Reviewed by Sarim Mir, MD — Board-Certified Neurologist, Mir Neurology (Hagerstown & Cumberland, MD)
Key takeaways
- Epilepsy treatment is shifting toward precision medicine — matching treatment to the specific cause of a person’s seizures rather than using one-size-fits-all medicines.
- This includes gene-targeted therapies and syndrome-specific treatments aimed at the underlying biology of certain epilepsies.
- Examples in development include RAP-219 (entering Phase 3 for focal onset seizures) and vormatrigine (studied for SCN2A/SCN8A developmental and epileptic encephalopathies).
- These therapies are investigational — they are still in clinical trials and are not yet FDA-approved or available to prescribe.
- Precision medicine is about better-matched, not guaranteed, treatment; it is not a cure, and standard care still matters today.
What changed
For years, most epilepsy treatment relied on broad-spectrum anti-seizure medicines — drugs designed to quiet seizures in general, regardless of their exact cause. In 2026, neurology updates highlight a clear shift toward precision medicine: choosing treatment based on the specific mechanism or gene driving a person’s seizures.
This change is being driven by a better understanding of the genetics and biology of epilepsy, plus new therapies designed to target those specific causes. Two examples often mentioned are RAP-219, which is moving into Phase 3 testing for focal onset seizures, and vormatrigine, studied for certain rare, severe childhood epilepsies linked to the SCN2A and SCN8A genes. Both are investigational, meaning they are still in clinical trials and not yet approved by the FDA.
Plain-English explainer: what is epilepsy, and what is precision medicine?
Epilepsy is a condition in which the brain produces sudden bursts of abnormal electrical activity, called seizures. Seizures can look very different from person to person — staring spells, jerking movements, unusual sensations, or loss of awareness — depending on where in the brain they begin and why.
Precision medicine means tailoring treatment to the individual rather than treating everyone with the same approach. In epilepsy, that can mean using genetic testing and detailed diagnosis to understand exactly what is causing the seizures, then choosing a therapy aimed at that specific cause. Some severe epilepsies, especially in children, are called developmental and epileptic encephalopathies — conditions where frequent seizures and abnormal brain activity can also affect development. When these are linked to a specific gene, a treatment aimed at that gene’s effect may, in theory, work better than a broad medicine. That is the promise precision medicine is trying to deliver.
How it works / what’s different
Broad-spectrum seizure medicines turn down brain excitability across the board. They help many people, but because they do not target the root cause, they can fall short for some and cause side effects in others. Precision approaches try to address the underlying problem more directly.
RAP-219 is being studied as a more targeted approach to focal onset seizures and is entering Phase 3 testing, the large stage of trials that compares a therapy against a control to confirm whether it works and is safe. Vormatrigine is being studied for epilepsies tied to the SCN2A and SCN8A genes, which affect tiny sodium channels that control how brain cells fire; the goal is to match the therapy to that specific genetic mechanism. The key difference is intent: instead of broadly calming the brain, these therapies aim at a defined target identified through diagnosis and, in some cases, genetic testing.
It is important to keep expectations grounded. These are investigational therapies in clinical trials. Their benefits and risks are still being measured, and being “targeted” does not guarantee success for any one person. Precision medicine is a promising direction, not a finished destination, and approved standard care remains the foundation of treatment today.
What this means for you in Hagerstown & Cumberland
For families in Western Maryland, precision medicine signals a future where epilepsy care can be matched more closely to each person — but today, the practical step is getting an accurate diagnosis and, when appropriate, considering genetic testing. At Mir Neurology, Dr. Sarim Mir provides EEG testing and long-term seizure management for adults and children, and can help determine whether a precise diagnosis points toward specific treatments or clinical trial opportunities. Learn more about our epilepsy care and EEG testing.
If you or your child has seizures that are hard to control, or a possible genetic epilepsy, a thorough evaluation is the best place to start. Call (301) 797-7600 to request an appointment, or request an appointment online.
Frequently asked questions
Is precision medicine for epilepsy available now? Some pieces are, and some are not. Accurate diagnosis and genetic testing are available today and can guide care. But specific gene-targeted therapies like RAP-219 and vormatrigine are still investigational, meaning they are in clinical trials and not yet FDA-approved for general use.
Does my child need genetic testing for epilepsy? Not everyone does, but for certain epilepsies — especially severe, early-onset, or hard-to-control seizures — genetic testing can help identify a specific cause. A neurologist can advise whether testing makes sense based on your child’s history, EEG, and symptoms.
Will precision medicine cure epilepsy? No. Precision medicine aims to match treatment more closely to the cause of seizures, which may improve results for some people. It is not a cure, and the targeted therapies discussed here are still being studied for safety and effectiveness.
This article is for general education and is not a substitute for personalized medical advice from your own physician.
Sources: European Academy of Neurology — Updates in Epilepsy Treatment 2026, Rapport Therapeutics — Announces Accelerated Initiation of RAP-219 Phase 3
Need to talk to a neurologist about this?
Mir Neurology offers same-week appointments in Hagerstown & Cumberland, MD. No referral needed for most plans.
