PTC Therapeutics, Inc.

Today the U.S. FDA has accepted our filing of the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia (#FA). The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 19, 2025. “We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich’s ataxia,” said Matthew Klein, MD, MS, FACS. “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.” Learn more: https://bit.ly/4b31Qtf

PTC will announce fourth quarter and full year 2024 financial results and provide an update on the company's business and outlook on Thursday, Feb. 27. Learn more: https://bit.ly/3Qhg145

Our CEO Matthew Klein, MD, MS, FACS was honored to accept the Innovator Award from BioNJ's CEO Debbie Hart for our efforts in gaining FDA approval of Kebilidi, the first-approved gene therapy directly administered to the brain and the only approved therapy for #AADCdeficiency. The award was given at the BioNJ 32nd Annual Dinner Meeting & Celebration, an inspiring event to come together and celebrate our industry’s impact on the future of medicine and our commitment to improving patients’ lives.

We are proud of our long-standing partnership with the Friedreich's Ataxia Research Alliance (FARA), and it's an honor to continue that path as we work to develop a treatment for children and adults living with #FriedreichsAtaxia (FA). Our CEO Matthew Klein, MD, MS, FACS recently sat down with Jen Farmer, CEO of FARA to share the latest updates on vatiquinone, PTC's potential therapy for treating FA based on our inflammation and ferroptosis platform. Hear more in the full recording: https://bit.ly/40SOjko

We're incredibly proud to be named one of BioNJ's 2025 Innovator Award Honorees in recognition of the first and only FDA-approved gene therapy for treating children and adults with #AADCdeficiency.

At this week’s 43rd Annual J.P. Morgan Healthcare Conference, our CEO Matthew Klein, MD, MS, FACS, shared insights on how PTC’s successful execution in 2024 has set the stage for a bright and successful future in 2025 and beyond. Hear the replay for the full story: https://lnkd.in/eB-X55ar

Today we provided an update on the company's progress and outlook for 2025 at the J.P. Morgan Healthcare Conference. "I am very proud of our team’s performance across every part of the company in 2024," said Matthew Klein, CEO. "We met every one of our planned clinical and regulatory milestones on schedule. Commercially, we outperformed guidance. And we begin 2025 with a strong cash balance, ready to build on our successes. PTC, now stronger and more innovative than ever, will continue to work to bring transformative therapies to the patient communities we serve in 2025 and beyond.” Learn more: https://bit.ly/426hS30 #JPM25

Today, we announced our submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA). Learn more: https://bit.ly/4gzoGdC

Supporting the next generation of talent and leaders in the life sciences industry is important to us at PTC. Since 2020, our Talent & Pipeline Program (TPP) has given recent grads the opportunity to participate in a year-long fellowship program that provides comprehensive training and real-world work experience across a range of functions. We're thrilled to launch a new cohort for 2025 and look forward to helping individuals make the next meaningful step in their careers. 👉 Click the link to learn more and apply: https://bit.ly/4gHhHiI

PTC was excited to learn, share and collaborate with other leading RNA experts at the recent 7th Annual RNA-Targeted Drug Discovery & Development Summit in Boston, MA, and the RNA at the Bench and Bedside IV conference in La Jolla, CA. Anu Bhattacharyya, Kerstin Effenberger, and Marla Weetall from our research team presented on ways PTC is leveraging its leading splicing platform to identify promising therapies for rare diseases with high unmet need. Learn more about our splicing platform here: https://bit.ly/4fi4w6L