may also cause QTc prolongation, which can increase the risk for irregular heartbeats,” it 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