Drugs

Reviewers Ask Tough Luxturna Questions

FDA medical reviewers are posing tough questions for members of the Cellular, Tissue, and Gene Therapies Advisory Committee when they meet 10/12 to consider a BLA submitted by Spark Therapeutics for Luxturna (voretigene neparvovec) for treating patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy. A briefing document released in advance of the meeting says that while the gene therapy met the main criteria for efficacy, questions about its use remain.

In the key study, the reviewers say, patients had a significant two point or more improvement in light levels in both eyes, but checking visual acuity showed that patients failed to demonstrate a significant improvement in that secondary endpoint.

Questions posed for committee members seek their opinion on:

the clinical meaningfulness of a two-light level improvement;
the optimal disease stage to treat patients with bilallelic RPE65 mutation-associated retinal dystrophy, especially the stage of clinical presentation at which therapy benefits outweigh risks, a reasonable minimal age for treatment, and how data from subjects with significant vision loss can be extrapolated to patients with earlier stages of disease with or without measurable vision loss before treatment; and
the potential benefits and risks of repeat administration of the drug into one eye and what, if any, additional data are required to support such administration.

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