Guidelines for Pediatric Migraine Clinical Trial Design Released, FDA Grants AMT-130 Fast Track Designation for Huntington Disease, ZX008 Receives RFL for Dravet Syndrome


Neurology News Network for the week of April 13, 2019.

This week, Neurology News Network covered the new guidelines released by the International Headache Society for preventive treatment of migraine in children and adolescents, fast track designation granted by the FDA to uniQure's AMT-130 for Huntington disease, and the FDA issuance of a refusal to file letter to Zogenix regarding its new drug application for ZX008 for treatment of seizures associated with Dravet syndrome. (Transcript below)


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

The International Headache Society has released new guidelines for the preventive treatment of migraine in children and adolescents, focusing on issuing a number of recommendations to address what it determined to be the major challenges with clinical trials in this population.

T the strategies included sought to provide a modern, uniform, and evidence-based approach to the design, conduct, and reporting of well-controlled clinical trials. Ultimately, they wrote, “these guidelines should be consulted and used in designing and conducting clinical trials of preventive treatments in children and adolescents with migraine.”

The FDA has granted fast track designation to uniQure's AMT-130, a gene therapy candidate for the treatment of Huntington disease.

The drug is comprised of a recombinant AAV5 vector and carries a DNA cassette encoding a microRNA that non-selectively lowers, or knocks down, human huntingtin protein in Huntington disease, and targets the exon1 protein fragment. Matt Kapusta, the chief executive officer at uniQure, said in a statement that the designation underscores the high unmet medical need for patients suffering from Huntington disease.

The FDA has issued Zogenix a refusal to file letter regarding its new drug application for ZX008 (Fintepla) for treatment of seizures associated with Dravet syndrome.

While under preliminary review, the FDA determined that the new drug application was not sufficiently complete to permit a substantive review. The agency cited 2 reasons for the decision: the first, being that certain nonclinical studies were not submitted to allow assessment of the chronic administration of fenfluramine; second, the application contained an incorrect version of a clinical dataset that prevented the completion of the review process necessary to support the new drug application filing.

The FDA did not request or recommend additional clinical efficacy or safety studies.

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