Drugs

Vertex Plans Mid-year Cystic Fibrosis NDA

Vertex Pharmaceuticals plans to file an NDA by mid-year and use a priority review voucher for its cystic fibrosis (CF) combination drug, once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza triple). The decision to submit the NDA comes as the company is reporting Phase 3 data that show the treatment met all primary and key secondary endpoints in two randomized controlled trials in people with CF ages 12 years and older.

“We are very pleased with today’s results, which demonstrate the vanza triple is non-inferior to [Vertex’s] Trikafta in improving lung function and superior to Trikafta in lowering levels of sweat chloride in people living with CF, setting a new standard for the level of CFTR protein function achievable, and raising the very high bar set by Trikafta,” the company says.

Vertex notes that vanza triple was well tolerated in all three studies, and safety was similar between the vanza triple and Trikafta treatment groups in the SKYLINE 102 and SKYLINE 103 studies. “The safety of the vanza triple in children 6 to 11 years old was similar to the safety in people 12 years of age and older,” 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