NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
SIGN THE PETITION
NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
SIGN THE PETITION
June 18, 2025
Biogen reports that they have initiated the global Phase 3 BRAVE study to evaluate the efficacy and safety of omaveloxolone in approximately 255 children with Friedreich ataxia (ages 2–15). The study includes a 52-week placebo-controlled period followed by a 104-week open-label extension. Enrollment is now open at U.S. sites, with additional international sites planned pending regulatory approval.
May 14, 2025
Biohaven announced that the Division of Neurology 1 within FDA’s Office of Neuroscience informed them that they are extending the PDUFA date for the troriluzole new drug application (NDA) for the treatment of Spinocerebellar Ataxia (SCA) by three months to provide time for a full review of Biohaven’s recent submissions related to information requests from the FDA. The Division also informed Biohaven that it is currently planning to hold an advisory committee meeting to discuss the application, but no date has been scheduled. The FDA did not raise any new concerns in the letter. The FDA’s decision regarding the NDA is now expected in 4Q 2025.
May 5, 2025
Biogen shared their plan to initiate the BRAVE study, a global Phase 3 study evaluating the effects and safety of omaveloxolone in children with Friedreich Ataxia between the ages of 2-15 years. Individuals interested in participating in the study should speak with their healthcare provider.
April 7, 2025
Lexeo Therapeutics announced positive interim data across all dose cohorts of LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. In both the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271), treatment with LX2006 was associated with clinically significant improvements in cardiac biomarkers and functional measures, and increased frataxin protein expression was observed in all participants with cardiac biopsies.
March 28, 2025
Solaxa Inc. has announced plans to conduct a registrational clinical trial evaluating its investigational therapy, SLX-100, for Spinocerebellar Ataxia type 27B (SCA27B). This study will be funded in part by a $7.3 million dollar grant awarded to Dr. Susan Perlman, MD, at the University of California, Los Angeles by the Congressionally Directed Medical Research Program. Solaxa will serve as the regulatory sponsor of the trial and partner with leading ataxia centers of excellence. Additional clinical trial sites will be announced later this year.
March 24, 2025
Lexeo Therapeutics shared updates about their investigational gene therapy candidate, LX2006, for the treatment of Friedreich Ataxia Cardiomyopathy.
Highlights include:
March 17, 2025
Biogen Canada Inc. announced that Health Canada has approved SKYCLARYS™ (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in patients 16 years of age and older. This approval – granted under Health Canada’s Priority Review process – marks an important milestone making SKYCLARYS the only treatment in Canada to specifically target the underlying mechanisms of this rare, progressive neurodegenerative disease.
February 19, 2025
PTC Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich’s ataxia (FA). The application has been granted Priority Review.
Learn more about what happens during the FDA review in our PrepRARE article: All About New Drug Applications (NDAs).
February 11, 2025
Biohaven announced that the US Food and Drug Administration (FDA) has accepted for review the Company’s New Drug Application (NDA) for troriluzole for the treatment of adult patients with spinocerebellar ataxia (SCA) and has granted Priority Review. This designation is assigned to applications for drugs that would offer a significant improvement over other available treatments for a given disorder or would provide a treatment option where none exists. In the case of SCA, a rare, genetic, neurodegenerative disease, troriluzole would be the first and only FDA-approved treatment for this life-threatening disorder. The FDA’s decision regarding the NDA is expected within 6 months of filing (during 3Q2025). Based on FDA Priority Review timelines and if ultimately approved, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025.
Andrew Rosen, Chief Executive Officer of the National Ataxia Foundation (NAF), stated, “Biohaven’s SCA program reflects years of dedicated clinical research and collaboration with leading world experts and advocacy groups to advance the ataxia field. We are proud that our multi-year effort to fund and support the Clinical Research Consortium for the Study of Cerebellar Ataxia played such a critical role in providing the external control arm of Biohaven’s study. The goal of CRC-SCA is to improve our understanding of SCA disease progression and to promote the development of disease-modifying therapies for SCA.” Mr. Rosen added, “On behalf of patients and families, who have watched generations of family members succumb to this devastating disease and have been waiting for decades for a treatment that could slow disease progression, I thank the FDA for not only accepting this NDA for review, but recognizing the need for urgency for our community in the form of a Priority Review.”
What is an NDA and What Happens Next?
Getting a new medicine approved for use in the United States involves a detailed process overseen by the U.S. Food and Drug Administration (FDA). This process is called the New Drug Application (NDA). NDAs are crucial for making sure medicines are safe and effective before they’re available to the public. Learn more about what happens during the FDA review in our PrepRARE article: All About New Drug Applications (NDAs).
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January 23, 2025
Larimar Therapeutics announced that dosing of adolescents 12-17 years old has started in the Company’s pediatric PK run-in study for patients with Friedreich’s ataxia (FA).
January 6, 2025
Solaxa Inc. announced that the U.S. Food and Drug Administration (FDA) has provided formal clearance of the company’s Investigational New Drug (IND) application to proceed with human clinical studies to evaluate SLX-100. Solaxa is developing SLX-100 as a treatment for patients with spinocerebellar ataxia (SCA).
December 19, 2024
PTC Therapeutics announced the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA). The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies including pediatric and adult FA patients. Data from these three studies demonstrate significant, durable and clinically meaningful evidence of slowing disease progression on key aspects of disease. In addition, these studies demonstrate that vatiquinone is safe and well tolerated in all age groups studied.
December 16, 2024
Larimar Therapeutics announced positive initial data from the ongoing long-term OLE study evaluating daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver in participants with FA. The Company also provided a nomlabofusp development program update.
November 13, 2024
Lexeo Therapeutics, Inc. provided a regulatory update on LX2006 for the treatment of FA cardiomyopathy. In October 2024, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for LX2006 for the treatment of FA cardiomyopathy based on interim clinical data announced in July 2024. They have also completed enrollment of SUNRISE-FA Phase 1/2 trial, with four participants administered lX2006 in cohort 3. For more details, read the full press release.
October 8, 2024
PTC Therapeutics provided today several positive updates on the vatiquinone Friedreich ataxia (FA) program. The pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression. In addition, PTC recently aligned with FDA on key aspects of the planned NDA submission for vatiquinone.
September 23, 2024
Biohaven announced positive topline results from pivotal Study BHV4157-206-RWE (NCT06529146) demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment. The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA at years 1 and 2 (Figure 1). SCA is a rare, progressively debilitating neurodegenerative disease that affects approximately 15,000 people in the United States and 24,000 in Europe and the United Kingdom. There are no FDA approved treatments for SCA.
August 8, 2024
As a result of regulatory interactions, Biohaven designed a new protocol to assess the effectiveness of troriluzole after 3 years of treatment in SCA. This new protocol, BHV4157-206-RWE (NCT06529146), uses real world evidence (“RWE”) of effectiveness using real-world data (“RWD”) sources to examine the treatment effects of troriluzole in SCA after up to 3 years of therapy compared to external control subjects collected from the US SCA Natural History cohort (“CRC-SCA”). The data analysis will incorporate new patient data that was previously unavailable and not analyzed in the prior FDA submission. The primary outcome measure for protocol BHV4157-206-RWE is the change from baseline on the f-SARA at 3 years. The protocol was designed to be consistent with FDA’s Guidance for Industry, “Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision Making for Drug and Biological Products.” The protocol and SAP were submitted and reviewed by FDA prior to database lock of newly available 3-year data. Topline data from this protocol is expected in the second half of 2024 and, if positive, could form the basis of a new drug application to the FDA. We will provide further updates on the SCA development program as warranted by any continued positive progress from the outcome of future regulatory interactions on this topic.
This new protocol is listed on ClinicalTrials.gov. Learn more: clinicaltrials.gov/study/NCT06529146?term=NCT06529146&rank=1
July 15, 2024
Lexeo Therapeutics, Inc. announced positive interim data of LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. Across both the Lexeo SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271), LX2006 was well tolerated with no treatment-related serious adverse events, and clinically meaningful improvements in cardiac biomarkers were observed with increasing improvement over time.
April 22, 2024
Lexeo Therapeutics, Inc. announced an in-license agreement with Cornell University to expedite development of the investigational gene therapy candidate LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy.
Under the license agreement, Lexeo has acquired certain rights1 including rights to current and future data generated in an ongoing investigator-initiated Phase 1A trial of AAVrh.10hFXN to treat FA cardiomyopathy (NCT05302271). The agreement will support Lexeo’s efforts to develop a potentially life-changing therapy for this unmet need.
April 16, 2024
Lexeo Therapeutics, Inc. announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to LX2006, the company’s AAVrh.10hFXN-based gene therapy candidate for the treatment of Friedreich’s ataxia (FA) cardiomyopathy. LX2006 is designed to deliver a functional frataxin gene to promote frataxin protein expression and restore mitochondrial function in myocardial cells.
Fast Track is a process designed to facilitate the development and expedite the review of new drugs intended to treat serious conditions and address unmet medical need. This designation was granted based on available preclinical data. SUNRISE-FA, a Phase 1/2 multicenter, 52-week, dose-ascending, open-label clinical trial, is ongoing to evaluate the safety and tolerability, as well as preliminary efficacy, of LX2006 in patients with FA cardiomyopathy.
March 11, 2024
Lexeo Therapeutics issued a press release announcing financial results and operational highlights. In this release, they report frataxin protein expression data from a subset of the second dose cohort of SUNRISE-FA, a Phase 1/2 clinical trial of LX2006 for the treatment of Friedreich’s Ataxia (FA) cardiomyopathy, showing positive change in post-treatment frataxin levels.
March 11, 2024
Larimar Therapeutics announced dosing of the first patient in an open label extension (OLE) study evaluating 25 mg daily subcutaneous injections of nomlabofusp in participants with Friedreich’s ataxia (FA). Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich’s ataxia (FA) by delivering frataxin to mitochondria.
February 29, 2024
Biohaven Ltd. announced recent business developments in their latest press release. Biohaven has continued to have constructive dialogue with the FDA regarding its SCA development program and potential future data analyses to address regulatory concerns in the previously issued refuse-to-file decision on its NDA application for SCA3. Biohaven will provide further updates on the SCA development program as warranted by any continued positive progress from the outcome of future regulatory interactions on this topic.
February 26, 2024
Voyager Therapeutics, Inc. announced that the joint steering committee with its collaborator Neurocrine Biosciences has selected a lead development candidate in the Friedreich’s ataxia (FA) program. The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER™ capsid discovery platform. The companies expect the program to advance into first-in-human clinical trials in 2025.
February 12, 2024
Biogen Inc. announced the European Commission (EC) has authorized SKYCLARYS® (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. SKYCLARYS is the first treatment approved within the European Union for this rare, genetic, progressive neurodegenerative disease.
Related News: FDA Approves First Treatment for Friedreich’s Ataxia
February 12, 2024
Larimar Therapeutics, Inc. today announced positive top-line data and successful completion of its four-week, placebo-controlled Phase 2 dose exploration study of nomlabofusp (CTI-1601) in participants with Friedreich’s ataxia (FA). Nomlabofusp was generally well tolerated and demonstrated dose dependent increases in frataxin (FXN) levels in all evaluated tissues (skin and buccal cells) after daily dosing of 14 days followed by every other day dosing until day 28 in the 25 mg and 50 mg cohorts. Participants in the 25 mg (n=13) and 50 mg (n=15) cohorts were randomized 2:1 to receive subcutaneous injections of nomlabofusp or placebo.
January 5, 2023
Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced a $60 million Series B financing. “This financing positions us to accelerate our efforts to advance our lead program VO659, currently in a Phase 1/2a clinical study of patients with spinocerebellar ataxia types 3 and 1 and Huntington’s disease,” said Micah Mackison, chief executive officer at Vico.
The financing will support overall company operations including advancement of the ongoing Phase 1/2a multi-center, open-label basket trial of VO659 in patients living with spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) and Huntington’s disease (HD). The trial is designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with mild to moderate SCA3, SCA1 and early manifest HD, with the first patient dosed in April 2023. The financing will also help support discovery and research efforts to further expand the company’s pipeline in other genetic neurological diseases.
November 30, 2023
Variantyx, a leader in genomic precision medicine, today announced that the set of short tandem repeat (STR) expansions detected by its Genomic Unity® line of whole genome-based tests has been expanded. With these additions, Variantyx becomes one of the first laboratories to offer testing for two recently characterized ataxia variants: the biallelic AAGGG expansion in RFC1 that causes cerebellar ataxia, neuropathy, vestibular areflexia syndrome (CANVAS) and the monoallelic, deep intronic GAA expansion in FGF14 that causes spinocerebellar ataxia 27B (SCA27B).
August 1, 2023
PureTech Health announced that the company has been awarded up to $11.4 million from the U.S. Department of Defense (DoD) to advance its therapeutic candidate, LYT-300 (oral allopregnanolone), for the treatment of Fragile X-associated Tremor Ataxia Syndrome (FXTAS). The funds will support a Phase 2 trial of LYT-300 in collaboration with the University of California, Davis (UC Davis).
Here is an SCAsource article that might be helpful in understanding the information in the press release above:
July 28, 2023
Biogen Inc. and Reata Pharmaceuticals, Inc. announced the companies have entered into a definitive agreement under which Biogen has agreed to acquire Reata for $172.50 per share in cash, reflecting an enterprise value of approximately $7.3 billion.
Reata has made significant advancements developing therapeutics that regulate cellular metabolism and inflammation in serious neurologic diseases. Reata’s FDA-approved SKYCLARYS® (omaveloxolone) is the first and only approved treatment for Friedreich’s ataxia (FA) in the United States, with a commercial launch underway, and European regulatory review ongoing. In addition, Reata is developing a portfolio of innovative products for a range of neurological diseases.
July 27, 2023
Biohaven Ltd. today announced multiple updates on key programs including a regulatory update on troriluzole. The FDA informed Biohaven that it would not review the recently submitted NDA application for troriluzole given that the study’s primary endpoint was not met and thus, would not permit a substantive review. The communication from the FDA indicated that the Company may request a Type A meeting within 30 days. Biohaven is committed to working closely with the FDA to bring troriluzole to people with SCA3 as quickly as possible given no therapy is currently approved for this ultra-rare genetic disorder and is requesting a Type A meeting to comprehensively address FDA’s concerns cited in the refusal to file letter. Any updates regarding the Type A meeting will be provided subsequent to the upcoming regulatory interaction.
Comment from NAF:
While NAF is disappointed to learn of the FDA’s decision to not review the new drug application for troriluzole, we are confident that FDA will follow established regulatory processes and precedent in determining next steps for the NDA. We look forward to learning more after the Type A meeting and will keep the Ataxia community informed of updates.
NAF’s Chief Scientific Officer Lauren Moore said, “It can be difficult to remain hopeful following setbacks in the drug development process. Having multiple family members affected by Ataxia, I understand the immense need for effective treatments NOW. While we wait to hear about what the future holds for troriluzole following this decision, I take strength in the momentum created by the unprecedented number of companies investing in Ataxia drug development.”
NAF will continue to represent the voice of the patient throughout the regulatory process and advocate for the needs of the Ataxia community.
July 25, 2023
Larimar Therapeutics announced that the FDA has cleared the Company’s four-week, placebo-controlled, Phase 2 dose exploration trial of CTI-1601 in patients with Friedreich’s ataxia (FA) to proceed to a 50 mg cohort in which participants will be dosed daily for the first 14 days, and then every other day until day 28. In addition, Larimar’s open label extension (OLE) trial was also cleared for initiation by the FDA. Participants in the OLE will receive 25 mg of CTI-1601 daily. CTI-1601 is a novel protein replacement therapy designed to deliver frataxin to the mitochondria of patients with FA who have low levels of frataxin.
Here is an SCAsource article that might be helpful in understanding the information in the press release above:
June 27, 2023
Reata Pharmaceuticals (RETA) announced that the United States Food and Drug Administration (FDA) has approved the Prior Approval Supplement (PAS) to update the drug substance specification for SKYCLARYS® (omaveloxolone), the first and only FDA approved drug for the treatment of Friedreich’s Ataxia in adults and adolescents aged 16 years and older. With the approval of the PAS, SKYCLARYS is now available to patients with Friedreich’s Ataxia in the United States.
Here is an SCAsource article that might be helpful in understanding the information in the press release above:
June 13, 2023
LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease, announced today the completion of the first dose cohort and the dosing of the first patient in the second dose cohort in SUNRISE-FA, a Phase 1/2 clinical trial of LX2006 in patients with Friedreich’s ataxia (FA) cardiomyopathy.
In the first dose cohort, LX2006 has been well tolerated with no unexpected events or toxicities observed. Following the Data Safety Monitoring Board recommendation to proceed, investigators have initiated dosing in the second cohort.
Here is an SCAsource article that might be helpful in understanding the information in the press release above:
May 31, 2023
Biohaven Ltd. (NYSE: BHVN) (“Biohaven”), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies for people with debilitating diseases, including ultra-rare disorders, will provide an overview of clinical progress, regulatory updates, and pipeline developments at an in-person R&D Day today, concurrently with the Yale Ventures’ Innovation Summit 2023 taking place on May 31-June 1.
The clinical progress, regulatory updates, and pipeline developments included:
Troriluzole in SCA: Submitted New Drug Application (NDA) to the U.S. FDA for troriluzole for the treatment of spinocerebellar ataxia type 3 (SCA3), an ultra-rare, genetically-defined, neurodegenerative disease associated with progressive disability, frequent falls, loss of ambulation, speech and swallowing impairment, and premature death that is the most common SCA genotype worldwide.
Here is an SCAsource article that might be helpful in understanding the information in the press release above:
May 23, 2023
PTC Therapeutics, Inc. (NASDAQ: PTCT) today reported topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia. The study did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population. However, vatiquinone treatment did demonstrate significant benefit on key disease subscales and secondary endpoints. In addition, in the population of subjects that completed the study protocol, significance was reached in the mFARS endpoint and several secondary endpoints.
May 15, 2023
Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced preliminary top-line data from the 25 mg cohort of its Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in participants with Friedreich’s ataxia (FA). Participants in the trial’s 25 mg cohort (n=13) were randomized to receive subcutaneous injections of 25 mg CTI-1601 (n=9) or placebo (n=4) daily for 14 days and then every-other-day thereafter until day 28. Data from the cohort indicate CTI-1601 was generally well tolerated and showed increases in frataxin (FXN) levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells) at day 14.
May 10, 2023
In early March 2023, Reata deployed their fully trained and experienced field sales team as well as the infrastructure that enables physicians to initiate start forms for FA patients. They have received approximately 500 patient start forms to date.
Reata has completed the final stages of SKYCLARYS drug product manufacturing and packaging. An NDA supplement was submitted to the FDA to increase the drug substance specification for a process impurity that was observed above the current specification during process validation. The approval of this supplement is required to release SKYCLARYS drug product to Reata’s specialty pharmacy. The FDA is reviewing the supplement as a Prior Approval Supplement under Priority Review with an approval target action date in mid-August 2023. The FDA stated that the review of the supplement was prioritized and that the approval potentially should come earlier than the action date. Reata believes that the information provided in the NDA supplement, which was developed based upon FDA and international harmonized guidance, is sufficient to support the proposed change. Provided that there are no major review issues with the supplement, Reata believes that SKYCLARYS will be available through the specialty pharmacy no later than mid-August 2023.
May 9, 2023
Design Therapeutics is evaluating its lead GeneTAC™ small molecule, DT-216, in an ongoing Phase 1 MAD clinical trial designed to evaluate the safety, tolerability, pharmacokinetic, biodistribution, and pharmacodynamic effects of three weekly doses of DT-216 in adults with FA. An unanticipated vendor issue related to the study drug vial stopper caused a short delay in product supply, which has been resolved. Design now anticipates presenting initial results from the MAD trial in the third quarter of 2023. The company plans to initiate a Phase 2 trial in the second half of 2023.
April 25, 2023
Biogen announced their decision to stop the MERA study for Spinocerebellar Ataxia type 3. The decision was made after careful assessment of the nonclinical safety data, clinical pharmacodynamic data, and future development of BIIB132.
Dr. Rosalind Chuang said, “This is the not the outcome we had hoped for and we acknowledge that this news may be disappointing to SCA3 patients and the entire Ataxia community. We greatly appreciate the dedication and support by patients, their families, and patient advocacy. Biogen remains committed to Ataxia research.”
April 3, 2023 – Leiden, Netherlands
Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced that the first patient has been dosed in a Phase 1/2a clinical study evaluating VO659 for the treatment of Huntington’s disease (HD), spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3). VO659 is an antisense oligonucleotide (ASO) investigational therapy designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases including HD, SCA1 and SCA3. The Phase 1/2a clinical trial is a multi-center, open-label basket study designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with early manifest HD or mild to moderate SCA1 or SCA3. The study is expected to enroll approximately 71 participants at locations in Europe.
For more information, read the press release.
Here are SCAsource articles that might be helpful in understanding the information in the press release above:
April 3, 2023
Seelos Therapeutics, Inc. (Nasdaq: SEEL) (“Seelos”), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced they are temporarily pausing additional enrollment of patients in the SLS-005-302 study in spinocerebellar ataxia type 3 (SCA3). Patients already enrolled will continue in the study and data will continue to be collected in order to make decisions for resuming enrollment in the future. This temporary pause has been implemented as a business decision due to financial considerations, and is not based on any data related to safety or therapeutic effects.
For more information, read the press release.
February 28, 2023
Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a biopharmaceutical company focused on developing and commercializing novel therapies for patients with severe diseases, announced that the U.S. Food and Drug Administration (“FDA”) has approved SKYCLARYS™ (omaveloxolone) for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. With this approval, the FDA granted a rare pediatric disease priority review voucher.
For more information, read the press release.
For information about SKYCLARYS, visit their patient website at www.SKYCLARYS.com.
October 27, 2022
LEXEO Therapeutics (LEXEO), a clinical-stage biotech company advancing a pipeline of adeno-associated virus (AAV)-based gene therapy candidates for cardiovascular and central nervous system (CNS) diseases, today announced it will be presenting clinical and preclinical data at three upcoming scientific meetings. LEXEO will present at the inaugural International Congress for Ataxia Research (ICAR) held live in Dallas, Texas from November 1-4, 2022, the American Heart Association Scientific Sessions held live in Chicago, Illinois and virtually from November 5-7, 2022, and the Clinical Trials for Alzheimer’s Disease (CTAD) conference held live in San Francisco, California from November 29-December 2, 2022.
For more information, read the press release.
For information about ICAR, visit www.ataxiacongress.org.
October 25, 2022
Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced dosing of the first patient in a registrational phase II/III study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of spinocerebellar ataxia focusing on participants with type3 (SCA3, also known as Machado-Joseph disease) and also announced that it will participate in the International Congress for Ataxia Research (ICAR) in Dallas, Texas November 1st – 4th, 2022.
For more information, read the press release.
If you have SCA3 and are interested in participating in this trial, read the recruitment information.
May 26, 2022
Reata Pharmaceuticals announced today the FDA have granted their Omaveloxolone application ‘Priority Review’ designation. This means the FDA will review their application for regulatory approval within 8 months rather than the usual 12 months. Omaveloxolone is a drug being tested for the treatment of FA.
For more information, read the press release.
May 23, 2022
Biohaven Pharmaceuticals reported top-line results of its phase 3 clinical trial of troriluzole, an investigational drug for patients with SCA. While we share in your disappointment that the drug did not meet its primary endpoint in the overall SCA population, there is much more to this story than just that headline. Further analysis by SCA type suggests a positive treatment effect in patients with SCA3, the most common form of SCA. There was also a reduction in the relative risk of falls in troriluzole-treated patients with all types of SCA included in the trial versus the placebo group.
We are pleased that Biohaven will be presenting this data to the FDA as soon as possible. “The f-SARA change in the SCA3 troriluzole treated group is compelling,” stated Dr. Susan Perlman, Director of Ataxia Clinic and Neurogenetics Clinical Trials at the David Geffen School of Medicine at UCLA. “Given the excellent safety profile of troriluzole, and the fact that there are no approved treatment options for patients with this devastating neurodegenerative disorder, I am thrilled for my patients that Biohaven is planning to engage with the FDA to potentially move this program forward.”
For more information, read the press release.
May 10, 2022
Reata Pharmaceuticals recently announced that the FDA has granted Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation to omaveloxolone for the treatment of Friedreich’s Ataxia.
For more information, read the press release.
November 8, 2021
Seelos Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today that the FDA has accepted Seelos’ Investigation New Drug (IND) application to study SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of Spinocerebellar Ataxia (SCA). The FDA has also granted the program Fast Track designation.
For more information, read the press release.
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