PTC Therapeutics (NASDAQ: PTCT) reported Q4 revenue for the quarter came in at $96.46 million versus the consensus estimate of $95.24 million.

"2019 was a year of outstanding execution for PTC. I\'m proud of the progress made in every area of the business," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. "We\'ve significantly expanded our rare disease portfolio, strengthened our global commercial engine and added vital gene therapy manufacturing capabilities, all while delivering on our revenue guidance. The innovation in our diverse, multiplatform pipeline continues to create value for stakeholders."

Key Fourth Quarter and Other Corporate Highlights:

Expanding commercial footprint including continued revenue growth

• Total revenue for the full year 2019 was $307 million. The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna™ (ataluren) and Emflaza® (deflazacort), continues to grow with 2019 revenue of $291 million.
• PTC continues to leverage its strong Latin American infrastructure to support the launch of multiple products. In 2019, Translarna received approval from the Brazilian health regulatory authority (ANVISA) for ambulatory nmDMD patients 5 years and older. In addition, Tegsedi (inotersen) received ANVISA approval for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). In 2019, PTC established an early access program for patients with Familial Chylomicronemia Syndrome (FCS) in Latin America. PTC anticipates filing for approval of Waylivra® (volanesorsen) for the treatment of FCS from ANVISA in 2H 2020.
• Real-world data from the Translarna STRIDE registry demonstrated that boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) treated with Translarna and standard of care, preserved the ability to walk for 3.5 years longer than compared with those in a matched natural history cohort. Importantly, pulmonary function was also preserved in those treated with Translarna.
• A recent publication on Emflaza adds to the growing body of evidence showing its relative benefit and supports the advantage of switching from prednisone to Emflaza. The data in the publication demonstrated that patients treated with Emflaza had better ambulatory function and were able to walk longer, had a lower risk of scoliosis, greater percentage lean body mass and lower weight than patients treated with prednisone.

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