Drugs

FDA Allows Modified Rating Scale for Omaveloxolone Trial

FDA has told Reata Pharmaceuticals that its modified Friedreich’s Ataxia Rating Scale (mFARS) is an acceptable primary endpoint for Part 2 of the company’s MOXIe trial for omaveloxolone in Friedreich’s ataxia (FA). The rare, degenerative, life-shortening neuro-muscular disorder affects children and adults and involves the loss of strength and coordination usually leading to wheelchair use; diminished vision, hearing and speech; scoliosis; increased risk of diabetes and a life-threatening heart condition. Currently, there are no FDA-approved treatments for FA.

According to the company, FDA’s recent communication indicated that it may consider either accelerated or full approval based on the overall results of the trial and strength of the data. The agency also recommended that it extend the treatment duration for Part 2 of the study and add a straightforward patient-reported or performance-based outcome endpoint to the study.

MOXIe Part 2 is a double-blind, randomized, placebo-controlled, multi-center, international trial designed to evaluate the safety, tolerability, and efficacy of omaveloxolone in patients with FA. It is intended to enroll about 100 FA patients randomized evenly to either 150 mg of omaveloxolone or placebo. The primary endpoint of the trial will be the change from baseline in mFARS of omaveloxolone compared to placebo at 48 weeks.

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