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A pipeline of innovation and hope

Our product pipeline is the best possible expression of who we are, what we do, and what we believe in. It showcases the many incredible ways we’re working to serve patients in need.
PTC Pipeline - Feb 28, 2024
PKU, phenylketonuria; FA, Friedreich ataxia; ALS, amyotrophic lateral sclerosis; HD, Huntington‘s disease; SCA-3, spinocerebellar ataxia type 3; MAP-tau, microtubule associated protein tau.

Approved Medicines

To serve the rare disease community, we’ve worked tirelessly to produce treatments for multiple rare diseases. We also have a robust pipeline of product candidates to expand our impact on rare diseases and provide more moments for patients and their families.

deflazacort

EMFLAZA®

EMFLAZA® (deflazacort) is approved in the US for the treatment of Duchenne muscular dystrophy in patients 2 years of age and older.

Learn more at www.emflaza.com
For medical information, product complaints, or to report an adverse event, please call 1‑866‑562‑4620.
You may report adverse events to FDA at 1‑800‑FDA‑1088 or www.fda.gov/medwatch

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Risdiplam

EVRYSDI®

EVRYSDI® (risdiplam) is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. EVRYSDI is designed to distribute evenly to all parts of the body, including the central nervous system (CNS). EVRYSDI is administered daily at home in liquid form by mouth or feeding tube. The U.S. Food and Drug Administration approved EVRYSDI for the treatment of SMA for adults and children 2 months and older on August 7, 2020 and the European Medicines Agency approved EVRYSDI on March 30, 2021 for the treatment of 5q SMA in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. EVRYSDI is marketed in the United States by Genentech, a member of the Roche Group. EVRYSDI is marketed by Roche outside of the United States.

EVRYSDI is a product from the SMA collaboration between PTC, the SMA Foundation, and Roche.

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inotersen

TEGSEDI®

TEGSEDI® (inotersen) was granted marketing approval from the Brazilian Health Regulatory Agency (ANVISA) for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR), to delay disease progression and improve quality of life. PTC has licensed the commercialization rights for TEGSEDI (inotersen) in Latin America from Akcea Therapeutics, now Ionis Pharmaceuticals. Ionis will commercialize TEGSEDI (inotersen) outside of Latin American. TEGSEDI is also approved in the United States, Canada and the European Union for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults.

Important clarification regarding Tegsedi in LATAM (PDF)

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ataluren

TRANSLARNA™

TRANSLARNA™ (ataluren)* has been approved in the European Union and Brazil for ambulatory patients aged 2 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene. *TRANSLARNA is an investigational new drug in the US

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eladocagene exuparvovec

UPSTAZA™

UPSTAZA™ (eladocagene exuparvovec) has been approved in the European Union, Great Britain, and Israel for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older.

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volanesorsen

WAYLIVRA®

PTC has gained the commercialization rights for WAYLIVRA® (volanesorsen) in Latin America. Ionis Pharmaceuticals will commercialize WAYLIVRA outside of Latin America. WAYLIVRA is approved in the European Union for the treatment of familial chylomicronemia syndrome (FCS).

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U.S. Expanded Access Policy

At PTC we are committed to providing novel treatment options to patients living with serious diseases. To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines, which may allow us to obtain the necessary regulatory approvals and provide patients with broader access to these medicines.

Science
Our Science

Leveraging our expertise and cutting-edge biotechnology platforms, we’re urgently turning the latest scientific advances into purposeful treatments

Therapeutic Areas
Therapeutic Areas

Diseases we’re on a mission to change

Clinical Trials
Clinical Trials

We're committed to making progress in rare disease through clinical trial research, each and every moment of every day