Key takeaways:
Pridopidine is a twice-daily oral capsule that may help improve thinking, behavior, and movement changes for those with Huntington’s disease. The FDA gave it a fast track development designation, but it’s still being studied.
Other future treatments for Huntington’s disease are in the works, too. Tominersen, ifezuntirgene inilparvovec, and WVE-003 are three examples of many.
Xenazine (tetrabenazine), Austedo (deutetrabenazine), and Ingrezza (valbenazine) are FDA approved for Huntington’s disease. They help calm movement problems (chorea symptoms) from the condition.
Save on related medications
A Huntington’s disease diagnosis isn’t something that most people expect to hear. But when the news comes from your neurologist, it’s natural for your mind to jump straight to treatment options. Most people can partially manage the condition through therapy, medication, and lifestyle changes.
More hope is on the horizon, though. Several medications for Huntington’s disease are being studied in clinical trials — including in trials you can sign up for.
With new medications in development for Huntington’s disease, it’s possible that we could be able to slow down or halt the progression of this genetic brain condition in the not-so-far future. Eleven possible future treatments for Huntington’s disease are highlighted below.
1. Pridopidine
Pridopidine is a twice-daily oral capsule that’s being studied for Huntington’s disease. It works by turning on a protein in the brain and spinal cord called sigma-1 receptor (S1R). This is thought to have a few effects, such as limiting inflammation and improving communication between nerve cells.
In a recent phase 3 study, people who took pridopidine without other medications experienced benefits related to thinking, behavior, and movement. But the same benefits weren’t seen among people taking antipsychotics or other movement-aiding medications.
More pridopidine studies are set to start in 2026, and a specific approval timeline hasn’t been announced. But, pridopidine did get a fast track designation from the FDA to help speed up its development. Researchers are also looking at information from several studies to better understand who is most likely to benefit from it.
Besides Huntington’s disease, pridopidine is being studied for amyotrophic lateral sclerosis (ALS).
2. Tominersen
Tominersen is an injectable medication that’s administered into the spine every 16 weeks (4 months). It’s a small strand of genetic material that stops the production of all forms of huntingtin protein. A mutated form of this protein is a likely cause of Huntington’s disease.
After first being shut down due to effectiveness concerns in early 2021, tominersen is now enjoying a revival. In early 2022, its manufacturer announced new plans to study the medication in a smaller group of people: Adults with early signs of Huntington’s disease.
This phase 2 trial is now studying tominersen’s safety and effectiveness more in depth. Its manufacturer is focusing its efforts on the 100 mg dose. The trial should finish up in 2026, but an FDA approval application likely won’t come before 2028.
3. Ifezuntirgene inilparvovec
Ifezuntirgene inilparvovec (AMT-130) is the first gene therapy medication to enter clinical trials for Huntington’s disease. Gene therapy medications aim to correct genetic disorders by directly tackling the genetic issue(s) at fault.
Ifezuntirgene inilparvovec is designed to give your body genetic instructions to stop making mutated huntingtin protein. It’s a one-dose medication that’s injected into specific parts of the brain.
Early results have been promising. In one study, people who received ifezuntirgene inilparvovec showed about 75% slower progression of their symptoms over 3 years compared with a group of similar people who didn’t get the treatment. They also had smaller increases in a brain damage marker called neurofilament light, suggesting less cell injury. The treatment has been well tolerated overall, but it does require brain surgery.
The company that makes ifezuntirgene inilparvovec is planning more meetings with the FDA to talk about how these results might support larger studies or a future path to approval.
4. WVE-003
WVE-003 is injected directly into the spine, similar to tominersen. But it’s only meant to be administered once, and it’s more specific in its tactics. While tominersen lowers huntingtin protein levels in general, WVE-003 prevents the production of mutated huntingtin protein in people with a genetic mutation called SNP3.
An ongoing phase 1/2 study is studying WVE-003’s safety. Early results show that WVE‑003 can safely lower the harmful form of huntingtin protein in spinal fluid for people who carry the SNP3 genetic change. A phase 2/3 study for adults is now in the works.
5. Votoplam
Votoplam (PTC518) is a once-daily capsule being studied for Huntington’s disease. It’s designed to lower the amount of huntingtin protein in the body. Votoplam does this by changing how the body processes huntingtin messenger RNA, which leads to less of the harmful protein being made.
In a phase 2 study, people who took votoplam for 1 year had lower levels of huntingtin protein in their blood compared to placebo. The higher the dose, the bigger the drop. Researchers are now studying votoplam in a long-term trial to learn more about its safety and benefits.
Larger late-stage studies need to be completed before the medication can be submitted to the FDA for review.
6. Bevantolol hydrochloride
Bevantolol hydrochloride (SOM3355) is a daily oral capsule. It’s being developed as another option to treat chorea symptoms (movement problems) from Huntington’s disease.
Bevantolol hydrochloride works in two different ways. It’s a beta blocker, like propranolol, and a vesicular monoamine transporter 2 (VMAT2) inhibitor, like Ingrezza (valbenazine). Its manufacturer promotes it as a more-tolerable alternative to other similar medications for Huntington’s disease.
A global, phase 3 trial in early-to-severe Huntington’s disease is set to start by the end of 2026. If the results are positive, an FDA approval application for bevantolol hydrochloride may come next.
7. Pepinemab
Pepinemab is an IV medication that’s injected once a month. A type of monoclonal antibody, it blocks a protein called semaphorin 4D (SEMA4D). This is thought to help fight inflammation and preserve brain activity.
Pepinemab’s phase 2 trial data published in August 2022 had mixed results. It didn’t have a significant benefit in people without noticeable symptoms of Huntington’s disease. It was more helpful for people who already had some degree of thinking or behavioral changes.
At this time, pepinemab’s development timeline is cloudy. The company hasn’t announced any new late‑stage Huntington’s trials since the initial phase 2 results, and current efforts appear to be focused more on other conditions.
8. SKY-0515
SKY-0515 is another once-daily pill being studied as a treatment for Huntington’s disease. It’s designed to act on the genetic instructions that cause the body to make too much huntingtin protein. It may also interfere with a protein called PMS1, which is linked to ongoing genetic changes that can make Huntington’s disease worse over time.
In an early phase 1 study, SKY-0515 lowered levels of the harmful huntingtin protein in a dose-dependent manner. After 3 months, people taking the higher dose saw about a 60% drop, while those taking the lower dose saw nearly a 30% drop. The medication has been well tolerated so far, with no major safety concerns reported.
Building on these results, a larger phase 2/3 trial is now underway. It’ll test multiple doses of SKY-0515 against placebo in people with early-to-mid Huntington’s disease. The study is expected to last for at least a year.
9. Tanruprubart
Tanruprubart (ANX-005) is an IV monoclonal antibody medication that’s given every 2 weeks. It’s designed to interfere with a specific part of your immune system (called the “complement pathway”). This pathway normally helps your body fight germs, but it has also been linked to damaging nerve connections in the brain. Tanruprubart could help preserve brain function by blocking it.
So far, tanruprubart appears to be safe and well tolerated in early studies for Huntington’s disease. But the company developing it is currently prioritizing an approval application for its use in Guillain-Barré syndrome. As a result, a large Huntington’s disease trial hasn’t been launched yet, and future plans for this use are still being reviewed alongside the company’s other priorities.
10. SRX246
SRX246 is an experimental pill being studied to help reduce irritability and aggression experienced by people with Huntington’s disease. It blocks a specific brain receptor, called the vasopressin V1a receptor, which may help with mood symptoms without affecting movement.
In a phase 2 trial, SRX246 appeared to lessen irritability and was generally well tolerated. It also received orphan drug and fast track status from the FDA. However, no new studies have launched recently, and SRX246 isn’t currently moving into larger trials or available outside of research settings.
11. Cellavita-HD
Cellavita-HD is a stem cell therapy that’s actively being studied. Stem cell therapies like Cellavita-HD aim to restore lost brain cells affected by the condition. It’s a monthly IV injection that’s given in cycles.
Cellavita‑HD has shown that it can be safely administered in small, early‑stage studies, but there isn’t clear proof yet that it actually improves Huntington’s symptoms or slows the disease. What’s more, this therapy is mainly being developed in Brazil, and the company that makes it hasn’t announced plans to pursue FDA approval.
FDA-approved Huntington’s disease medications
As of December 2025, three medications are FDA approved to treat chorea symptoms from Huntington’s disease. They’re called Xenazine (tetrabenazine), Austedo (deutetrabenazine), and Ingrezza. A longer-lasting form of Austedo, called Austedo XR, is also available. They’re all VMAT2 inhibitors that work by calming excessive dopamine levels in the brain.
Want to learn how Xenazine and Austedo stack up? Another GoodRx Health article delivers a full comparison.
Common off-label medications
Most medications used to treat Huntington’s disease today are given off-label, or in a way that the FDA hasn’t officially approved.
Depending on the situation, your neurology team may recommend one or more of these medications:
Antidepressants to treat depression, like fluoxetine (Prozac) or sertraline (Zoloft)
Antipsychotics to help control movement and/or behavioral changes, like olanzapine (Zyprexa) or risperidone (Risperdal)
Anti-seizure medications to treat certain behavioral changes, like lamotrigine (Lamictal) or carbamazepine (Tegretol)
Keep in mind: Medications aren’t the only way to treat Huntington’s disease. Lifestyle changes, occupational therapy, and deep brain stimulation are also options.
Can cannabis treat Huntington’s disease?
Possibly, yes. A large literature review found that medical cannabis can help lessen muscle spasms, movement problems, and sleeping troubles from Huntington’s disease. Your neurology team can tell you if cannabis is a possible option to manage your symptoms.
The bottom line
Many new Huntington’s disease treatments are in the works. Several medications in clinical trials — including pridopidine, ifezuntirgene inilparvovec, WVE-003, and others — are showing early signs that they may help manage symptoms or target the underlying biology of the disease. Others, such as tanruprubart, have also shown encouraging results.
For now, three medications — Xenazine (tetrabenazine), Austedo (deutetrabenazine), and Ingrezza (valbenazine) — are FDA approved to treat movement problems (chorea symptoms) from Huntington’s disease. Many other medications, including antidepressants, antipsychotics, and anti-seizure medication, are prescribed off label to help address various symptoms.
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