Drugs

FDA Nixes Accelerated Approval Option for Duchenne Drug

FDA has told Santhera Pharmaceuticals that its planned clinical trial for Raxone (idebenone) in Duchenne muscular dystrophy (DMD) patients will not support an accelerated approval pathway, but it will support, if the results are positive, a traditional NDA approval route. The company had proposed an accelerated approval pathway using its planned SIDEROS trial in DMD patients not taking concomitant glucocorticoids. Santhera had hoped that the trial would provide confirmatory efficacy evidence in these patients while a longer term study would support treating glucocorticoid-using patients under a traditional NDA route.

“From its review of this strategy, the FDA concluded that results from the SIDEROS trial, which is powered to detect a difference in the established surrogate endpoint Forced Vital Capacity percent predicted (FVC%p) in glucocorticoid-using patients, should be provided at the time of filing to support an NDA for the treatment of DMD patients irrespective of their glucocorticoid use status,” the company says. “The protocol of the SIDEROS trial has previously been reviewed by the FDA which confirmed that this trial has the potential, if positive, to provide the necessary efficacy data, along with data from previous trials to support NDA filing in patients with DMD.” Santhera added that enrolment in the SIDEROS trial will start shortly.

SIDEROS is a Phase 3 double-blind, randomized, placebo-controlled trial of Raxone in DMD patients receiving concomitant glucocorticoids. “Patients with declining respiratory function on any stable glucocorticoid treatment scheme irrespective of the underlying dystrophin mutation or ambulatory status will be eligible for participation,” the company says. Study participants will receive either Raxone (900 mg/day; given as 2 tablets 3 times a day with meals) or placebo for 78 weeks (18 months). The primary endpoint of the trial is change from baseline to week 78 in forced vital capacity precent predicted (FVC%p).

Santhera characterizes idebenone as a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) “capable of stimulating mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS) and supplementing cellular energy levels.”

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