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Fenfluramine

Generic Name
Fenfluramine
Brand Names
Fintepla
Drug Type
Small Molecule
Chemical Formula
C12H16F3N
CAS Number
458-24-2
Unique Ingredient Identifier
2DS058H2CF
Background

Dravet syndrome is a pediatric encephalopathy that typically manifests within the first year of life following exposure to elevated temperatures. It is characterized by recurrent pharmacoresistant seizures, which increase in frequency and severity with disease progression. Concomitantly with these seizures, patients typically display delayed development and neurocognitive impairment. Fenfluramine is a serotonergic phenethylamine originally used as an appetite suppressant until concerns regarding cardiotoxicity in obese patients lead to its withdrawal from the market in 1997. Through its ability to modulate neurotransmission, fenfluramine has reemerged as an effective therapy against pharmacoresistant seizures, such as those involved in Dravet syndrome.

Fenfluramine was granted initial FDA approval in 1973 prior to its withdrawal; it was granted a new FDA approval on June 25, 2020, for treatment of patients with Dravet syndrome and Lennox-Gastaut syndrome through the restricted FINTEPLA REMS program. It is currently sold under the name FINTEPLA® by Zogenix INC.

Indication

Fenfluramine is indicated for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients aged two years and older.

Associated Conditions
Seizures

CDMO Market Report: Key Regulatory Approvals and Clinical Advances in March-April 2025

• Multiple CDMOs secured significant contract manufacturing opportunities as regulatory bodies approved new indications for established drugs, particularly in oncology and rare diseases. • AstraZeneca's portfolio saw substantial growth with expanded approvals for Imfinzi, Tagrisso, and Lynparza, strengthening partnerships with contract manufacturers including Lonza, Dottikon, and Samsung Biologics. • Contract manufacturers supporting treatments for autoimmune conditions showed strong performance, with Argenx's Vyvgart Hytrulo receiving expanded indications for myasthenia gravis and CIDP.

NHS Approves Fenfluramine for Children with Lennox-Gastaut Syndrome, Offering New Hope for Severe Epilepsy Treatment

• The NHS has approved fenfluramine, the first non-cannabis based treatment for Lennox-Gastaut syndrome (LGS), offering new hope for children aged 2 and above with this severe form of epilepsy. • Clinical trials demonstrated that fenfluramine reduces drop seizure frequency by 26.5% on average, with over 25% of patients experiencing a 50% or greater reduction in seizures. • The treatment will be fast-tracked through NHS England's Innovative Medicines Fund, making it available three months earlier for eligible patients who cannot tolerate existing cannabis-based treatments.

Biogen and Stoke Therapeutics Collaborate to Advance Zorevunersen for Dravet Syndrome

• Biogen and Stoke Therapeutics will collaborate to develop and commercialize zorevunersen for Dravet syndrome outside the U.S., Canada, and Mexico. • Stoke's Phase 3 EMPEROR study of zorevunersen, expected to begin in mid-2025, aims to reduce seizure frequency and improve cognition and behavior in children with Dravet syndrome. • Zorevunersen, an antisense oligonucleotide targeting the SCN1A gene, has shown promising results in earlier trials, including an 87% median reduction in convulsive seizure frequency. • Biogen will provide Stoke with an upfront payment of $165 million, potential milestone payments up to $385 million, and tiered royalties on net sales in Biogen's territory.

UCB Pharma Eyes Further Expansion in South Korea Following Recent Drug Approvals

• UCB Pharma aims to expand its portfolio in South Korea after securing approvals for Bimzelx (bimekizumab) for plaque psoriasis and Zilbrysq (zilucoplan) for myasthenia gravis. • Fintepla (fenfluramine) is under review for Dravet syndrome and Lennox-Gastaut syndrome, potentially receiving expedited approval via the GIFT program. • Rystiggo (rozanolixizumab), a monoclonal antibody for myasthenia gravis, may also see accelerated approval if designated under Korea’s GIFT program. • Bimzelx is poised to compete in the plaque psoriasis market, showing superior efficacy in trials against Humira, Stelara, and Cosentyx.

Stoke Therapeutics Plans Phase 3 Trial of STK-001 for Dravet Syndrome Following Promising Phase 1/2a Data

• Stoke Therapeutics is advancing STK-001, a novel treatment for Dravet syndrome, into a Phase 3 trial in 2024 based on encouraging Phase 1/2a results. • Interim data from the MONARCH and ADMIRAL trials showed STK-001 safely and effectively reduced seizure frequency in children and adolescents with Dravet syndrome. • STK-001 aims to restore normal levels of NaV1.1 protein in nerve cells by increasing its production from the healthy SCN1A gene copy. • Additional data expected in 2023 will inform dose level and frequency for the Phase 3 program, potentially shifting Dravet syndrome treatment from seizure management to comprehensive syndrome management.
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