PTC Therapeutics, Inc.

The US Food and Drug Administration (FDA) has rejected PTC Therapeutics ’ new drug application (NDA) for vatiquinone in Friedreich’s Ataxia . This follows the biotech’s decision to submit the 15-lipoxygenase (15-LO) inhibitor for approval in the indication, despite it failing to meet its primary endpoint in the Phase III MOVE-FA trial (NCT04577352). PTC Therapeutics’ vatiquinone for Friedrich ataxia has been rejected by the FDA. Image credit: Piotr Swat / ShutterStock.com. Hedging its bets, PTC applied for FDA approval based on statistically significant improvements observed in two subscales of the modified Friedreich Ataxia Rating Scale (mFARS) in the Phase III trial. These related to bulbar and upright stability improvements, which the company claimed were “reflective of key aspects of disease morbidity” in a December 2024 statement. However, the FDA issued PTC with a complete response letter, stating that the application did not provide substantial evidence of vatiquinone’s efficacy, and that “an additional adequate and well-controlled study would be needed to support NDA resubmission”. Despite the FDA’s decision, PTC Therapeutics’ CEO Matthew Klein said: “We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich’s Ataxia.” GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence The company plans to meet with the FDA to discuss potential next steps for the drug in this indication. Friedreich’s Ataxia is a rare, inherited disorder that causes progressive damage to the nervous system. This can cause movement and sensory symptoms, as well as trouble with walking and gait. If the FDA were to be convinced of vatiquinone’s efficacy further down the line, it could become the second drug on the market for Friedreich’s Ataxia – joining Biogen’s Skyclarys (omaveloxolone), which was approved in February 2023 . It could also be the first to be authorised for use in a paediatric population, as Skyclarys is only permitted for use in patients aged 16 years or older. According to GlobalData’s Intelligence Center, Skyclarys made $383m in 2024, with analysts predicting that the drug will become a blockbuster in 2029, raking in $1bn for Biogen. GlobalData is the parent company of Pharmaceutical Technology . PTC’s rare disease woes persist Vatiquinone’s FDA rejection is the most recent event in a swathe of disappointing turnouts for the company, as the conditional approval for its nonsense mutation Duchenne muscular dystrophy (DMD) drug Translarna (ataluren) was pulled by the European Medicines Agency (EMA) in January 2024. This follows a back-and-forth between the two between 2023 and 2024, with the regulator’s committee doubling down on its negative stance after PTC submitted further documentation to the EMA, stating that it “could not draw conclusions on the benefits of Translana” from the new data provided. The EMA also cited “differences between the registries and biases making study comparisons inconclusive” as a key reason for the drug’s recent rejection. PTC has had even less luck in its attempts to get Translarna approved in the US, having faced rejections in 2016 and 2017. The first was related to the insufficient completion of the new drug application (NDA) while the FDA cited a lack of clinical evidence as the primary reason for the second application’s rejection. The company has now filed for approval for the third time in the US, with the FDA accepting its application in October 2024. An action date has not yet been provided by the agency. The drug remains available in the UK through the Medicines and Healthcare products Regulatory Agency (MHRA). Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now

The FDA rejected PTC Therapeutics’ Friedreich’s ataxia drug vatiquinone, the company announced Tuesday morning. The agency said that “substantial evidence of efficacy” was not shown for the Friedreich’s ataxia therapy, according to PTC’s press release. An additional “adequate and well-controlled” study would be needed for a resubmission to the FDA. Vatiquinone failed its pivotal study in Friedreich’s ataxia in 2023, but the company pointed to measures that suggested the therapy could improve balance and fatigue in asking the FDA for approval. In Friedreich’s ataxia, patients lack the protein frataxin, which is key for the mitochondria to function properly. Vatiquinone is a small molecule drug that’s designed to address that mitochondrial dysfunction and oxidative stress by blocking an enzyme called 15-lipoxygenase. Currently, the only approved drug for Friedreich’s ataxia is Skyclarys, which the FDA approved in early 2023. Biogen acquired the drug in a $7.3 billion deal for its maker Reata later that year. “We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL,” PTC’s CEO Matthew Klein said in a press statement.

The FDA rejected approval of PTC Therapeutics' vatiquinone for the treatment of children and adults with Friedreich's ataxia (FA). In a complete response letter (CRL), the agency said that substantial evidence of efficacy had not been demonstrated and an additional study would be needed to support a resubmission.CEO Matthew Klein remarked PTC was "of course disappointed" by the FDA's decision, which the company disclosed on Tuesday. "We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for…Friedreich's ataxia."The filing, which was accepted by the FDA and granted priority review with a target action date of August 19, was supported by results from the placebo-controlled MOVE-FA study, as well as findings from two long-term studies including pediatric and adult FA patients. PTC said that it plans to meet with the agency to discuss potential steps to address the issues raised in the CRL.Vatiquinone is a small-molecule selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in FA.