Ionis Pharmaceuticals

The article outlines the oligonucleotide synthesis market, covering synthesis technology, applications, industry players, market factors, recent developments, and global market overview. It highlights advancements in synthesis technology, the role of diagnostics, and the impact of COVID-19, alongside profiles of key companies and market segmentation by length, application, product, category, and type.

December 2024 FDA updates: Approved treatments include ustekinumab-kfce for inflammatory diseases, crinecerfont for CAH, nemolizumab for AD, and olezarsen for FCS. Notable actions: RMAT designation for rexlemestrocel-L, Boxed Warning for fezolinetant, and CRLs for glepaglutide and sotagliflozin. Pipeline updates: Tirzepatide outperformed semaglutide in weight loss, and novel therapies for HCV, PNH, and EoE showed promise.

The FDA made significant regulatory decisions, including the first drug approval for obstructive sleep apnea, a novel regenerative medicine for trauma patients, and treatments for rare diseases and cancer. Notable approvals include Eli Lilly’s Zepbound, Ionis Pharmaceuticals’ Tryngolza, and Humacyte’s Symvess. The FDA also expanded uses for existing drugs and issued rejections and warnings for others.

In 2024, Angioedema News highlighted top advancements in angioedema treatment, including deals for donidalorsen, NTLA-2002's 95% attack reduction, Takeda's partnerships, Takhzyro's efficacy in adolescents, deucrictibant's Phase 3 trials, FDA's lift on deucrictibant hold, Ionis' FDA application for donidalorsen, garadacimab's safety, Haegarda's real-world effectiveness, and Orladeyo's long-term benefits in Japan.

DelveInsight's 2024 COVID-19 Pipeline Insight report details over 180 companies and 200+ pipeline drugs, covering clinical and nonclinical stages, therapeutic assessments, and inactive products. Highlights include Novavax's JN.1 subvariant vaccine and Gilead Sciences' obeldesivir trial. Promising therapies like DAS181 and Sarconeos are in advanced clinical trials.

Tryngolza (olezarsen) approved by FDA for reducing triglycerides in adults with FCS, significantly lowering AP risk. Self-administered monthly, it showed a 57% mean triglyceride reduction and fewer AP events over 12 months in trials. Ionis Pharmaceuticals developed it, marking a first for FCS treatment.

Tryngolza (olezarsen) has been FDA-approved to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), significantly lowering triglyceride levels and acute pancreatitis events when used with a low-fat diet. It's self-administered monthly via an autoinjector, showing a favorable safety profile.

The FDA approved Ionis Pharmaceuticals' Tryngolza (olezarsen), the first treatment for familial chylomicronemia syndrome (FCS), significantly reducing triglyceride levels and acute pancreatitis events. Based on the Phase III Balance study, Tryngolza showed a 57% triglyceride reduction at 12 months and an 84% reduction in hospitalizations. It offers a favorable safety profile and is being evaluated in three Phase III trials for severe hypertriglyceridemia.

Ionis Pharmaceuticals announced FDA approval of Tryngolza (olezarsen), the first treatment for familial chylomicronemia syndrome (FCS), reducing triglycerides and acute pancreatitis risk. Tryngolza, self-administered monthly, showed significant reductions in triglyceride levels and AP events in a Phase 3 study, with a favorable safety profile. This marks a pivotal moment for Ionis, transitioning to a commercial-stage biotech company.

The FDA approved olezarsen (Tryngolza) as the first drug for adults with familial chylomicronemia syndrome, reducing triglyceride levels by 42.5% in a phase III trial. It works by lowering apolipoprotein C-III production and is indicated with a low-fat diet. Common side effects include injection site reactions, decreased platelet count, and arthralgia.