SOUTH 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.
SOUTH 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.
LONDON--(BUSINESS WIRE)--Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the Scottish Medicines Consortium (SMC) has enabled access to volanesorsen on the National Health Service (NHS) in Scotland for the treatment of familial chylomicronaemia syndrome (FCS).1 This decision will allow patients in Scotland with this rare and life-threatening condition to access the treatment through NHS Scotland. SMC’s acceptance follows recent approval from the National Institute for Health and Care Excellence (NICE) for the use of volanesorsen on the NHS in England.2
Patients with the rare disease familial chylomicronemia syndrome (FCS) will be able to access treatment with Akcea’s Waylivra via the NHS, after a U-turn by NICE.
Akcea Therapeutics UK's Waylivra (volanesorsen), the first and only therapy for Familial Chylomicronaemia Syndrome (FCS), will be funded on the NHS after all, following a u-turn by the National Institute for Health and Care Excellence (NICE).
LONDON--(BUSINESS WIRE)--Akcea Therapeutics UK Ltd., a subsidiary of Akcea Therapeutics Inc. (NASDAQ: AKCA), announced today that the National Institute for Health and Care Excellence (NICE) has issued a positive Final Evaluation Document (FED) for volanesorsen for the treatment of Familial Chylomicronaemia Syndrome (FCS) for routine care on the National Health Service (NHS) in England. FCS is an under-recognised condition characterised by extremely high triglycerides levels (10 to 100 times normal values) and abnormal accumulation of lipoprotein particles called chylomicrons in the blood.1,2 FCS can put the patient at an increased risk of recurrent episodes of potentially fatal acute pancreatitis as well as chronic abdominal pain and long term morbidity.3 Volanesorsen is the first and only treatment for FCS currently available to patients in the UK.
BOSTON, Aug. 13, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that the German Association of Statutory Sick Funds has agreed upon new pricing and reimbursement of WAYLIVRA® (volanesorsen) as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. WAYLIVRA has been commercially available in Germany since August 15, 2019. As part of reimbursement and market access protocol in Germany, Akcea was required to renegotiate the WAYLIVRA launch price 12 months after first commercialization, which will go into effect on August 15, 2020.
Retrotope announced today that it has dosed its first patient in a Phase 2/3 clinical trial of RT001 in Friedreich’s ataxia, the most common of the inherited ataxias. RT001, a stabilized fatty acid drug, has been shown to reduce lipid peroxidation leading to cell death in patients across a wide swath of degenerative diseases, including FA. More information and a list of participating clinical sites for the current trial in FA can be obtained at ClinicalTrials.gov http://bit.ly/2T2FWA5. With this trial, the company expands its pipeline of indications for RT001 which is currently being studied in a pivotal trial in another fatal, neurodegenerative disease, Infantile Neuroaxonal Dystrophy (INAD), which is fully enrolled.
The National Institute for Health and Care Excellence (NICE) has issued an Evaluation Consultation Document (ECD), refusing Waylivra (volanesorsen) as a treatment for Familial Chylomicronaemia Syndrome (FCS).
As Akcea Therapeutics get rolling with new rare-disease med Waylivra, it's filling a new post. Its first commercial chief is joining the C-suite from inside the company.