A Letter From PPMD’s Pat Furlong

I am thrilled to share that the FDA has officially accepted PTC Therapeutics’ New Drug Application (NDA) resubmission for Translarna (ataluren). This acceptance is an important step, meaning the FDA will now carefully evaluate the data to determine ataluren’s potential for approval. 

According to PTC, the NDA resubmission is based on findings following 72-weeks of Translarna treatment, for which there was significant benefit demonstrated on the key study endpoints of six-minute walk distance (6MWD), NorthStar Ambulatory Assessment, 10-meter walk/run, four-stair climb, and time to 10% worsening of 6MWD. Additionally, the NDA includes findings of significant long-term benefit of Translarna treatment, including a 3.5-year delay in loss of ambulation and a 1.8-year delay in reaching a predicted forced vital capacity of less than 60% a critical threshold of lung function.

PTC has indicated that as this was an NDA resubmission following a complete response letter to the NDA which was filed over protest in 2016, FDA is not obligated to follow PDUFA review timelines. With this, an action date has not been provided. PPMD will share additional information with the community as it becomes available.

We appreciate the FDA’s commitment to conduct a full review of PTC’s NDA and we welcome this opportunity to elevate the voices of clinicians and patients as it relates to ataluren. As we await their decision, we remain hopeful about the potential for a positive outcome that could bring meaningful benefits to individuals and families affected by Duchenne.

As always, we are grateful to the community of patients, families, advocates, and researchers who have worked  tirelessly to support clinical trials and research. Your strength, resilience, and unwavering commitment remain vital in these moments of progress.

Together, we will continue fighting for every future.

Read PTC’s community letter:

Update to the U.S. Duchenne Community:

We are pleased to share that the resubmission of the New Drug Application (NDA) for Translarna (ataluren) for the treatment of boys and young men with nonsense mutation Duchenne muscular dystrophy (nmDMD) has been accepted for review by FDA. This is a significant, positive step towards bringing this important treatment to those living with nmDMD in the United States.

The NDA resubmission is based on the findings of statistically significant benefit demonstrated on several key study endpoints in the overall intent-to-treat (ITT) population (N=359) of the global placebo- controlled trial, Study 041, as well as the findings of significant long-term Translarna treatment benefit in delaying time to loss of ambulation as captured in the STRIDE registry. We believe this evidence of short and long-term benefit, along with the established favorable safety profile of Translarna, supports approval.

The FDA has not provided a timeline for its review of the application due to the circumstances related to the last NDA review in 2016.

We are grateful for your steadfast support, advocacy and passion to advance care for those living with nmDMD and look forward to continued collaboration with the community and FDA. We will continue to keep you informed as we have more information regarding the progress of this application.