For PTC, 2022 was a milestone-reaching, patient-supporting, award-winning, employee-engaging, community-driven year. It was also another year of many firsts. The first approved disease-modifying treatment for AADC deficiency, and the first marketed gene therapy directly infused into the brain, was approved in Europe and the UK. We also had the first global regulatory approval for treatment of familial partial lipodystrophy (FPL). For the first time, we were the proud sponsor of the 2022 IWAS power chair hockey tournament. And we launched the first Emerging Leaders program for PTC employees.

Let’s dive into some of the incredible moments of 2022:


We had several exciting product and pipeline milestones this year, which helped us to reach more patients and drive progress in the study of our investigational treatments. The EC marketing authorization and MHRA authorization of our innovative gene therapy, Upstaza™ (eladocagene exuparvovec) was momentous for patients and the gene therapy community.

The U.S. FDA also approved a label expansion for Evrysdi® (risdiplam), providing treatment to even more babies with spinal muscular atrophy (SMA). Waylivra™ (volanesorsen) received Category 1 classification to treat familial chylomicronemia syndrome (FCS) in Brazil; the drug was also granted approved for FPL in Brazil. We also shared topline results from Study 041 of Translarna™ (ataluren) in patients with nonsense mutation Duchenne muscular dystrophy (DMD).

On the pipeline and clinical development front, we also had a busy year: we initiated the PIVOT-HD study to evaluate our investigational treatment PTC-518 in patients with Huntington’s disease, presented preliminary results of the Phase 1b study of unesbulin in leiomyosarcoma and made significant progress in our clinical trials for PKU, Mitochondrial Disease Associated Seizures (MDAS) and Friedreich ataxia.


Supporting patients is truly at the center of everything we do at PTC, and this year, we achieved this mission through our work with advocacy organizations, providing resources for patients and their families and showcasing patient voices.

We proudly supported the 2022 IWAS Powerchair Hockey World Cup, the most important international competition for powerchair hockey athletes. It was an incredibly inspiring, fast-moving, heart-pumping event, and we congratulate all of the competitors.

To help support patients and their families – whether they are just starting out on their rare disease journey or already well into it – we created the education series Insightful Moments. This year, we also launched My VIBE: My Voice is Inspirational, Brave and Empowering, an audio platform where the rare disease community can share their stories. We recorded patient stories at the myriad advocacy organization events we attended this year.

Once again, we were thrilled to announce recipients of the STRIVE awards, our program that provides grants to non-profit patient advocacy organizations serving the DMD community. This year, grants funded projects focused on formal learning, psychological support, sexuality and skills-based training for people with Duchenne.

We also acknowledged World Duchenne Awareness Day by highlighting inspiring women in the Duchenne community and celebrating the day across the globe. We marked the third annual AADC Deficiency Awareness Day and celebrated this important day together with AADC advocacy organizations. Together with Economist Impact, we commissioned a report highlighting key policies for improving care, patient outcomes and quality of life of patients in Europe. We also joined Screen4Care, an innovative research partnership aimed at shortening the diagnosis journey for people living with rare diseases.


More firsts: We opened offices in Japan and Poland, expanding our global presence to reach more rare disease patients around the world. We celebrated the first class of graduates from the Emerging Leaders Program, a tailor-made program for PTC, with its core mission to create a rich pipeline of future PTC Leaders. We are proud to have achieved a best-in-class retention rate due to our strong and supportive culture. This year, we also launched the second “class” of our Talent Pipeline Program, a one-year, immersive, global fellowship aimed at providing recent graduates real-world experience in the biopharmaceutical industry and related professions.

Community Driven

Our Zug, Switzerland team helped make impactful change for the environment by planting trees. In partnership with OneTree Planted and Almighty Tree this year, 100 oak trees were planted to help combat climate change and prevent biodiversity loss for generations to come.

Our first-ever interactive Corporate Sustainability Report was published. which outlined our efforts and impact in key areas: our patients, our people, our community, our values and our environment.


We couldn’t highlight our achievements without mentioning the several awards our team has received this year. We were selected as the 2022 Biotechnology Innovator of the Year by BioTech Breakthrough. Our Hopewell Manufacturing Center of Excellence was recognized in the New Good Neighbor Awards by the New Jersey Business & Industry Association (NJBIA). PTC was recognized a second time as a Great Place to Work in Brazil and in Italy, we received an award for Excellence of the Year in Innovation and Biopharmaceutical Leadership. We were also a finalist in the Pharma Industry Awards Ireland in two categories, Biotech Company of the Year and Supply Chain Achievement Award. Congratulations to our #OnePTC team for these well-deserved awards!

Finally, we had exciting financial news this year: In October, we announced a strategic financing collaboration with Blackstone Life Sciences that provides up to $1 billion in funding to PTC. This strategic financing will support the acceleration of our robust and diversified pipeline, business development opportunities and general corporate initiatives.

We are so proud of all we’ve accomplished in 2022, and look forward to continuing this momentum in 2023, as we celebrate PTC’s 25th anniversary.