News & Meetings

Eplontersen halted ATTRv-PN disease progression and improved neuropathy impairment and quality of life in Phase 3 study through 66 weeks

CARLSBAD, Calif., April 24, 2023 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the Phase 3 NEURO-TTRansform study for AstraZeneca and Ionis’ eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) met all co-primary endpoints and secondary endpoints at 66 weeks versus an external placebo group. The positive results are being presented today […]

Eplontersen halted ATTRv-PN disease progression and improved neuropathy impairment and quality of life in Phase 3 study through 66 weeks Read More »

Acoramidis Reduces All-Cause, Cardiovascular Mortality at 30 Months in ATTR-CM

The 30-month results of the phase 3 ATTRibute-CM trial provide the latest insight into the effects of acoramidis in people with transthyretin amyloid cardiomyopathy (ATTR-CM) ahead of a potential regulatory submission for the agent. Presented during a hot line session at the European Society of Cardiology (ESC) Congress 2023, results of the trial suggest the acoramidis cohort experienced

Acoramidis Reduces All-Cause, Cardiovascular Mortality at 30 Months in ATTR-CM Read More »

Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting

– Cardiovascular and Renal Drugs Advisory Committee Voted 9:3 That the Benefits of Patisiran Outweigh its Risks for the Treatment of the Cardiomyopathy of ATTR Amyloidosis – – Prescription Drug User Fee Act Target Action Date is October 8, 2023 – CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sep. 13, 2023– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics

Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting Read More »

Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

– FDA Cites Insufficient Evidence of Clinical Meaningfulness – – No Clinical Safety, Study Conduct, Drug Quality or Manufacturing Issues Identified – – CRL Does Not Pertain to, nor Impact Commercial Availability of, ONPATTRO® (patisiran) for Existing Indication for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis in Adults – – Alnylam to Host

Alnylam Announces Receipt of Complete Response Letter from U.S. FDA for Supplemental New Drug Application for Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis Read More »

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