Research
This page provides information about the FDA's new drug approval process, the current status of some ataxia drugs and therapies that are later in the approval pipeline, and some pipelines for different types of ataxias.
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About the FDA's New Drug Approval Process
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The process of approving a new drug or therapy by the FDA (U.S. Food and Drug Administration) involves several rigorous steps to ensure the product’s safety and effectiveness before it becomes available to the public. Here is a summary of the main stages:
1. Preliminary Research and Discovery
Involves laboratory studies and tests on cell cultures and animals to identify substances with therapeutic potential.
2. Preclinical Studies
At this stage, the compound is tested in animal models to evaluate its safety and to gather initial data on efficacy and dosing. These data are used to request Investigational New Drug (IND) approval, which allows testing in humans.
3. Clinical Trials (Phases I, II, and III)
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Phase I: The drug is tested on a small group of healthy volunteers or patients (20–100) to assess safety, safe dosage ranges, and possible side effects.
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Phase II: Involves a larger group (100–300 participants) and focuses on the drug’s efficacy and continued safety assessment under controlled conditions.
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Phase III: Includes hundreds to thousands of patients and evaluates the drug’s efficacy compared to standard treatments or placebo. This phase aims to confirm effectiveness and document side effects in greater detail.
4. FDA Review and Approval
After clinical trials, all data are submitted to the FDA in a New Drug Application (NDA) or Biologics License Application (BLA) for biological therapies. The FDA reviews the data and may request further information or additional trials.
5. Post-Market Monitoring (Phase IV)
Once approved and marketed, the drug continues to be monitored in the broader population to detect rare or long-term side effects, ensuring ongoing safety and effectiveness.
This entire process typically takes 10 to 15 years on average.
This is the process for approving a new drug for commercialization in the United States. A similar process occurs in Europe (regulated by the EMA – European Medicines Agency) and in Brazil (regulated by ANVISA – Brazilian Health Regulatory Agency).
Here is a summary of the regulatory process in Brazil
Once a drug is approved by the FDA (for example), if a pharmaceutical company wishes to sell it in Brazil, it must submit an application to ANVISA, which will conduct its own evaluation suited to the Brazilian context. If ANVISA approves the drug or therapy, it can be marketed in Brazil for the approved indication. To do so, the pharmaceutical company must define the drug’s price in the Brazilian market through CMED. If the market cost for patients is too high, it becomes the responsibility of CONITEC (National Commission for the Incorporation of Technologies into the Unified Health System – SUS) to evaluate the case and, when appropriate, recommend that the drug or therapy be provided free of charge through SUS.
Patient associations (such as Abahe, in the case of ataxias in Brazil) play an important role in many stages of this long and challenging process — from supporting the recruitment of registered patients to participate in clinical trials conducted by pharmaceutical companies, to monitoring discussions between the pharmaceutical company and ANVISA (the Brazilian regulatory agency) when the company expresses interest in marketing the drug in Brazil. They also help connect patients for the implementation of “Real-World Evidence” studies, which can generate data to accelerate the regulatory process. If ANVISA approves the treatment, the association may also follow up on negotiations with CMED (for pricing decisions), and subsequently with CONITEC, to advocate for the treatment to be made available through the public healthcare system (SUS), when applicable. It is a lengthy and rigorous process, but necessary to ensure that all approved medications undergo scientific evaluation for safety and efficacy.
Make your voice heard. Learn more about how to support the availability of new treatments for ataxias through Brazil’s public healthcare system (SUS) on the ADVOCACY page.
Learn more about the rigorous and time-consuming FDA approval process for a new drug or therapy (for ataxias or other conditions) on the " All About New Drug Applications " page on the NAF website.
The topic is also explored in the article From Research to Reality: The 5 Steps in Drug Development, December 1, 2023 (The article is in English, but the page has automatic translation into other languages).
Current status of the most advanced drugs for ataxias
Current Status of Drugs Under Investigation for Ataxias
Last updated: June 2025
1 – FDA-Approved Medications for Ataxias
SKYCLARYS (omaveloxolone) → Specific for Friedreich’s Ataxia
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The drug omaveloxolone (brand name SKYCLARYS), developed by Reata Pharmaceuticals (acquired by Biogen), was approved by the FDA in February 2023. It is the first and only approved treatment specifically for Friedreich’s ataxia.
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Biogen submitted a request for marketing authorization to ANVISA in March 2024.
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On April 11, 2025, ANVISA approved SKYCLARYS for commercialization in Brazil, indicated for patients aged 16 or older with Friedreich’s Ataxia.
AQNEURSA → For Niemann-Pick Type C (NPC)
Aqneursa (levacetylleucine) is indicated for Niemann-Pick disease type C (NPC) — a rare, progressive, autosomal recessive genetic disorder caused by mutations in the NPC1 (most cases) or NPC2 genes. NPC affects lipid metabolism within cells and has a strong impact on the cerebellum, often leading to symptoms of ataxia.
For more information, visit: https://www.aqneursa.com
2 – Drugs Still Under FDA Review
2.1. Medications for Friedreich’s Ataxia
Several therapies are currently under review, at various stages of the FDA approval process.
Some examples of drugs and therapies specifically under review for Friedreich’s Ataxia include:
Vatiquinone (PTC-743) – PTC Therapeutics
Currently under FDA review
PTC Therapeutics also submitted a request to ANVISA in December 2024 for marketing approval of Vatiquinone in Brazil.
The request is under review.
Vatiquinone is a small molecule and a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO) — an enzyme that plays a key regulatory role in cellular energy and oxidative stress pathways, which are disrupted in Friedreich’s Ataxia. Inhibiting 15-LO helps mitigate the effects of mitochondrial dysfunction and oxidative stress by reducing inflammation and promoting neuronal survival. Vatiquinone has been evaluated in multiple clinical trials — many of them focused on pediatric patients — and has shown impact on mortality risk, fatigue, and various neurological and neuromuscular symptoms (source: original text).
Other Therapies for Friedreich’s Ataxia Awaiting FDA Approval
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Elamipretide – Children’s Hospital of Philadelphia
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Leriglitazone (MIN-102) – Minoryx Therapeutics
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Dimethyl Fumarate – University of Federico II
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Nomlabofusp (CTI-1601) – Larimar Therapeutics
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LX2006 (gene therapy) – Lexeo Therapeutics
For the most up-to-date information on the progress of these clinical trials, please refer to the FARA (Friedreich’s Ataxia Research Alliance) clinical trial pipeline.
2.2. Medications for Spinocerebellar Ataxias (SCAs)
As of now, there are no approved medications specifically designed to modify the progression of spinocerebellar ataxias. However, several drugs and therapies are being tested at different stages of the FDA regulatory approval process. As a reference, here are some investigational treatments for Spinocerebellar Ataxia Type 3 (SCA3):
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Troriluzole (BHV-4157) – A glutamate reducer developed by Biohaven Pharmaceuticals Inc.
Already under regulatory review by the FDA for potential approval in the United States. See the next section for more details. -
Intravenous Trehalose (SLS-005) – An aggregation reducer, developed by Seelos Therapeutics, currently in Phase 2.
Unfortunately, the trials are on hold at the moment, despite promising preliminary data. -
ASO Gene Therapy (VO-659) – An antisense oligonucleotide (ASO) approach aimed at silencing the mutant gene responsible for the disease, developed by Vico Therapeutics. Currently in Phase 2.
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Stem Cell Therapies (MSC Therapy) – Stemitent, currently in Phase 1 in the clinical pipeline.
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ASO Gene Therapy for SCA3 – The mission of Cure Rare Disease (CRD) is to develop and fund genetic therapies for ultra-rare diseases. In this context, CRD is developing an ASO (antisense oligonucleotide) therapy to treat spinocerebellar ataxia type 3 (SCA3). See the exclusive interview with Rich Horgan, CEO of CRD.
LEARN MORE ABOUT TRORILUZOLE (BIOHAVEN)
The videos and podcasts below provide more information about Biohaven's drug Troriluzole, a glutamate modulator indicated for spinocerebellar ataxias awaiting FDA approval.
Live Abahe - December 2024
Abahe Volunteer
Administrator of the ataxia.info portal
Language: Portuguese narration with AI generated English subtitles
A podcast with Melissa Beiner, Senior Medical Director at Biohaven Pharmaceuticals, discusses spinocerebellar ataxias and the drug Troriluzole, which is awaiting FDA approval.
Credits: RARECAST - Global Genes Podcast
The First Treatment for a Rare Neurodegenerative Condition Awaits FDA Approval
Date: April 3, 2025
NAF Webinar (National Ataxia Foundation)
All about Troriluzole
Dr. Susan Perlman
Dec 18, 2024
Video in English, but it is possible to activate subtitles and enable automatic translation of subtitles into Portuguese and other languages
NAF Pipeline (National Ataxia Foundation)
The NAF (National Ataxia Foundation) is an American foundation whose mission is "To accelerate the development of treatments and a cure while working to improve the lives of people living with ataxia."
In addition to providing a wealth of useful information on its Portal, NAF also offers a "Pipeline" that graphically and easily displays progress in developing key scientific research into therapies and medications for various types of ataxias. The original content is in English, but you can click a button on the page (Translate) to obtain translations into several other languages, including Portuguese.
TO FIND OUT ABOUT ONGOING RESEARCH, MEDICINES AND THERAPIES FOR ATAXIAS, ACCESS THE NAF PIPELINE HERE .