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PTC Therapeutics Reports First Quarter 2019 Financial Results and Provides a Corporate Update
SOUTH PLAINFIELD, N.J., May 2, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"SOUTH PLAINFIELD, N.J., May 2, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the first quarter ending March 31, 2019. \n\"We believe we are making great strides towards our vision of having many more treatments for patients of rare disorders,\" said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. \"The approval of Translarna expands our footprint in Brazil. We are now preparing for the launches for multiple products, including Tegsedi for hATTR and Waylivra for FCS in Latin America. These differentiated therapies for rare disorders have the potential to be part of growing our revenue base to $1.5 billion by 2023.\" \nKey First Quarter and Other Corporate Highlights:\nExpanding commercial platform\nTranslarna received approval from the Brazilian health regulatory authority (ANVISA) which will allow for expanded market access. Waylivra™ received a positive CHMP opinion from the European medical authorities in the first quarter 2019 and will be referred to the European Commission for consideration. After ratification by the European Commission, PTC plans to initiate early access programs in Latin America in 2019. Tegsedi™ application submitted to the Brazilian health regulatory authority (ANVISA) was granted priority review, with expected approval by year end 2019. PTC has initiated early access programs for Tegsedi to support patient diagnosis and monitoring in Latin America. PTC has submitted a sNDA with the FDA for Emflaza® for patients 2 to 5 years old and received an approval action date of July 4, 2019.Advancing gene therapy portfolio\nAs previously disclosed, PTC plans to submit a BLA with the FDA in late 2019 followed by an MAA in Europe for the AADC deficiency gene therapy program with anticipated commercial launch in the United States in 2020. Friedreich ataxia program is advancing with an expected IND submission in late 2019 and subsequent entry in the clinic. PTC is in the process of finalizing a long-term lease on an existing biologics manufacturing facility for the gene therapy pipeline. Risdiplam SMA data expected at American Academy of Neurology (AAN)\nAs previously disclosed, based on regulatory interactions an NDA and MAA is planned for the second half of this year with the intention to support a broad label to treat SMA Types 1, 2, &...