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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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