Drugs

Retrophin Gains FDA Agreement on Retrophin Phase 3 Trial

Retrophin has reached an agreement with FDA under a Special Protocol Assessment (SPA) for a Phase 3 clinical trial evaluating RE-024, an investigational replacement therapy for treating pantothenate kinase-associated neurodegeneration (PKAN). The SPA provides agency concurrence that the trial’s design can adequately support a future NDA submission. The company says it will begin the the trial before year-end.

The SPA is based on an international, randomized, double-blind, placebo-controlled study evaluating RE-024 in about 82 patients with PKAN aged 6 to 65 years, Retrophin says. The primary endpoint will be the change in score on the Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale, from baseline through 24 weeks of treatment. After completing the 24-week treatment period, all patients will be eligible to receive RE-024 as part of an open-label extension. The PKAN-ADL is a patient-reported outcome scale measuring motor abilities to function in daily living for patients with PKAN.

“Preclinical findings suggest RE-024 has the ability to distribute to the brain and restore CoA levels,” Retrophin says. In a Phase 1 study, RE-024 was found to be safe and well-tolerated in healthy volunteers. In 2015, FDA granted the therapy an orphan drug designation and Fast Track status for treating PKAN.

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