27. Diacomit

blue illustration of neurons
Dravet syndrome is a rare pediatric genetic epilepsy syndrome that affects 2,000 to 8,000 patients in the U.S. (Colin Behrens)

Active ingredient: stiripentol
Disease: Dravet syndrome for patients taking clobazam
Peak sales estimate: n/a
Approved: Aug. 20
Company: BioCodex

The scoop: The anticonvulsant approved to treat seizures in Dravet syndrome patients older than age two will launch in the U.S. January 2019. The Biocodex-developed drug is already approved in Europe, Japan and Canada.

Diacomit is an add-on therapy recommended in the U.S. as a complement to Lundbeck’s Onfi, and in Europe as a complement to both Onfi and AbbVie’s Depacon. Diacomit works in two ways. The first is by inhibiting brain cell activity by binding to GABA receptos and blocking neurons and the second by inhibiting a specific enzyme called cytochrome P450 that can degrade pharma agents such as Onfi.

Dravet syndrome is a rare pediatric genetic epilepsy syndrome that affects 2,000 to 8,000 patients in the U.S. The seizures can be resistant to typical treatments with most patient requiring two or more seizure medications for control. Some anticonvulsants, called sodium channel blockers, can actually making the seizures worse for Dravet patients. The outlook for Diacomit is positive with U.S. physicians who treat epilepsy already commending the approval of the long-time European standard they’ve imported under compassionate use cases for families in the U.S.— Beth Snyder Bulik

27. Diacomit

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