Drugs

PTC Therapeutics’ Friedreich’s Ataxia Drug Rejected

FDA has issued PTC Therapeutics a complete response letter (CRL) on its NDA for vatiquinone and its use for treating Friedreich’s ataxia, calling for additional evidence of the drug’s effectiveness. The CRL said PTC’s submission did not demonstrate “substantial evidence of efficacy” and that another well-controlled clinical trial would be required before resubmitting it, according to the company.

“We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich's ataxia,” the company says in a release. “We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL.”

Vatiquinone is a first-in-class small molecule designed to inhibit 15-Lipoxygenase, an enzyme linked to oxidative stress and mitochondrial dysfunction — processes that play a key role in Friedreich’s ataxia, PTC says. The drug has been evaluated in several clinical studies, including in pediatric patients, and has shown potential benefits in reducing mortality risk and improving neuromuscular symptoms, according to the company.

PTL says Friedreich’s ataxia is a rare inherited disorder that causes progressive damage to the nervous system and heart. Symptoms often emerge in childhood or adolescence and include loss of coordination, muscle weakness, speech and swallowing difficulties, scoliosis, diabetes, and serious heart conditions. The disease affects about 25,000 people worldwide and currently has no cure.

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