果冻影院

Through the application of first principles to drug design, a novel orphan drug鈥痟as been created for treating narcolepsy, excessive daytime sleepiness (EDS)鈥痑nd Prader-Willi Syndrome.

Narcolepsy is a disabling, long-term neurological disease that affects 1 in 2,000 individuals (approximately 30,000 people in the UK鈥痑nd 165,000 people鈥痠n the USA).

It significantly decreases the quality of life of the affected individuals, with two-thirds of patients being unable to work. Prior to the commercial release of pitolisant, there was an unmet need for an effective, safe, and tolerable treatment regime in the management of narcolepsy.听听

Professor Robin Ganellin (果冻影院 Department of Chemistry) had been researching with biologists since 1964, when he worked in industry, focusing on histamine receptors. Upon joining 果冻影院, Professor Ganellin started collaborating with French neuroscientist Professor Jean-Charles Schwartz. His research group in Paris identified histamine H3 receptors that controlled histamine transmission and demonstrated that histamine was a neurotransmitter that probably led to wakefulness.

Professor Ganellin鈥檚 research group synthesized novel chemical compounds that were tested in Paris. After many years without discovering a drug, he took a different, first principles听approach to generate a type of chemical structure that led to Wakix鈩�.听

This discovery led to the development and commercialisation of pitolisant (Wakix鈩�) by Bioprojet in Europe听and Harmony Biosciences in the USA. Pitolisant, the first in its class, has changed the way narcolepsy (with or without cataplexy) is managed and dramatically improved the quality of life for those suffering from the condition.听听

Production of Pitolisant听

Pitolisant鈥�(Wakix鈩�) is the first - and so far, only - medicament to be marketed that acts on the histamine H3 receptor. This leads to increased histamine transmission in the brain, enhancing wakefulness and alertness and decreasing听cataplexies in patients with narcolepsy.

A patient reported:鈥�鈥淲akix鈩⑩�痠s the most wonderful drug I鈥檝e known over my 50 years suffering narcolepsy. If I鈥檇 been able to have it 30 years ago my life would have been so much easier.鈥�

Pitolisant represented the first new therapy in over a decade for the treatment of鈥痭arcolepsy. After being granted marketing authorisation鈥痓y鈥痶he European Medicines Agency's Committee for Medicinal Products in 2016, pitolisant received NHS appraisal by the National Institute for Clinical Excellence (NICE) and is deemed to be鈥�42% more鈥痗ost-effective鈥痜or the NHS.听

In鈥疉ugust 2019,鈥痶he鈥疐ood and Drug Administration鈥�(FDA) approved鈥痯itolisant鈥�(trade name鈥疻akix鈩�, Harmony Biosciences)鈥痜or use in鈥痶he USA to treat EDS in adult patients with narcolepsy. As of June 30, 2020,鈥痮ver 1,750 unique health care providers have prescribed Wakix鈩� since it became available in November 2019, representing 2,700 unique patients.听听

As direct consequence of鈥痶he licensing agreement between Bioprojet and Harmony Biosciences, Harmony Biosciences raised $270 million in equity funding from 29 investors鈥痑nd鈥痵ecured a $200 million debt facility to provide additional working capital to fuel the company鈥檚 continued growth.鈥� Since its launch in November 2019, the drug has accounted for net sales of $57.8 million for Harmony Biosciences.听

Links

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• 果冻影院 Department of Chemistry
• 果冻影院 Faculty of Maths & Physical Sciences听
• 果冻影院 Faculty of Maths & Physical Sciences听REF 21

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