Drugs

SpringWorks Filing Mirdametinib NDA in 2024

SpringWorks Therapeutics says it will file an NDA in the first half of 2024 for mirdametinib, an investigational MEK inhibitor, indicated for use in pediatric and adult patients with neurofibromatosis Type 1-associated plexiform neurofibromas. The company says its move follows just-announced “positive topline results” from the pivotal Phase 2b ReNeu trial.

The ReNeu trial enrolled 114 pediatric and adult patients across 50 sites in the U.S, and the primary endpoint was a confirmed objective response rate, which was defined as greater than or equal to a 20% reduction in target tumor volume as measured by MRI and assessed by a blinded independent review, according to the company. The recent data showed that 52% (29/56) of pediatric patients and 41% (24/58) of adult patients had objective responses within the 24-cycle, 28-day treatment period. “An additional pediatric patient and two additional adult patients achieved confirmed objective responses after Cycle 24 in the long-term follow-up phase of the trial, where patients continue to receive mirdametinib treatment. Median best percent change from baseline in target tumor volume was -42% and -41% in the pediatric and adult cohort, respectively.”

During the study, the most frequently reported adverse events were rash, diarrhea, and vomiting in the pediatric cohort and rash, diarrhea, and nausea in the adult cohort, SpringWorks says. “Twenty-five percent of pediatric patients and 16% of adult patients experienced a Grade 3 or higher treatment-related AE,” it says. “Additional data are expected to be presented at an upcoming medical conference in the first half of 2024 and to be submitted for publication in a peer-reviewed journal.”

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