For PTC, 2021 was about expansion – not just literally, as we grew our global reach and added team members, but what we can do for patients, too, through our awareness efforts and continued dedication to scientific innovation.

Expansion by definition is the act of becoming larger or more extensive. And in fact, we had so much good news in 2021, it was hard for us to pick the highlights to include in this wrap up.

To name a few: We opened new offices and a gene therapy manufacturing facility, and we hired our 1,000th team member. For the first time ever, we have five registrational clinical trials running and had several regulatory milestones across the globe, increasing the number of patients we can help in their rare disease journeys.

We want to share this news of expansion with you – read on to learn more.

Expanding Our Work for Patients

2021 was a hallmark year for achieving our mission of discovering, developing, and commercializing therapies for diseases with high unmet medical needs.

  • PTC has five registrational clinical trials in progress in mitochondrial epilepsy, Friedreich ataxia, phenylketonuria (PKU), COVID-19, and nonsense mutation Duchenne muscular dystrophy (nmDMD).
  • The Phase 1 healthy volunteer study of PTC518 for Huntington’s disease met its objectives. The Phase 2 study is expected to be initiated in 2021.
  • We raised awareness of aromatic L-amino decarboxylase (AADC) deficiency by:
    • Establishing several treatment Centers of Excellence around the world to enable access to treatment for patients.
    • Participating in the AADC Family Network’s second annual AADC Deficiency Awareness Day with a live virtual event, webcast from multiple U.S. locations, with MassBio. First of its kind legislation was enacted in California and New Jersey enabling the share of accurate and timely AADCd rare disease information and inspiration.
    • Publishing and presenting extensive data to increase understanding of the condition and ultimately help advance care for patients with AADCd.
  • Good news from around the world for Translarna™ (ataluren) included access to therapy and approvals in Italy, Finland, The Netherlands, Belarus, Lithuania, and Russia, as well as a label expansion to reach more patients in Brazil. We also announced five-year STRIDE registry data which showed Translarna was seen to slow nmDMD disease progression compared to standard of care alone.
  • WAYLIVRA™ (volanesorsen) was approved as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil. We also treated the first patient with WAYLIVRA in Argentina.
  • TEGSEDI®(inotersen) received Category 1 classification from in Brazil. This classification is given to innovative treatments that provide greater efficacy than current standards of care and allows for pricing in line with international markets.

Rising Up for Our Team

  • The PTC team reached a significant milestone when we hired our 1,000th employee.
  • We launched employee resource groups (ERGs) with the goal of strengthening inclusivity and diversity at work, aspiring to enhance the employee experience and empower our employees to make a difference within internal and external communities.
  • PTC Brazil was certified as a Great Place to Work.

Magnifying Our Science

  • PTC was recognized by EuropaBio, an international industry association that promotes an innovative and dynamic European biotechnology industry, as one of the top companies with a scientific breakthrough in last 25 years.
  • Evrysdi®(risdiplam) received multiple awards that recognize it as one of the most innovative therapies of 2021 and for its life-saving impact. The British Pharmacological Society chose Evrysdi as the 2022 Drug Discovery of the Year award, and it was awarded the 2021 Drug Discovery Prize by the Society for Medicines Research and Pharmaceutical Technology Sector Excellence award.

Growing Our Global Footprint

  • In October, we celebrated the opening of our new, state-of-the-art gene therapy manufacturing facility in Hopewell, NJ with a live-streamed ribbon-cutting ceremony.
    • “Gene therapy holds tremendous promise to treat intractable rare genetic disorders. We have built our capabilities to both perform gene therapy research and to manufacture these biological products. We are proud to have such a talented team and state-of-the-art manufacturing facility here in Hopewell, N.J. that will help expedite bringing these therapies to patients.” –Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics.
  • As of this year, we now have offices in 25 countries and a footprint in 50.
  • PTC Japan became operational in the second quarter of 2021 allowing the company to expand into an important new market. Japan is the second-largest market for specialty pharma and rare disease therapies and offers opportunities to leverage the solid scientific and research foundation of Japan.
  • We expanded our operations, reach and access in South America with the goal of bringing our treatments to patients in this part of the world. We set up and continue to build PTC operations in Mexico while we strengthened our business in the Andean Region, with broader reach in Colombia and Ecuador.
  • In the South Cone Region of Latin American, we bolstered our business in Argentina that had been affected by the COVID-19 pandemic, increased patient access to treatments in Chile, and recently expanded our business to Uruguay.

None of this could have happened without our incredible global team. Thank you, and let’s get ready for 2022!