PTC to Expand Product Portfolio in Latin America with WAYLIVRAâ„¢

WAYLIVRA, the first and only therapy for the treatment of familial chylomicronemia syndrome (FCS), has received a positive opinion from the CHMP

SOUTH PLAINFIELD, N.J., March 4, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its partner, Akcea Therapeutics, affiliate of Ionis Pharmaceuticals, received a positive opinion for WAYLIVRA™ by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). PTC plans to initiate activities to bring WAYLIVRA to patients in Latin America following European Commission ratification.

"We are excited that the CHMP gave a positive opinion to WAYLIVRA™," said Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "Patients with FCS have no effective treatment. We, along with Akcea, look forward to bringing WAYLIVRA to those suffering from FCS. We have the team in place to bring WAYLIVRA to people with FCS in Latin America as quickly as possible."

Upon ratification of the positive option of the CHMP by the European Commission, PTC will pay Akcea the balance of the up-front licensing fee. WAYLIVRA was discovered by Ionis Pharmaceuticals and co-developed by Ionis and Akcea.

About PTC Therapeutics, Inc.

PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

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