Drugs

Harmony Bio Granted Orphan Status for Pitolisant

FDA has granted Harmony Biosciences Holdings an orphan drug designation for pitolisant and its use in treating Prader-Willi syndrome (PWS). The company says about 20,000 people in the U.S. have PWS, and the majority experience behavioral symptoms and more than half suffer from excessive daytime sleepiness.

The orphan designation follows a positive end-of-phase 2 meeting with the agency, according to the company. Harmony’s Phase 2 pitolisant study with individuals with PWS showed overall improvement in daytime sleepiness on the ESS-CHAD Parent/Caregiver Version by 3.7 to 5.5 points, it says.

Harmony is planning a Phase 3 registrational study called TEMPO that is expected to start before April. The randomized, double-blind, placebo-controlled trial will evaluate the drug in patients six years and older with PWS.

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

Read more

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