PTC Therapeutics is a pharmaceutical company developing treatments for a number of conditions across metabolic and neurological conditions including Friedreich’s ataxia (FA).
The article below starts with the first update from 2023, with newer updates below
PTC Therapeutics have announced the initial results of their Phase III MOVE-FA trial of vatiquinone in people with FA. Vatiquinone targets an enzyme called 15-lipoxygenase, which is a regulator of energetic and oxidative stress pathways that are disrupted in FA. Although the trial did not reach its primary endpoint, which is the main measurement to show whether the drug works, PTC Therapeutics did observe meaningful clinical benefit in some of the trial’s other endpoints. Therefore, they plan to discuss a potential path forward with the drug regulators in the US and EU.
The trial enrolled 146 children and adults with FA. Participants received either vatiquinone or a placebo three times a day for 72 weeks. The primary endpoint was a statistically significant change in the overall mFARS score at 72 weeks in participants aged 7-21 years (primary analysis population, 123 people). The mFARS is a clinical rating scale which measures FA progression and is made up of four components. These are: bulbar function (which assesses coughing and speech), upper limb coordination (which assesses tremor and coordination of arms and hands), lower limb coordination (which assesses coordination of legs and feet), and upright stability (which assesses ability to stand and walk).
Although the study did not achieve its primary endpoint as there was not a statistically significant change in the overall mFARS score, PTC Therapeutics did observe meaningful clinical benefit in some components of mFARS. There was a slowing of progression in both bulbar and upright stability components at 72 weeks. There were also improvements in the impact of fatigue with vatiquinone treatment compared to placebo.
As the trial was conducted during the Covid-19 pandemic, there was some disruption in how the trial could be conducted in a number of participants. Because of this PTC also analysed the data to only include participants who completed the trial on the assigned therapy without disruption. When analysing this population (called the primary analysis population completers), vatiquinone showed a greater effect on FA progression, with the drug slowing disease progression by 75%.
As there is evidence of meaningful clinical benefit and a high unmet need for a treatment for children with FA, PTC Therapeutics plan to discuss a potential path to try and get the drug approved with the drug regulators in the US and EU.
In addition to their trial results, PTC Therapeutics announced that they are discontinuing their preclinical and early research programmes in gene therapy. This includes their preclinical gene therapy programme for FA. This decision was taken as part of their strategic portfolio prioritisation.
For more information, read the full press releases here and here.
Updates October 2023
Following results from their MOVE-FA phase 3 clinical trial of the drug vatiquinone in children and adults with Friedreich’s Ataxia (FA), the pharmaceutical company PTC Therapeutics wrote to the US regulatory body the Food and Drug Administration (FDA) to determine whether the data from the study would be sufficient to support an application for New Drug Application (NDA) for accelerated approval.
The study did not meet its primary endpoint, the main measurement to show whether the drug works, as there was not a statistically significant change in the overall mFARS score (a scale that measures FA progression). However, PTC Therapeutics did observe meaningful clinical benefit in some components of mFARS, including a slowing of progression in both bulbar stability (relating to muscles in the head and neck for swallowing and coughing) and upright stability components at 72 weeks. There were also improvements in the impact of fatigue with vatiquinone treatment compared to those who were not given vatiquinone.
In their written response, the FDA stated that while they see the value of upright stability as a clinically meaningful endpoint, they believed a confirmatory study would likely be needed to support NDA submission. PTC has requested a follow-up live meeting to address the issues raised by the FDA.
PTC is participating in a scientific advice procedure with the EU regulatory body, the European Medicines Agency (EMA), to determine if the MOVE-FA data could support a conditional marketing authorisation application in the European Economic Area (EEA). PTC expects to have the outcome of this procedure in the first quarter of 2024.
You can read the PTC Therapeutics press release here and more about the trial results here.
Update February 2024
In February, PTC announced that they expect a potential NDA submission for vatiquinone for FA in late 2024. PTC had a meeting with the FDA in the first quarter of 2024 to discuss the vatiquinone program for FA, and based on discussions with the FDA has a potential path to an NDA submission in late 2024 based on results of the placebo-controlled MOVE-FA trial, along with data from the ongoing open-label extension study.
Read the press release from PTC Therapeutics here.
On 8th October 2024, the pharmaceutical company PTC Therapeutics announced positive results from their phase III trial of their drug vatiquinone for adults and children with Friedreich’s ataxia (FA).
Analysis of their MOVE-FA long-term study demonstrated that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit on the modified Friedreich Ataxia Rating Scale (mFARS) relative to untreated individuals with FA from a natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Measures) disease registry.
This treatment difference represents a 50% slowing in disease progression over 3 years. PTC plan to submit a new drug application to the FDA in the US in December 2024. Read their press release here.
You can read our previous article on the trial here.
Updates December 2024
On 19th December 2024, PTC Therapeutics announced they have submitted a New Drug Application to the FDA (the US drug regulators), for vatiquinone as a treatment for FA in children and adults.
This submission is based on data from the MOVE-FA study and two long term studies in children and adults with FA.
Read their press release here.
Update February 2025
On 19th February 2025, PTC Therapeutics announced that the FDA has accepted their New Drug Application for Vatiquinone, to treat adults and children with Friedreich’s ataxia. The FDA will now review Vatiquinone with a priority review, with a decision expected in August 2025.
Read their press release here.
Update August 2025
PTC announced the disappointing news that the US drug regulators, the FDA, have not approved vatiquinone to treat adults and children with FA. The FDA has said that substantial evidence of the drug’s effectiveness was not demonstrated, and an additional clinical trial is needed.
PTC plans to meet with the FDA to discuss next steps. We will share any updates with the community through our channels.
Read PTC’s letter to the community here. Read more about vatiquinone here.
Update February 2026
In February, PTC provided an update via press release. This update explained that in December 2025, as planned, they had a meeting with the FDA to discuss next steps. The FDA indicated to PTC Therapeutics that an additional study is needed to support them resubmitting an NDA (New Drug Application) for vatiquinone, and that this could be an open-label study with a natural history control group. To read the press release click here.
Updates May 2026
A meeting with FDA (called a Type C meeting) was held in April 2026 to discuss a vatiquinone study to support NDA (New Drug Application) resubmission for the Friedreich’s ataxia programme. Based on the meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history control in Q3 2026. The primary endpoint will be change in mFARS from baseline to 24 months.
Read about the trial here. Read the press release here. On 29th May PTC published a letter to the FA community regarding the plans for the initiation of the new trial which you can read here.
Updates August 2026
PTC’s phase III PROVE-FA trial of the long-term efficacy of vatiquinone in people aged 7 – 21 years has now started in the US and a number of countries in Europe. There is not currently a UK trial site. This is a 2-year open-label trial. PTC plan to measure the change in mFARS score from baseline to 24 months.
Read more in their press release here as well as the trial page here.
