FDA Rejects Duchenne Muscular Dystrophy Drug Golodirsen, Pfizer Recalls Acute Migraine Treatment Relpax, Alzheimer Disease Update


Neurology News Network for the week ending August 24, 2019.

This week, Neurology News Network covered the complete response letter to Sarepta Therapeutics for its NDA for golodirsen to treat Duchenne muscular dystrophy, Pfizer's recall of its acute migraine treatment Relpax, and the second episode of the Mind Moments podcast on the amyloid hypothesis and future of Alzheimer disease (transcript below).


Welcome to Neurology News Network. I’m Jenna Payesko. Let’s get into the news from this week.

The FDA has issued a complete response letter to Sarepta Therapeutics for its NDA for golodirsen, previously known as SRP-4053, to treat Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 53 skipping. The rejection was based on 2 concerns: the risk of infections related to intravenous infusion ports, and renal toxicity seen in preclinical models and observed following administration of other antisense oligonucleotides.

Doug Ingram, president and CEO of Sarepta, said in a statement that Sarepta was “very surprised to have received the complete response letter.” Sarepta is immediately requesting a meeting with the FDA to determine next steps.

Pfizer is voluntarily recalling its acute migraine treatment Relpax. The recall includes 2 lots of 40 mg tablets, which are indicated for acute treatment of migraine with or without aura in adults. The recall is due to possible in-house microbiological contamination.

Those with an existing inventory of the lots that are being recalled should stop use and distribution and quarantine immediately. Any adverse reactions or problems with Relpax should be reported to the FDA’s MedWatch Adverse Event Reporting program.

The latest episode of the NeurologyLive Mind Moments podcast is now live! Episode 2 title "Alzheimer Disease Update: Is Amyloid Really Dead?” features an interview with Dr. Reisa Sperling, the Director of the Center for Alzheimer Research and Treatment at Brigham and Women’s Hospital and Massachusetts General Hospital, and professor of Neurology at Harvard Medical School. Sperling shares her thoughts on the amyloid hypothesis and discusses the future of Alzheimer disease research focused on early detection and intervention.

For more direct access to expert insight, head to neurologylive.com. This has been Neurology News Network. Thanks for watching.

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