SOURCE: Brabant Pharma

November 25, 2013 08:00 ET

Brabant Pharma Reports Two-Year Follow-Up Data From a 19-year Observational Study Using Low-Dose Fenfluramine for the Treatment of Dravet Syndrome

92% of Dravet Syndrome Patients Were Either Seizure-Free or Had Greatly Reduced Seizure Frequency With an Average Treatment Period of 13 Years 4 Months

Results Presented at the European Pediatric Neurology Society Congress (EPNS)

LONDON, UNITED KINGDOM--(Marketwired - Nov 25, 2013) - Brabant Pharma ("Brabant") today announced that Professor Berten Ceulemans of the University of Antwerp recently presented additional long-term data from a continuing prospective study of 12 children and adults with Dravet Syndrome, who were treated with low-dose fenfluramine for an average of 13 years 4 months. The data were presented in a Special Lecture on Dravet Syndrome that was hosted by Brabant Pharma at the European Pediatric Neurology Society Congress (EPNS).

The results show that during the period 2011 to 2012, 92% of the patients taking low-dose fenfluramine were either seizure-free or had greatly reduced seizure frequency. Two non-responders dropped out of the 19-year observational study and were replaced by two new patients.

Prof. Berten Ceulemans commented, "These data represent a major advance in the treatment of Dravet Syndrome where seizure-free status is the treatment goal which until now has been extremely difficult to achieve. Low-dose fenfluramine has shown success with both long and short-term treatment plus a dramatic improvement in seizure-free status or reduction in the number of seizures."

In May 2012, Professor Ceulemans et al published the results of a long-term observational study (average treatment period of 11 years and 4 months) using low-dose fenfluramine for the treatment of Dravet Syndrome in Epilepsia. The study showed that of the 12 patients treated, three-quarters experienced seizure cessation or had a greater than 75% reduction in seizure frequency. Seven of these patients had remained seizure-free during the previous 12-months. On average, patients were seizure-free for 6 years (range 1 to 19 years).

Side-effects of treatment were mild and transient for the entire 21-year study period with no reports of pulmonary hypertension. Two patients showed sub-clinical evidence of cardiac valve thickening that was judged to be clinically insignificant by cardiologists. Similar findings spontaneously resolved in a third patient.

Rick Stewart, Chairman and CEO of Brabant, commented, "Low-dose fenfluramine for the treatment of Dravet Syndrome has shown great success in making patients seizure-free or substantially reducing the number of seizures experienced. Making these children seizure-free for an average of six years is immensely impressive and we are continuing to successfully treat patients who have been on the drug for over twenty-one years. The average treatment duration is now over thirteen years."

Mr. Stewart continued, "Dravet Syndrome is a rare and catastrophic form of epilepsy with very limited treatment options but with life-threatening consequences for patients. We are very encouraged by these data and expect to advance our clinical strategy with Phase III trials in 2014."

"Extended seizure-free periods, which have not been reported with any previous Dravet Syndrome treatment should provide meaningful and significant improvements in quality of life," commented Dr Anthony Clarke, Chief Scientific Officer of Brabant. "The risk-benefit to patients is strongly in favour of low-dose fenfluramine given the life-threatening nature of the disease from status epilepticus and Sudden Unexpected Death in Epilepsy (SUDEP) plus the progressive brain damage caused by frequent seizure activity," added Dr. Clarke.

Brabant has met with the U.S. Food and Drug Administration (FDA) and European regulatory authorities to discuss the future clinical pathway for its new low-dose formulation of fenfluramine. Brabant expects to commence Phase III clinical trials in the second half of 2014 in the U.S. and Europe.

About Dravet Syndrome:
Dravet Syndrome (also known as Severe Myoclonic Epilepsy of Infancy) is a rare, severe and therapy resistant form of epilepsy most often caused by an identifiable gene defect that causes abnormal functioning of a sodium channel in the brain. Children with Dravet's Syndrome experience severe long lasting fever-related seizures in the first year of life. Other seizures typically arise later, including myoclonus (involuntary muscle spasms) and status epilepticus (prolonged seizures), often resulting in severe cognitive and developmental impairment. Episodes of status epilepticus require immediate emergency care and can be fatal.

About Brabant Pharma: Brabant Pharma is a privately held specialty pharmaceutical company developing a new low-dose formulation of fenfluramine specifically formulated to treat children with Dravet Syndrome.


Contact Information

  • Enquiries:
    Brabant Pharma Limited
    Rick Stewart
    Chief Executive Officer
    +1 (212) 203 0616

    Dr Anthony Clarke
    Chief Scientific Officer
    +44 208 123 5368