Diacomit: A Breakthrough For Dravet Syndrome Treatment

Introduction

In July of this year, the U.S. Food and Drug Administration (FDA) extended the approval for stiripentol (Diacomit) in the management of Dravet syndrome. This notable expansion permits the use of the medication for patients as young as 6 months, provided they are also on clobazam. Previously, Diacomit was sanctioned in the U.S. for patients aged 2 years and older. It remains the sole treatment specifically endorsed for Dravet syndrome, effectively addressing seizures in children as young as 6 months.

FDA Approval

Stiripentol (Diacomit) received its initial FDA approval for the treatment of Dravet syndrome in patients aged 2 years and older. The recent expansion of its authorization to include children as young as 6 months underscores its growing importance in managing this condition.

Indication

Diacomit is prescribed for the management of seizures linked to Dravet syndrome in patients aged 2 years and older who are simultaneously receiving clobazam. It is essential to emphasize that there is no clinical evidence supporting the use of Diacomit as a standalone treatment for Dravet syndrome.

Mechanism of Action

The mechanism by which Diacomit exerts its anticonvulsant effect in humans is unknown. Possible mechanisms of action include direct effects mediated through the gamma-aminobutyric acid (GABA)A receptor and indirect effects involving inhibition of cytochrome P450 activity with resulting increase in blood levels of clobazam and its active metabolite

Dosage and Administration

The recommended dosage for Diacomit is 50mg/kg/day, administered orally in 2 or 3 divided doses. Special care must be taken when reducing or discontinuing the dosage. Diacomit is available in capsule and powder for oral suspension forms, both of which should be taken with a meal.

Contraindications and Warnings

Diacomit does not have any specific contraindications, but it’s vital to be aware of warnings and precautions, including the monitoring of somnolence, decreased appetite, decreased weight, neutropenia, and thrombocytopenia. Special attention is also required for patients with phenylketonuria (PKU) and the potential for suicidal behavior and ideation.

Drug Interaction

Diacomit has significant drug interactions, notably increasing the plasma concentration of clobazam and its metabolite due to its impact on CYP3A4 and CYP2C19 enzymes, potentially requiring clobazam dosage reduction for managing adverse reactions. 

Furthermore, substrates of CYP2C8, CYP2C19, P-gp, and BCRP may necessitate dosage reduction when co-administered with Diacomit, while substrates of CYP1A2, CYP2B6, and CYP3A4 may require dosage adjustments. 

Strong inducers of CYP1A2, CYP3A4, or CYP2C19 might prompt consideration of a Diacomit dosage increase. These interactions underscore the importance of careful medication management when using Diacomit in patients with Dravet syndrome.

Side Effects

Common adverse reactions to Diacomit include somnolence, decreased appetite, agitation, ataxia, weight loss, hypotonia, nausea, tremor, dysarthria, and insomnia.

Recent Research

A recent study published by Dravet Syndrome News analyzed data from 131 infants with Dravet syndrome in France who were treated with Diacomit (stiripentol) before the age of 2 and were followed for 30 years (1991-2021). The study found that long-term Diacomit treatment was safe and clinically effective at reducing seizures in these patients. Specifically, 67% of patients experienced at least a 50% reduction in prolonged tonic-clonic seizures (TCS), and 71% had a similar reduction in status epilepticus. 

Additionally, 62% of patients saw a cessation of prolonged TCS, and 67% experienced a halt in status epilepticus. Short-term treatment also resulted in a significant reduction in overall TCS frequency, but this effect was not as pronounced with long-term treatment.

Conclusion

The expanded use of Diacomit for younger children with Dravet syndrome is a significant advancement, offering hope to patients and their families. Ongoing research will further improve the management of this condition. Diacomit price in India depends on the product requirement. Request more details by contacting our Patient Support Team at ‎(+91) 93157 05373 or help@sansfro.com.  

Reference

1. https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/206709s000,207223s000lbl.pdf
2. https://dravetfoundation.org/stiripentol-diacomit-history-and-new-expanded-approval/
3. https://www.biospace.com/article/releases/fda-approves-diacomit-stiripentol-for-the-treatment-of-seizures-associated-with-dravet-syndrome-ds-in-patients-2-years-of-age-and-older-taking-clobazam/