PTC Therapeutics Plans FDA Submission for Vatiquinone in Friedreich's Ataxia
核心洞察
PTC Therapeutics plans to submit a New Drug Application (NDA) to the FDA for vatiquinone to treat Friedreich's Ataxia (搜索) by the end of the year.
The submission is supported by positive data from the Phase 2/3 MOVE-FA trial and extension studies, showing a slower decline in mFARS scores compared to untreated patients.
Vatiquinone demonstrated a strong safety profile and potential benefits, particularly in children and adolescents, where there are currently limited approved therapies.
PTC Therapeutics is set to request FDA approval for vatiquinone, an oral therapy for Friedreich's ataxia (搜索) (FA), by the end of this year. The planned New Drug Application (NDA) submission is largely based on positive outcomes from the Phase 2/3 MOVE-FA clinical trial and subsequent extension studies.
MOVE-FA Trial and Extension Data
The MOVE-FA trial involved 146 adults and children (ages 7+) with FA. Although the trial did not meet its primary endpoint of significantly slowing disease progression as measured by the modified Friedreich Ataxia Rating Scale (mFARS), notable benefits were observed in the upright stability subscale, which assesses a person’s ability to balance upright. According to PTC, this subscale is the most relevant and sensitive mFARS component for children and young adults.
Following the placebo-controlled phase of the MOVE-FA trial, participants had the option to enroll in an extension study where all received long-term vatiquinone treatment. Results from a pre-specified analysis comparing data from the extension study against the FA Clinical Outcome Measures Study (FACOMS), a natural history study, indicated that the decline in total mFARS scores was approximately 50% slower for patients on vatiquinone, showing a 3.7-point benefit. Typically, mFARS scores worsen by about two points per year in untreated FA.
Impact and Safety Profile
Jennifer Farmer, CEO of the Friedreich’s Ataxia Research Alliance (FARA), noted that the MOVE-FA trial was well-conducted and the data from the trial and its open-label extension are compelling with positive results in clinical endpoints meaningful to the FA community. She also highlighted that vatiquinone treatment continues to be safe and well-tolerated.
An earlier open-label study of vatiquinone showed a similar benefit of 4.8 points compared to an untreated patient group. According to Matthew B. Klein, MD, PTC’s CEO, the results of the extension studies provide further evidence of the potential benefit of vatiquinone in slowing disease progression. Safety data has been positive, with no serious side effects related to vatiquinone reported.
Implications for Pediatric Patients
The potential approval of vatiquinone is particularly significant for pediatric patients, as Skyclarys (omaveloxolone), the only FDA-approved FA treatment, is authorized only for patients aged 16 and older. "The strong safety profile of vatiquinone positions it to be a potentially meaningful therapy for all Friedreich ataxia patients, particularly children and adolescents for whom there are no approved therapies," Klein stated.
FARA has welcomed the planned submission, emphasizing the high unmet need, especially in the pediatric population.