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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

We’ve worked tirelessly to produce treatments for multiple rare diseases.

EMFLAZA® (deflazacort)

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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EVRYSDI® (risdiplam)

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 approved in the US and by the European Medicines Agency.

Learn more at https://www.evrysdi.com/

Please see full Prescribing Information for additional Important Safety Information.

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

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TEGSEDI® (inotersen)

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). 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 U.S., Canada and the European Union for the treatment of polyneuropathy of hATTR in adults.

Important clarification regarding Tegsedi in LATAM (PDF)

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TRANSLARNA™ (ataluren)

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.

Learn more at https://www.ema.europa.eu/en/medicines/human/EPAR/translarna.

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UPSTAZA™ (eladocagene exuparvovec)

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.

Learn more at https://www.ema.europa.eu/en/medicines/human/EPAR/upstaza.

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WAYLIVRA® (volanesorsen)

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