History
A journey with many milestones
PTC Therapeutics
A Brief History of Innovation
PTC Therapeutics was founded in 1998 focused on leveraging its expertise in RNA biology to discover and develop treatments for patients living with rare diseases.
In the first 25 years, PTC achieved historic breakthroughs in the treatment of several rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy and aromatic l-amino acid decarboxylase (AADC) deficiency.
PTC is looking forward to the next quarter century, utilizing its expanded scientific expertise and strong commercial infrastructure to continue in its mission of developing treatments for more patients living with rare diseases.
2023
- Matthew B. Klein, M.D. appointed Chief Executive Officer
- Statistically significant results from sepiapterin Phase 3 PKU trial
- PTC celebrates its 25th anniversary and 10 years as a public company
2022
- Upstaza™ (eladocagene exuparvovec) for the treatment of AADC deficiency, the first ever gene therapy directly administered into the brain, was approved in Europe and the UK
2021
- Evrysdi® (risdiplam) approved in the EU and Japan for patients with spinal muscular atrophy (SMA)
- Received Gallup’s “Don Clifton Strengths-Based Culture” Award
- Proof of mechanism established for PTC518 demonstrating broad distribution for Huntington’s disease
- Expanded partnership with the SMA Foundation to advance drug discovery and development research in regenerative medicine
2020
- Expanded clinical pipeline to include a treatment for phenylketonuria (PKU)
- Evrysdi® (risdiplam) approved by the FDA for patients with spinal muscular atrophy (SMA)
2019
- Expanded platform technologies with the acquisition of a bio-electron transfer platform to address diseases of oxidative stress
- Announced clinical results for its gene therapy treatment for patients with AADC deficiency that demonstrated sustained improvements
- Announced Real-World Evidence demonstrating Translarna™ (ataluren) slows disease progression in patients with Duchenne muscular dystrophy
2018
- Addition of Tegsedi® (inotersen) and Waylivra® (volanesorsen) expands the product portfolio in Latin America
- Acquired a targeted gene therapy platform targeting rare CNS disorders such as AADC deficiency
2017
- Acquired Emflaza® (deflazacort) for the treatment of Duchenne muscular dystrophy: The only drug approved in the US for all patients living with Duchenne
2014
- Translarna™ (ataluren) is approved in the EU: The first drug in the world to be approved for Duchenne muscular dystrophy
2013
- Successful IPO in the US public markets (NASDAQ: PTCT)
2011
- PTC and the SMA Foundation partner with Roche to continue the development of a treatment for spinal muscular atrophy (SMA)
2010
- Awarded grant from the Wellcome Trust that supported research and development of unesbulin
2006
- Partnered with the SMA Foundation to discover and develop a treatment for spinal muscular atrophy (SMA)
2004
- Initiated the first clinical trials for PTC124, the compound that would become Translarna™ (ataluren), to target the underlying cause of rare disorders caused by a nonsense mutation
1998
- PTC was founded with the mission to discover, develop, and commercialize treatments for patients with rare disorders