Marinus Pharmaceuticals has released positive initial results of ganaxolone in TSC-related epilepsy, with the phase 2 trial demonstrating a reduction in seizure frequency in people living with TSC-related epilepsy. The encouraging findings pave way for a larger study of the medicine, expected to begin at the end of 2021.

Ganaxolone’s phase 2 trials showed a 16% reduction in seizure frequency after 28 days, with 30% of participants achieving at least a 50% seizure reduction. During the trial, patients with focal seizures showed a median 25% reduction in focal seizure frequency.

Phase 2 trials aim to increase the number of people that a medicine is tested on, and to understand potential benefits and side-effects of the medicine. Positive phase 2 trials open the door to larger phase 3 trials, which include a large number of participants and aims to further understand the medicine’s benefits and compare its effectiveness against existing treatments.

The trial update also coincided with the U.S. Food and Drug Administration (FDA) granting orphan drug designation to ganaxolone for the treatment of TSC-related epilepsy in the United States. This gives certain benefits, including reduced FDA fees for clinical trials.

Ganaxolone works by encouraging greater activity of transmitters in the brain, called GABA, which are responsible for blocking certain nerve cells from activating. Following the completion of all trials (which ganaxolone must still do), it is typical but not certain for a medicine’s company to first seek approval and licensing for the medicine in the United States, before then moving on to the UK and/or European Union.

The TSA will continue to speak with decision-makers and campaign to push for access to medicines that have the potential to improve lives in the TSC community. We will also keep the TSC community informed at every step.

The press release by Marinus Pharmaceuticals can be found here.