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Huntington’s disease (HD) is a neurodegenerative disease inherited in an autosomal dominant manner. It is caused by an expansion of cytosine, adenine, guanine (CAG) repeats within the huntingtin (HTT) gene, which is located on chromosome 4. This pathological expansion of CAG repeats results in the production of a mutant huntingtin protein with an abnormally long polyglutamine […]

Kelly Knupp, AAN 2022: Long-term safety and efficacy of flenfluramine for patients with Lennox-Gastaut syndrome – Phase 3 interim analysis

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Published Online: Apr 28th 2022

The efficacy and safety of fenfluramine, an anti-seizure medication, in patients with Lennox-Gastaut syndrome was assessed in phase 3 and open label extension studies (NCT03355209). Dr Kelly Knupp (Children’s Hospital Colorado, University of Colorado, CO, USA) discusses the findings of these studies and the impact its recent approval will have on clinical practice.

The abstract entitled ‘Interim Analysis of Long-Term Safety and Efficacy of FINTEPLA (fenfluramine) in Patients with Lennox-Gastaut Syndrome’ was presented at the American Academy of Neurology (AAN) Annual Meeting, April 2-7, 2022.

Questions:

  1. Could you tell us a little about fenfluramine and its approved treatment indications? (0:13)
  2. What did the phase 3 Study 1601 teach us about fenfluramine’s efficacy and safety in patients with Lennox-Gastaut syndrome? (1:03)
  3. Which patients were eligible to enter the open label extension study (OLE)? (3:07)
  4. What were the findings from the OLE? (4:06)
  5. Since its recent approval, what do you think will be the impact of fenfluramine on clinical practice? (4:51)

Disclosures: Kelly Knupp is a consultant for epilepsy study consortium, Biocodex, Eisai, Encoded Therapeutics, Jazz pharmaceuticals, Longboard, Stoke Therapeutics, and Zogenix. Kelly Knupp has conducted contracted research for Eisai, Encoded Therapeutics, Stoke Therapeutics, Takada, and Zogenix.

Support:  Interview and filming supported by Touch Medical Media. Interview conducted by Shanice Allen.

Filmed as a highlight of AAN 2022

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