Drugs

Positive Data in Stargardt Disease Pivotal Trial

Belite Bio is reporting positive topline results from the global Phase 3 DRAGON trial of tinlarebant, marking what the company says is the first successful pivotal trial in patients with Stargardt disease Type 1 (STGD1). The eye disease leads to progressive vision loss, usually beginning in childhood or young adulthood, and currently has no approved treatment worldwide.

The trial enrolled 104 patients with STGD1 and it met the primary efficacy endpoint, demonstrating a statistically significant and clinically meaningful 36% reduction in the growth rate of retinal lesions compared with placebo. “Not only was tinlarebant shown to be efficacious in slowing retinal degeneration, but this is also the first time that an oral treatment was able to demonstrate a clinically meaningful outcome in retinal degenerative disease,” the company says. “With this data, we are advancing our regulatory interactions globally and moving closer to delivering the first approved treatment for people living with Stargardt disease.”

As expected, the company says the overall change in visual acuity was minimal over the period of 24 months in both study groups, consistent with natural history data. The safety profile remains consistent with what the company previously reported, and tinlarebant was well tolerated with only four treatment-related discontinuations.

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