Lumasiran

Canada Expands Access to Rare Disease Medications Through Provincial Agreements

2 months ago

• The Government of Canada has signed bilateral agreements with Yukon and Nova Scotia, investing over $8.5 million and $39 million respectively over three years to improve access to drugs for rare diseases. • The agreements will provide funding for several specialized medications including Yescarta, a CAR T-cell therapy for B-cell lymphomas, and five additional drugs in Nova Scotia for conditions including mycosis fungoides and von Hippel-Lindau disease. • These initiatives are part of Canada's National Strategy for Drugs for Rare Diseases, which aims to help the one in 12 Canadians living with rare diseases access treatments that can cost between $100,000 and $4 million annually.

FDA Approves Alnylam's Oxlumo as First Treatment for Ultra-Rare Kidney Disease PH1

2 months ago

• The FDA has approved Oxlumo (lumasiran), the first-ever treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare genetic disorder that causes kidney damage through excessive oxalate production. • Oxlumo, Alnylam's third RNA interference therapeutic to reach market, works by silencing genes that lead to oxalate production, with clinical trials showing significant reductions in urinary oxalate levels. • The approval gives Alnylam a competitive advantage in this small but potentially profitable market, with pricing expected to be substantial given the rarity of PH1 and the company's previous rare disease therapies costing $450,000-575,000 annually.

Alnylam Advances RNAi Therapeutics Pipeline, Awaits Key Regulatory Decisions

4 months ago

• Alnylam anticipates FDA approval for vutrisiran to treat ATTR amyloidosis with cardiomyopathy by March 23, 2025, and aims for EU and Japan approvals later in the year. • A Phase 3 trial for nucresiran in ATTR amyloidosis with cardiomyopathy is set to begin in the first half of 2025, expanding Alnylam's focus on cardiac amyloidosis. • Alnylam's pipeline progresses with zilebesiran in hypertension, mivelsiran in Alzheimer's, and ALN-6400 in bleeding disorders, with key data readouts and trial initiations expected in 2025.

FDA Accepts Alnylam's Vutrisiran Application for ATTR Amyloidosis with Cardiomyopathy

6 months ago

• The FDA has accepted Alnylam's sNDA for vutrisiran to treat ATTR amyloidosis with cardiomyopathy, setting a PDUFA date of March 23, 2025. • The application is based on Phase 3 HELIOS-B trial results, which demonstrated favorable cardiovascular outcomes and improved survival in ATTR-CM patients. • If approved, vutrisiran would be the first treatment for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis in the U.S. • Vutrisiran's sNDA acceptance signifies a major advancement for Alnylam, potentially expanding the drug's market and solidifying its position in the ATTR space.

Acoramidis Gains Global Momentum: FDA Approval, EU Recommendation, and Promising Clinical Data

6 months ago

• Acoramidis (Attruby), developed by Stanford Medicine and BridgeBio, receives FDA approval for transthyretin amyloid cardiomyopathy (ATTR-CM) treatment, marking a significant milestone. • The European Medicines Agency's CHMP recommends acoramidis for EU marketing authorization based on positive Phase 3 ATTRibute-CM trial results. • Clinical trials demonstrate acoramidis' efficacy in reducing cardiovascular-related hospitalizations and improving survival rates for ATTR-CM patients. • Bayer and BridgeBio collaborate to commercialize acoramidis, with Bayer holding EU rights and plans for a launch in Europe in early 2025.

Non-Viral CRISPR Therapy Shows Promise for Primary Hyperoxaluria Type 1 in Preclinical Study

7 months ago

• A non-viral CRISPR-Cas9 therapy targeting the _Hao1_ gene significantly reduced oxalate levels in a mouse model of primary hyperoxaluria type 1 (PH1). • The therapy, delivered via lipid nanoparticles (LNPs), demonstrated lasting effects over a 12-month period with no significant off-target effects observed. • In a humanized mouse model, the CRISPR therapy targeting human _Hao1_ also showed dose-dependent reduction in urinary oxalate levels, supporting clinical translation. • YolTech Therapeutics is advancing this LNP-mediated CRISPR therapy, YOLT-203, into clinical trials for PH1, offering a potential new treatment approach.

Alnylam Anticipates Strong Growth in 2025 Driven by RNAi Therapeutics

a year ago

• Alnylam forecasts 2025 net product revenues between $2.05 billion and $2.25 billion, a 31% increase from 2024, driven by its TTR and rare disease franchises. • The company anticipates FDA approval and launch of vutrisiran for ATTR amyloidosis with cardiomyopathy, expecting significant revenue growth from this indication. • Alnylam plans to initiate a Phase 3 study of nucresiran for ATTR amyloidosis with cardiomyopathy and report results from the zilebesiran KARDIA-3 Phase 2 hypertension study. • The company expects to achieve non-GAAP operating income profitability in 2025, marking a key milestone in its "Alnylam P5x25" strategy.