PTC to ask for FDA approval of vatiquinone for FA by year’s end
Oral therapy has been tested in both children and adults in MOVE-FA trial
PTC Therapeutics says it’s planning, this December, to ask the U.S. Food and Drug Administration (FDA) to approve its oral therapy vatiquinone — which has been tested in pediatric patients as well as adults — as a treatment for Friedreich’s ataxia (FA).
“We look forward to submitting the NDA [new drug application] by the end of the year,” Matthew B. Klein, MD, PTC’s CEO, said in a company press release.
The company said it has aligned with the FDA on its plans for the submission — which will be based in large part on positive results from the Phase 2/3 MOVE-FA clinical trial (NCT04577352), completed late last year.
MOVE-FA enrolled 146 FA adults and children, ages 7 and older, who were able to walk at least 10 feet in one minute, with or without assistance. The participants were randomly assigned to take vatiquinone thrice daily, at a dose based on weight, or a placebo for about 1.5 years.
The study had hoped to show that vatiquinone could slow disease progression, as measured by a standardized assessment called the modified Friedreich Ataxia Rating Scale, or mFARS. However, the results failed to show a significant slowing in mFARS decline with the therapy relative to the placebo, missing the study’s main goal.
But despite the negative primary results, some promising findings were seen in MOVE-FA. Importantly, vatiquinone significantly outperformed the placebo at slowing decline in the upright stability subscale of the mFARS. This subscale assesses a person’s ability to balance upright, and, according to PTC, it is “the most relevant and sensitive mFARS component for [children] and young adults.”
Such benefits in the part of the mFARS with the greatest relevance for pediatric patients are particularly noteworthy because Skyclarys (omaveloxolone), the first and so far only FA treatment to win FDA approval, is authorized only for patients ages 16 and older.
Vatiquinone submission to be based largely on positive MOVE-FA data
After the placebo-controlled part of the MOVE-FA trial, participants had the option to enter an extension study in which all were being given long-term vatiquinone treatment.
PTC has now announced results from a prespecified analysis comparing data from the extension study against a large natural history study known as FACOMS, fully the FA Clinical Outcome Measures Study. The data from the extension study covered almost three years of treatment with vatiquinone.
The results showed that the decline in total mFARS scores was about 50% slower for patients on vatiquinone than untreated patients in the natural history study, with a 3.7-point benefit. In contrast, mFARS scores typically worsen by about two points per year in untreated FA.
“MOVE-FA was a well-conducted international clinical trial in children and adults with Friedreich’s ataxia. Both this trial data and the open-label extension data are compelling with positive results in clinical endpoints that are meaningful to the FA community,” said Jennifer Farmer, CEO of the Friedreich’s Ataxia Research Alliance (FARA). “We are also encouraged that vatiquinone treatment continues to be safe and well-tolerated.”
Data from an earlier open-label study of the experimental therapy showed a similar benefit of 4.8 points when compared with findings from a patient group given no treatment.
According to Klein, “the results of the extension studies provide further evidence of the potential benefit of vatiquinone in slowing disease progression.”
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.
Safety data also were positive, with no serious side effects related to vatiquinone reported.
“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 said.
PTC’s planned application to the FDA will also include data from these extension studies, as well as main findings from MOVE-FA.
The news of the planned submission was welcomed by FARA.
“Given the high unmet need, especially in the pediatric population, we are excited that PTC Therapeutics is submitting an NDA,” Farmer said.
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