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Waylivra™ Receives Innovative Drug Category Pricing to Treat Familial Chylomicronemia Syndrome in Brazil
- Additional application submitted to ANVISA for a new indication to treat familial partial lipodystrophy - SOUTH PLAINFIELD, N.J., Jan. 4, 2022 /PRNewswire/
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Additional application submitted to ANVISA for a new indication to treat familial partial lipodystrophy -\n\n\nSOUTH PLAINFIELD, N.J., Jan. 4, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Waylivra™ (volanesorsen) has successfully received Category 1 classification from Câmara de Regulação do Mercado de Medicamentos - CMED (Drug Market Regulation Chamber) in Brazil. Waylivra is the only treatment for familial chylomicronemia syndrome (FCS) in Brazil. Category 1 classification is given to innovative treatments that provide greater efficacy than current standards of care, and it allows for pricing in line with international markets.\n\n \n \n \n \n \n \n\n \nIn addition, PTC submitted an application to the Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária (ANVISA), for approval of Waylivra for the treatment of familial partial lipodystrophy (FPL). If approved, Waylivra will be the first approved treatment for FPL in Brazil, and this will mark the first approval globally for this indication. The application has been submitted under the Rare Disease pathway RDC205/2017. PTC anticipates a decision from the regulatory authorities in the second half of 2022.\n\"Both FCS and FPL are rare genetic diseases that cause significant issues for patients suffering from these disorders,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. \"I am pleased with the decision by CMED to grant FCS patients access to Waylivra in Brazil. This innovative treatment will have a positive impact on patients' lives. I am also very enthusiastic about the possibility of bringing Waylivra to patients suffering from FPL who are also in need of a treatment.\"\nFCS is a rare genetic disease which results in significant disease burden to patients, including potentially fatal pancreatitis and chronic complications due to permanent organ damage. FPL is a rare genetic, highly burdensome disease, which is both visually and physically distressing for the patient. It leads to significant metabolic complications that are not managed by current therapies.\nAbout Waylivra™Waylivra (volanesorsen), a product of Ionis Pharmaceuticals, Inc.'s proprietary antisense technology, has received conditional marketing approval in the European Union as a treatment for FCS. In addition, Waylivra has been g...