XEN007

As the parent of a child with CACNA1A, I eagerly await the day there will be a new treatment or a cure for my daughter. In fact, that’s what motivated me to co-found the CACNA1A Foundation. Currently, it’s trial and error as parents, caregivers and patients themselves search for drugs that will mitigate symptoms of epilepsy, ataxia and hemiplegic migraine, among others. We often find ourselves doing our own research and then bringing our discoveries back to our doctors to inform them.

For those in the US, there is some exciting news regarding a new medication being developed for our children with Gain of Function variants. The CACNA1A Foundation recently had a call with Xenon Pharmaceuticals, a clinical-stage biopharmaceutical company committed to developing innovative therapeutics that will improve the lives of patients with neurological disorders and central nervous system (CNS) conditions, with a focus on epilepsy. Their new drug, XEN007, whose active ingredient is flunarizine, is in phase 1 trials. Flunarizine (sibelium), currently available in Europe and Canada, but not in the USA or Japan, is a cav2.1 or calcium channel inhibitor. It has been shown to have clinical benefit and is being evaluated for those with absence epilepsy and hemiplegic migraines. It has also successfully been used to control the frequency and severity of attacks in Alternating Hemiplegia of Childhood. Finally, doctors can prescribe it as a prophylactic treatment for migraine.

While Xenon had not considered marketing the drug specifically for those with CACNA1A, we discussed the possibility of a clinical trial that includes our community.

We’d love to hear from those of you in Europe or Canada who are using the drug and having success with it. While FDA approval is still a long way off, we’re heading in the right direction!