The FDA has accepted the New Drug Application (NDA) for vatiquinone, submitted by PTC Therapeutics, for the treatment of Friedreich's ataxia (FA) in both children and adults. The FDA granted the application priority review, setting a Prescription Drug User Fee Act (PDUFA) target action date of August 19, 2025.
Vatiquinone: A Potential Breakthrough for FA Patients
Vatiquinone, a first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), represents a novel approach to treating FA. 15-LO is an enzyme that regulates energetic and oxidative stress pathways disrupted in FA. By inhibiting 15-LO, vatiquinone aims to mitigate mitochondrial dysfunction, reduce oxidative stress, and promote neuronal survival.
Matthew B. Klein, MD, CEO of PTC Therapeutics, stated, "We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich's ataxia. If approved, vatiquinone would be the first therapy for pediatric patients with FA and provide a potential safe, well-tolerated, and effective treatment alternative for adults. The granting of priority review by FDA reflects the significant unmet need for younger patients with FA. We look forward to working collaboratively with FDA during the review process."
Supporting Clinical Data
The NDA submission is based on data from the placebo-controlled MOVE-FA study (NCT04577352) and two long-term studies involving pediatric and adult FA patients. These studies provide evidence that vatiquinone slows disease progression and is safe and well-tolerated across all age groups.
The MOVE-FA trial enrolled 146 FA patients aged 7 years and older. While the primary endpoint of change from baseline in the overall mFARS score did not reach statistical significance (p=0.14), a statistically significant effect (p=0.021) was recorded on the mFARS upright stability subscale. A consistent vatiquinone treatment benefit was observed across primary, secondary, and exploratory endpoints. Notably, nominally significant benefits were recorded in the Upright Stability subscale (USS) of mFARS (-1.26 [p = 0.021]), a key metric for disease progression in younger, ambulatory FA patients, and the Modified Fatigue Impact Scale (MFIS), -5.05 (p = 0.025). Vatiquinone was well tolerated, with no difference in treatment-related adverse events between treatment and placebo groups.
Addressing an Unmet Need in Friedreich's Ataxia
Friedreich's ataxia is a rare, inherited neuromuscular disorder affecting approximately 25,000 people worldwide. It is characterized by progressive loss of coordination and muscle strength, leading to impaired balance, difficulty speaking and swallowing, and heart conditions. Currently, there is no approved treatment that directly targets disease progression.
If approved, vatiquinone could represent a significant advancement in FA treatment, particularly for pediatric patients who currently lack approved therapeutic options.
