ROAR Outstanding Innovation Award to PTC Therapeutics for Translarna

by Margarida Azevedo, MSc | November 20, 2014

Biopharmaceutical company PTC Therapeutics was honored with the Outstanding Innovation Award at The 2014 Rare & Orphan Advocacy & Research Awards. The prize was granted to PTC for their lead therapy Translarna, which has recently become the first treatment for Duchenne muscular dystrophy to be approved in the European Union. The ROAR awards were given during the 5^th Annual World Orphan Drug Congress Europe, which took place between November 12 and 14, in Brussels, Belgium.

The Outstanding Innovation Award was granted to the company in recognition of the innovation and advancements they have brought to treating a rare and orphan disease in terms of research, development, or advocacy. The 2014 ROAR Awards are designed to honor the contributions to rare and orphan diseases, and the achievements over the past year. The winners are chosen by a panel of judges spanning the research, development, and advocacy fields, who give the awards chosen from a peer-nominated list.

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“We are extremely honored to receive the ROAR award for Outstanding Innovation,” said the CEO of PTC Therapeutics, Inc., Stuart Peltz, Ph.D. “This award reflects over a decade and a half of research and development by a dedicated group of scientists and clinicians both at PTC and externally. Translarna would not have been possible without the commitment and constant support from patients and their families, advocacy groups, investigators and all the employees at PTC. We are proud to be recognized for the first drug approved for the underlying cause of DMD. ”

Translarna (ataluren) has been widely accepted worldwide for the treatment of DMD, and has already been granted marketing authorization in the European Economic Area as a therapy for ambulatory patients aged five years or older with nonsense mutation DMD. In addition, the investigational new drug designation has also been granted by the U.S. Food and Drug Administration in the United States. In Italy, the government has not only approved the drug, but also included it in its medication list for reimbursement by the Italian National Health System, making it available in the country, after being advocated by both specialists and DMD families’ associations.

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About the Author

Margarida Azevedo, MSc Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.