Narcolepsy is a disease that causes prolongation of extreme daytime sleepiness which can leads to muscle weakness and trouble sleeping at night. The complication associated with the disease are cataplexy, hallucinations and sleep paralysis. Due to these complication, the person is at increased risk of developing mental disorders such as schizophrenia. The environmental factors such as toxins that contains heavy metals, pesticides and weed killers and environmental tobacco smoke are thought to be the cause that narcolepsy therapeutic pipeline is expanding. Researchers have found that the disease can occur due to genetic abnormality, that can transfer from one generation to the next generation. There are various novel gene therapies which are under development for the treatment of Narcolepsy. Increasing number of road accidents causing brain injury, autoimmune disorders and fungal or bacterial infections are some of the key factors responsible for the growth of narcolepsy therapeutic pipeline.
In July 2016, Flamel Ireland Limited started a Phase III, randomized, double-blind, placebo controlled, two arm multi-center study to assess the efficacy and safety of a once nightly formulation of Sodium Oxybate extended-release oral suspension (FT218) for the treatment of excessive daytime sleepiness and cataplexy in subjects with narcolepsy. The trial is expected to be completed in July 2017. In June 2016, Jazz Pharmaceuticals plc started a Phase II, randomized, double-blind, placebo-controlled, crossover on-road driving study assessing the effect of JZP-110 on driving performance in subjects with excessive sleepiness due to narcolepsy. The trial was expected to be completed in February 2017. In September 2015, Balance Therapeutics Inc. started a Phase II, randomized, placebo-controlled, double-blind, fixed-dose, multiple cohort, multiple crossover, dose-finding study of oral BTD-001 in adults with idiopathic hypersomnia (IH) or narcolepsy type 2. The trial is expected to be completed in January 2018. In January 2017, Jazz Pharmaceuticals plc started a Phase III, double-blind, placebo-controlled, randomized-withdrawal, multicentre study of the efficacy and safety of JZP-258 in subjects with narcolepsy with cataplexy. The trial is expected to be completed in November 2018.
In Feb 2013, Jazz Pharmaceuticals plc signed a collaborative agreement with Concert Pharmaceuticals, Inc. for the development and commercialization of sodium oxybate (D-SXB) compounds, including C-10323. Under the terms of the agreement, Jazz Pharmaceuticals plc was granted worldwide commercial rights to C-10323, as well as principal responsibility for ongoing development activities. Concert Pharmaceuticals, Inc. received an upfront payment and was eligible to receive additional milestone payments as well as tiered royalties based on potential worldwide sales of any D-SXB products. Through Deuterated chemical entity (DCE) platform, Concert Pharmaceuticals, Inc. synthesized various deuterium-containing analogs of sodium oxybate. Among them, C-10323 proved to be efficacious in Pre-Clinical studies, and it has prolonged pharmacokinetic profile due to its specific deuterium modification pattern. In March 2017, Reset Therapeutics Inc. signed a collaborative agreement with a subsidiary of Alkermes plc for the discovery, development and commercialisation of drugs which could act as orexin receptor modulators. The technology involved in the development of these compounds was Reset’s drug discovery platform. Under the terms of the agreement, Reset Therapeutics Inc. agreed to grant an approval license of orexin modulators, to receive an upfront fee for development and regulatory and sales-based milestone payments, as well as royalties on any future product sales.
Some of the companies having a pipeline of narcolepsy therapeutics include, Flamel Ireland Limited, Jazz Pharmaceuticals plc, Balance Therapeutics Inc., Theranexus SAS, UCB Pharma SA, Teva Pharmaceutical Industries Ltd., Pfizer Inc., Alza Corporation, Concert Pharmaceuticals Inc, Nivalis Therapeutics, Inc., Merck Sharp & Dohme Corp., Insmed Incorporated, Forest Laboratories Ltd., Vertex Pharmaceuticals Incorporated, Novartis AG, AlgiPharma AS, Spyryx Biosciences, Inc., Gilead Sciences Inc., ProMetic BioSciences Inc., Anthera Pharmaceuticals, etc.