FDA Policy/General

Beren’s Infantile Niemann-Pick disease Drug Review Extended

FDA has extended by three months its review timeline of a Beren Therapeutics NDA for adrabetadex for treating infantile-onset Niemann-Pick disease, Type C (NPC). The new user fee review action target date is 11/17.

The extension follows a 3/18 Beren response to an FDA information request that included updates and clarifications to existing data and supporting documentation. The agency classified the response as an NDA major amendment, which under FDA regulations extends the review period by three months.

The NDA was previously granted a priority review, and adrabetadex previously received orphan drug and breakthrough therapy designations.

Niemann-Pick disease, Type C is described as a rare, autosomal-recessive, severe, heterogeneous, neurodegenerative disorder caused by pathogenic variants in the NPC1 or NPC2 genes, leading to impaired intracellular cholesterol trafficking resulting in progressive neurological decline and premature mortality.

The company says clinical ( XXX delete capital C XXX)and nonclinical data demonstrate that adrabetadex directly targets the underlying pathophysiology of NPC by re-establishing intracellular cholesterol trafficking. The most common adverse events reported in clinical trials are hearing impairment (manageable with hearing aids when necessary) and post-dose fatigue and ataxia.

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