Drugs

SpringWorks’ Gomekli Approved for Neurofibromatosis

FDA has approved a SpringWorks Therapeutics NDA for Gomekli (mirdametinib), a kinase inhibitor for treating adult and pediatric patients two years of age and older with neurofibromatosis Type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

Approval was based on data from the ReNeu (NCT03962543) multicenter, single-arm trial in 114 patients with symptomatic, inoperable NF1-associated PN causing significant morbidity, an FDA release says. “The major efficacy outcome measure was confirmed overall response rate (ORR), defined as the percentage of patients with complete response (disappearance of the target PN) or partial response (greater than 20% reduction in PN volume).”

Regarding safety, the most common adverse reactions (greater than 25%) in adult patients were rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue, FDA says. The most common Grade 3 or 4 laboratory abnormality was increased creatine phosphokinase.

“Mirdametinib can also cause left ventricular dysfunction and ocular toxicity including retinal vein occlusion, retinal pigment epithelial detachment and blurred vision,” the agency adds. “Mirdametinib should be withheld, dosage reduced or permanently discontinued based on the severity of adverse reactions.”

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