MedPath

Volanesorsen

Generic Name
Volanesorsen
Brand Names
Waylivra
Drug Type
Biotech
CAS Number
915430-78-3
Unique Ingredient Identifier
2O4BE0K238
Background

Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA to prevent its translation. It is indicated to treat familial chylomicronemia, a genetic condition that prevents breakdown of triglycerides and chylomicrons. This drug is not commonly prescribed as it is used as an adjunct to diet in patients at high risk for pancreatitis, who have had inadequate response to triglyceride lowering therapy.

Volanesorsen was granted a conditional approval by the European Medicines Agency.

Indication

Volanesorsen is conditionally approved by the EMA as an adjunct to diet in adults with familial chylomicronemia, at high risk for pancreatitis, and who have had an inadequate response to diet and triglyceride lowering therapy.

Associated Conditions
Familial Chylomicronemia Syndrome (FCS)
Clinical Trials
Related News

Ionis and Ono Forge $940 Million Deal for Sapablursen in Polycythemia Vera Treatment

• Ionis Pharmaceuticals has licensed sapablursen, an RNA-targeted medicine for polycythemia vera, to Ono Pharmaceutical in a deal worth up to $940 million including $280 million upfront. • Sapablursen, currently in Phase 2 trials, has received FDA Fast Track and Orphan Drug designations and works by increasing hepcidin production to regulate iron homeostasis in PV patients. • Ono will gain exclusive global rights for development and commercialization, while Ionis will complete the ongoing IMPRSSION study before transferring responsibilities.

IDEAYA Biosciences Reports Strong Pipeline Progress and $1.1B Cash Position for 2024

• IDEAYA Biosciences advances multiple oncology programs, with over 230 patients enrolled in potential registration-enabling trial for darovasertib in metastatic uveal melanoma, targeting key readouts by end of 2025. • Company strengthens its position with approximately $1.1 billion cash reserve as of December 2024, expected to fund operations into at least 2028. • Pipeline expansion includes progress on DLL3 TOP1i ADC IDE849 showing 73% response rate in early SCLC data, and advancement of three new candidates targeting solid tumors.

PTC Therapeutics Announces Key Clinical and Regulatory Milestones for 2025

• PTC Therapeutics anticipates a CHMP opinion on sepiapterin's MAA in Q2 2025, marking a crucial step toward potential European approval for PKU treatment. • The FDA is expected to decide on sepiapterin's NDA by July 29, 2025, potentially bringing a new therapeutic option to phenylketonuria patients in the US. • Phase 2 results from the PIVOT-HD study of PTC518 in Huntington's Disease are anticipated in Q2 2025, offering hope for a novel treatment approach. • Vatiquinone's NDA is expected to be accepted in Q1 2025, with potential regulatory approval in the second half of the year, expanding PTC's pipeline.

Ionis' Tryngolza Approved for FCS as Arrowhead's Plozasiran Awaits FDA Review

• The FDA has approved Ionis Pharmaceuticals' Tryngolza (olezarsen) as the first treatment for adults with familial chylomicronemia syndrome (FCS). • Tryngolza, an RNA-targeted therapy, significantly reduces triglyceride levels and the risk of acute pancreatitis in FCS patients when used with a low-fat diet. • Arrowhead Pharmaceuticals' plozasiran, another RNA interference therapeutic for FCS, has been accepted for FDA review, with a decision expected by November 2025. • Clinical trials showed Tryngolza achieved a 57% placebo-adjusted mean reduction in triglycerides at 12 months, while plozasiran demonstrated an 80% median reduction.
© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy