dm+d

Unassigned

New Medicines

Dravet syndrome

Information

New molecular entity
Takeda
Takeda

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials

Category

Potent inhibitor of cholesterol 24-hydroxylase (CH24H) which modulates the brain NMDA receptor thus improving seizure intensity and frequency. [3]
Dravet syndrome is often associated with a mutation in the SCN1A gene. It affects ~0.0064% of the population. [1,2]
Dravet syndrome
Oral

Lennox-Gastaut syndrome

Information

New molecular entity
Takeda
Takeda

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

Potent inhibitor of cholesterol 24-hydroxylase (CH24H) which modulates the brain NMDA receptor thus improving seizure intensity and frequency
Lennox-Gastaut syndrome is a severe form of epilepsy with a peak onset at 3 to 5 years of age, affecting boys more commonly. Annual incidence in children is estimated to be 2 per 100,000 children [1].
Lennox-Gastaut syndrome
Oral