What is the new medicine for ataxia?

October 10, 2025 •

4 min read

In a significant development for the rare disease community, the FDA approved the first-ever treatment for Friedreich's ataxia in early 2023, offering a major breakthrough after years with no disease-specific drugs. This milestone, along with other promising therapies currently under review, provides a clearer answer to the question, "What is the new medicine for ataxia?" and fuels optimism for future treatments across various forms of this debilitating condition.

Quick Summary

The first approved medication is omaveloxolone (Skyclarys) for Friedreich's ataxia in patients aged 16 and older. Several other investigational treatments, including troriluzole for spinocerebellar ataxia and multiple gene therapies, are currently in late-stage clinical trials. These new and emerging therapies offer hope for modifying disease progression.

Key Points

First-ever approved drug for Friedreich's ataxia: The FDA approved Skyclarys (omaveloxolone) in 2023 for FA patients aged 16 and older, which helps slow disease progression by activating the Nrf2 pathway.
Troriluzole for spinocerebellar ataxia: Biohaven's drug troriluzole is under FDA review for spinocerebellar ataxia, with a decision expected in late 2025. It aims to modulate glutamate to slow disease progression.
Gene therapy in development: Promising gene therapies like LX2006 and ASP2016 for Friedreich's ataxia cardiomyopathy are in clinical trials, with the goal of delivering a functional frataxin gene to heart cells.
Other investigational treatments: Vatiquinone for FA and a specialized antisense oligonucleotide (ASO) for SCA3 are among other therapies advancing through clinical development.
Symptomatic and supportive care remains vital: Alongside new drugs, therapies like physical, occupational, and speech therapy, along with off-label medications, are crucial for managing symptoms and maintaining quality of life.
Patient advocacy and research collaboration drive progress: Success stories like omaveloxolone highlight the importance of joint efforts between patient groups, researchers, and pharmaceutical companies in accelerating rare disease treatments.

The Breakthrough for Friedreich's Ataxia: Skyclarys (Omaveloxolone)

For many years, treatment options for hereditary ataxias were limited to managing symptoms. This changed with the February 2023 approval of Skyclarys (omaveloxolone) by the U.S. Food and Drug Administration (FDA), the first and only approved drug for Friedreich's ataxia (FA). This is an oral capsule taken once daily and is approved for use in patients aged 16 and older with genetically confirmed FA.

Omaveloxolone works by activating the Nrf2 pathway, which regulates the cellular defense against oxidative stress. Patients with FA have impaired Nrf2 signaling due to low levels of the mitochondrial protein frataxin, which is the root cause of the disorder. Clinical trials demonstrated that omaveloxolone significantly slowed the progression of neurological symptoms compared to a placebo, as measured by the modified Friedreich's Ataxia Rating Scale (mFARS). Post-hoc analysis showed the effect was sustained in an open-label extension study. The most common side effects reported were mild and included increased liver enzymes, headache, and nausea. Biogen is also studying omaveloxolone in a Phase 3 trial for pediatric patients with FA.

Promising Investigational Drugs for Other Ataxias

While Skyclarys is a major step forward, many other forms of ataxia still lack approved disease-modifying therapies. However, the pipeline for novel treatments is expanding rapidly, with several candidates showing promise in clinical trials.

Troriluzole for Spinocerebellar Ataxia (SCA): In early 2025, Biohaven's troriluzole received FDA Priority Review for the treatment of SCA. SCA is a group of genetic ataxias that currently have no approved disease-specific treatments. Troriluzole is a glutamate modulator, a different mechanism from omaveloxolone, and clinical trial results showed a significant slowing of disease progression over a 3-year period in SCA patients. The FDA's decision is expected in late 2025.
Vatiquinone for Friedreich's Ataxia: In February 2025, PTC Therapeutics also had its New Drug Application (NDA) for vatiquinone accepted by the FDA for Friedreich's ataxia, with a target decision date in August 2025. Vatiquinone aims to improve mitochondrial function and cellular health, particularly in the pediatric population.

Cutting-Edge Gene Therapy Approaches

For genetic ataxias like FA and SCA, gene therapy and gene editing represent the future of treatment by addressing the underlying genetic defect.

Gene Replacement for FA: Lexeo Therapeutics is developing LX2006, a gene therapy designed to deliver a functional frataxin gene to the heart cells of FA patients with cardiomyopathy. Interim Phase 1/2 data released in 2024 showed increased frataxin protein expression, supporting its advancement to a pivotal study. Astellas is also working on a similar gene therapy, ASP2016, with a Phase 1 trial planned for 2024.
Protein Replacement for FA: Larimar Therapeutics is testing nomlabofusp, a novel protein replacement therapy that delivers a full-length frataxin protein directly to the mitochondria. Recent long-term data indicates a sustained increase in frataxin levels.
Gene-Targeting for SCA3: Cure Rare Disease received a significant grant in 2025 to advance a splice-switching antisense oligonucleotide (ASO) therapy for Spinocerebellar Ataxia type 3 (SCA3), which is designed to prevent the production of toxic proteins.

The Importance of Supportive Care

Regardless of new drug developments, supportive treatments remain crucial for managing ataxia symptoms and maximizing quality of life.

Physical Therapy: Specialized exercises can improve balance, coordination, strength, and mobility, helping patients manage gait problems and reduce fall risk. Vestibular rehabilitation can also be effective for certain types of ataxia.
Occupational Therapy: Therapists can provide strategies and adaptive devices to make daily living tasks, like eating, dressing, and writing, easier and safer.
Speech-Language Pathology: Speech therapy can help with dysarthria (slurred speech) and dysphagia (difficulty swallowing).
Symptomatic Medications: Off-label drugs, such as riluzole for coordination or baclofen for spasticity, may be used under a physician's guidance to address specific symptoms.

Comparing New and Emerging Ataxia Treatments


Feature	Skyclarys (Omaveloxolone)	Troriluzole (Investigational)	Gene Therapies (e.g., LX2006, ASP2016)
Target Condition	Friedreich's Ataxia (FA)	Spinocerebellar Ataxia (SCA)	Friedreich's Ataxia (FA), focusing on cardiomyopathy
Mechanism	Activates the Nrf2 pathway, reducing oxidative stress and improving mitochondrial function.	Modulates glutamate neurotransmission to protect neurons and reduce excitotoxicity.	Delivers a functional copy of the frataxin gene to heart cells, restoring frataxin levels.
Status	FDA-approved for ages 16+ (Feb 2023).	FDA Priority Review granted (Feb 2025); decision expected in late 2025.	In Phase 1/2 clinical trials for FA cardiomyopathy.
Formulation	Oral capsules, taken once daily.	Oral medication, once daily.	Single intravenous infusion.
Side Effects	Increased liver enzymes, nausea, headache, abdominal pain.	Mild liver enzyme increases, vertigo (reported for older version).	Potential immune reactions to the viral vector; monitored with immunosuppression.

Conclusion

The approval of Skyclarys marked a monumental shift in the treatment landscape for ataxia, proving that targeted, disease-modifying therapies are possible for rare neurodegenerative diseases. As research continues at an unprecedented pace, the outlook for other ataxia types is improving. Upcoming regulatory decisions on troriluzole and vatiquinone, along with progress in gene therapies, could provide specific treatments for more patients in the coming years. While a cure for hereditary ataxias remains elusive, the combination of groundbreaking pharmacological research and effective supportive care offers renewed hope and the potential to significantly slow disease progression for many. The sustained efforts of researchers, clinicians, and patient advocacy groups are critical to building on this momentum and bringing more life-changing treatments from the laboratory to the clinic. For the latest developments, organizations like the National Ataxia Foundation serve as an excellent resource.

Frequently Asked Questions

The new FDA-approved medication for ataxia is Skyclarys (omaveloxolone), which was approved in February 2023 for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.

Skyclarys works by activating the Nrf2 pathway, a cellular defense mechanism that helps manage oxidative stress. In Friedreich's ataxia, where frataxin levels are low, this pathway is impaired, and omaveloxolone helps restore its function to slow disease progression.

There is currently no FDA-approved treatment for SCA. However, the investigational drug troriluzole from Biohaven is under FDA Priority Review, with a decision expected in late 2025.

Several gene therapies are in development, including LX2006 from Lexeo Therapeutics for Friedreich's ataxia cardiomyopathy, which delivers a functional frataxin gene to the heart via a single infusion. Cure Rare Disease is also advancing an ASO therapy for SCA3.

Yes, other drugs are in the pipeline. Vatiquinone from PTC Therapeutics is also under FDA review for FA, with a Prescription Drug User Fee Act (PDUFA) date set for August 2025. Other approaches, like protein replacement therapies, are also being explored.

Supportive therapies include physical, occupational, and speech therapy, which help with coordination, daily tasks, and swallowing issues. Adaptive devices like walkers or specialized utensils can also help improve daily function.

The most common side effects reported for Skyclarys are elevated liver enzymes, headache, nausea, abdominal pain, fatigue, and musculoskeletal pain.

Newer medications like omaveloxolone and pipeline candidates like troriluzole are designed to be disease-modifying by targeting the underlying genetic or molecular mechanisms of specific ataxias. This differs from older treatments that only address symptoms.

No, Skyclarys is specifically approved for Friedreich's ataxia, an autosomal recessive disorder caused by a frataxin gene mutation. It is not indicated for other forms of ataxia.

You can follow updates from patient advocacy organizations like the National Ataxia Foundation and the Friedreich's Ataxia Research Alliance (FARA). You can also search for active trials on ClinicalTrials.gov.