Drugs

Acer Refiles NDA for Urea Disorder Drug

Acer Therapeutics has resubmitted its NDA for ACER-001 (sodium phenylbutyrate) for oral suspension for treating patients with urea cycle disorders (UCDs). The resubmission addresses items raised by the agency in a complete response letter issued last month, which identified the company’s third-party contract packaging manufacturer as not being ready for inspection as the primary driver for the letter.

Acer says it stated in the resubmission that the third-party facility is now ready for inspection. While it says FDA did not cite any other approvability issues in the letter, it did request additional existing nonclinical information which was identified as “not an approvability issue,” as well as labeling and other routine updates to the original NDA. These information updates were also included in the resubmission.

Previously, Acer said its original submission was based on data from two bioequivalence trials in which Acer-001 showed similar relative bioavailability to the reference drug, Ucyclyd Pharma’s Buphenyl (sodium phenylbutyrate).

UCDs are caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream (hyperammonemia), Acer says. “Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits,” 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