Diacomit® (stiripentol) has been approved by the US FDA for the treatment of seizures associated with Dravet syndrome (DS) in patients 2 years of age and older taking clobazam. The approval came on August 20, 2018.
Before this final approval, Diacomit® was granted orphan drug designation by the FDA back in 2008. An orphan drug designation is granted to a limited number of medicines that treat rare diseases or conditions upon request of a sponsor.
The disease that Diacomit® has been approved to treat is called Dravet syndrome or severe myoclonic epilepsy in infancy (SMEI). This is a rare type of early onset epileptic syndrome that is thought to affect anywhere between 2,000 to 8,000 people in the US.
Diacomit® and its clinical trials
The approval from the FDA was based on two clinical trials called STICLO France and STICLO Italy. The two trials were similar in terms of disease characteristics and prior treatment of patients. Also, for both of the trials, the primary endpoint was the responder rate. The responder rate for these two trials was defined as a patient who experienced a greater than 50% decrease in frequency of seizures over a 30-days period.
The STICLO France trial showed that for patients receiving Diacomit®, the responder rate was 71% compared to 5% for placebo.
As for the STICLO Italy trial, the results showed a responder rate of 67% and 9.1% for placebo.
Diacomit® was also granted orphan drug designation from the European Medicines Agency (EMA) back in December, 2001. However, the Dravet syndrome medicine was fully approved by the EMA in January, 2014. In the EU, it is approved for use in conjunction with valproate and clobazam as adjunctive therapy of refractory generalized tonic-clonic seizures in patients with Dravet syndrome (SMEI) whose seizures are not adequately controlled with clobazam and valproate.
Diacomit® is furthermore approved in Canada and Japan.
Diacomit® is a registered trademark of Biocodex