Drugs

Vertex Expanded Use for Cystic Fibrosis Drug

FDA has approved an expanded use for Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to include use in children ages two through five years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation, making it the first therapy approved to treat the underlying cause of CF in this population. Approval is based on data from a Phase 3 open-label safety study in 60 patients that showed treatment with Orkambi was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients ages six years and older, according to the company. Improvements in sweat chloride, a secondary endpoint, were observed at week 24, it says.

Orkambi was originally approved in 2015 to treat cystic fibrosis patients age 12 and older who have the F508del mutation that causes the production of an abnormal protein that disrupts how water and chloride are transported in the body. An FDA statement at the time said that having two copies of this mutation—one inherited from each patient—is the leading cause of cystic fibrosis.

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