What treatments are on the horizon for SCN8A?
For years, many of us have been struggling with anti-seizure meds (ASMs) that don't really help our SCN8A children. They may reduce (or increase) the seizures for a while, but they are drugs that were made with epilepsy more generally in mind, not kids with mutations on their sodium channel.
There has never been a more exciting time for our community! As of early 2021, there are two compounds in development by two different pharmaceutical companies. We are constantly exploring additional options for treatment, whether they are off-label drugs, research being conducted or discussions with other drug companies.
Praxis Precision Medicines has recently received rare pediatric disease designation for it's compound, PRAX-562, for treatment SCN8A-DEE. See the latest community statement we helped them craft to answer YOUR questions below!
Neurocrine Biosciences received FDA approval to begin Phase II clinical trials for their drug, NBI-921352 (XEN901) for children and young adults 12-21 years old.
Neurocrine Biosciences has the first drug in clinical trials for SCN8A - Developmental and Epileptic Encephalopathy syndrome - SCN8A-DEE
This drug is a selective Nav1.6 sodium channel inhibitor with potential in treating SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE) as well as other forms of epilepsy, including focal epilepsy.