Drugs

FDA OKs Amicus Pompe Disease Combo Drugs

FDA has approved an Amicus Therapeutics NDA for Pombiliti (cipaglucosidase alfa-atga) in combination with Opfolda (miglustat) 65mg capsules for treating adults living with late-onset Pompe disease weighing more than 40 kg and who are not improving on their current enzyme replacement therapy (ERT). Amicus says the rare, life-threatening lysosomal disorder is caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). “Reduced levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscle cells and glycogen buildup causes muscle damage,” it says.

Approval was based on data from the Phase 3 PROPEL study that showed clinically meaningful and positive changes in the key manifestations of this disease for patients taking the drug combination, according to the company. “Particularly impressive are the clinically significant improvements in musculoskeletal and respiratory endpoints for patients switching from ERT standard of care to [the drug combination] AT-GAA,” it says.

Pombiliti is described as a recombinant human GAA enzyme that is designed for increased uptake into muscle cells. “Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen,” Amicus says. “Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.”

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