IONIS PHARMACEUTICALS, INC.

Ionis Pharma's olezarsen, approved by FDA, is the first drug therapy for adults with familial chylomicronaemia syndrome (FCS), reducing triglycerides. It is set to launch at $595,000 annually, replacing the need for a restrictive low-fat diet. The approval is based on the BALANCE study, showing significant triglyceride reduction and zero acute pancreatitis cases at the highest dose. Ionis plans to commercialize olezarsen without a partner, with potential for over $1 billion in annual sales.

Ionis plans to launch rare disease therapy Tryngzola, but may face competition from a more potent rival by mid-2025.

The FDA approved Ionis’ Tryngolza as the first treatment for familial chylomicronemia syndrome, significantly lowering triglycerides and reducing acute pancreatitis events when combined with a low-fat diet.

Ascension disclosed a cyberattack affecting 5.6M patients and staff, Gilead will halt free HIV drug access, Ionis won FDA approval for a rare disease drug, a new health clinic aims to reduce barriers in Denver, and Amazon settled worker safety citations for $145,000.

FDA approves Tryngolza (olezarsen) to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare genetic form of severe hypertriglyceridemia. Tryngolza is the first FDA-approved treatment for FCS, significantly reducing triglyceride levels and acute pancreatitis events when used with a low-fat diet. It is self-administered via an auto-injector once monthly.

The FDA is set to decide on ten drug applications, including Ionis' olezarsen for familial chylomicronemia syndrome, Lexicon's sotagliflozin for type 1 diabetes, AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC, Zealand Pharma's glepaglutide for short bowel syndrome, Rhythm Pharmaceuticals' Imcivree for genetic obesity in children, Checkpoint Therapeutics' cosibelimab for cutaneous squamous cell carcinoma, Mirum's chenodiol for cerebrotendinous xanthomatosis, Bristol Myers Squibb's subcutaneous Opdivo, and Neurocrine's crinecerfont for congenital adrenal hyperplasia.

Replimune announced a $125M offering four days after...

FDA approves BridgeBio Pharma's acoramidis (Attruby) for ATTR-CM, setting up a market competition with Pfizer's Vyndamax/Vyndaqel/Vynmac. Attruby, priced at $18,759 for a 28-day supply, aims to reduce cardiovascular death and hospitalization in ATTR-CM patients. BridgeBio plans to pursue regulatory approvals in Europe, Japan, and Brazil. Analysts predict potential peak sales of $2 billion or more for Attruby.

The FDA approved acoramidis (Attruby) for transthyretin amyloid cardiomyopathy (ATTR-CM), reducing cardiovascular death and hospitalization. Acoramidis, an orally-administered TTR stabilizer, showed a 42% reduction in mortality and recurrent hospitalization events at Month 30 in a phase 3 trial.

Aficamten treatment in FOREST-HCM trial showed 70% of SRT-eligible oHCM patients no longer needing SRT after 24 weeks, with 92.9% improving at least 1 NYHA class.