Patricia Inácio, PhD, science writer —

Encoded Therapeutics has raised $135 million to support the first human clinical trials for ETX101, its gene therapy for SCN1A-positive Dravet syndrome patients. The funding also will be used for a natural history study to better understand the progression of the disease in people carrying SCN1A mutations, which are…

Epidiolex (cannabidiol), as an add on-therapy administered at either a low or high dose, leads to similar clinically relevant reductions in the frequency of convulsive seizures in children with treatment-resistant Dravet syndrome, Importantly, a lower Epidiolex dose shows a better safety and tolerability profile when compared to the higher dose.

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

The U.S. Food and Drug Administration (FDA) extended by three months the review period for Zogenix’s New Drug Application (NDA) for Fintepla (ZX008), an investigational anti-seizure therapy for patients with Dravet syndrome. The agency now expects to provide a decision by June 25 on whether…

Editor’s note: This story has been updated to add that Zogenix resubmitted a new drug application for Fintepla to the FDA in late September.  Treatment with Fintepla (ZX008) can provide a clinically meaningful and profound reduction in the frequency of seizures in Dravet syndrome patients, according to…

Neucyte and Trillium Therapeutics have established a license agreement to develop a new therapy for drug-resistant epilepsies, including Dravet syndrome and related disorders. The agent, whose name is still undisclosed, has shown promising results during studies carried out by the National Institute of Neurological Disorders and…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation — allowing various development incentives — to Stoke Therapeutics’ STK-001 for the treatment of Dravet syndrome. The company now intends to test STK-001 in a Phase 1/2 clinical study in the first half of…

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), is recommending approval of GW Pharmaceutical’s oral cannabidiol solution as an add-on to clobazam (Onfi and Sympazan) for treating seizures in children and adults, ages 2 and older, with Dravet syndrome or  Lennox-Gastaut syndrome. The…

Encoded Therapeutics will further develop its lead precision gene therapy platform for Dravet syndrome and other severe genetic disorders after a financing round brought in $104 million from several investors. The company’s goal is to receive approval and market this platform, which aims to overcome key limitations of…

Zogenix has announced it will resubmit a New Drug Application (NDA) for Fintepla (ZX008), its investigational anti-seizure therapy for patients with Dravet syndrome, likely in the third quarter of this year. The U.S. Food and Drug Administration (FDA) refused the first NDA for Fintepla…