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PTC Therapeutics Announces Waylivra™ Approval in Brazil as First Treatment for Familial Chylomicronemia Syndrome
- Approval Addresses Unmet Needs of Patients with No Other Treatment Options - SOUTH PLAINFIELD, N.J., Aug. 23, 2021 /PRNewswire/ -- PTC Therapeutics, Inc.
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Approval Addresses Unmet Needs of Patients with No Other Treatment Options -\n\n\nSOUTH PLAINFIELD, N.J., Aug. 23, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil. FCS 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.\n\n \n \n \n \n \n \n\n \n\"We are excited to announce Waylivra's approval in Brazil as the first treatment for the underlying cause of FCS,\" said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. \"This approval is another example of PTC leveraging its strong capabilities in Latin America to bring transformational therapies to patients with high unmet needs.\"\n\n ANVISA's approval of Waylivra is based on results from the Phase 3 APPROACH study and the APPROACH Open-Label Extension study and is supported by results from the Phase 3 COMPASS study. Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that treatment with Waylivra reduced triglycerides 94% when compared to placebo. All patients in the trial maintained a low-fat diet.\n\n Waylivra is a self-administered, subcutaneous injection that comes in a single-use, prefilled syringe and is the first approved pharmacotherapy indicated for the treatment of FCS. It is an innovative antisense oligonucleotide (ASO), with a novel mode of action designed to reduce the production of apolipoprotein C-III (Apoc-III), a protein that slows down the breakdown of fats called apolipoprotein C-III. By blocking the production of this protein, the medicine reduces the level of triglycerides in the blood and, as a result, fat accumulation in the body, which is expected to reduce the risk of pancreatitis.\n\n \n About Waylivra\n Waylivra (volanesorsen), a product of Ionis Pharmaceuticals, Inc.'s proprietary antisense technology, has received conditional marketing approval in the E.U. as a treatment for FCS. In addition, Waylivra has been granted Orphan Drug Designation by the European Medicines Agency for the treatment of FCS. Waylivra has been in-license...