Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) approved ONPATTRO™ (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. ONPATTRO is the …
Tag Archives: Alnylam
August, 2018
July, 2018
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5 July
Alnylam Announces Publication of Phase 3 Results for Investigational RNAi Therapeutic Patisiran in Hereditary ATTR Amyloidosis
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the pivotal study results from the APOLLO Phase 3 trial of patisiran were published online today in The New England Journal of Medicine (NEJM). The study showed that patisiran improved measures of polyneuropathy, quality of life, activities of daily living, …
June, 2018
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8 June
Alnylam Reports Updated Positive Results from Phase 1/2 Study of Lumasiran in Primary Hyperoxaluria Type 1
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1). Results were presented at the OxalEurope, European Hyperoxaluria Consortium, taking …
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4 June
FDA Grants Orphan Drug Designation to Alnylam’s Investigational RNAi Therapeutic for Treatment of Transthyretin-Mediated Amyloidosis
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALN-TTRsc02, an investigational RNAi therapeutic for the treatment of transthyretin-mediated amyloidosis. ALN-TTRsc02 has the potential to be a once-quarterly, low volume, subcutaneously …
December, 2017
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15 December
FDA Lifts Clinical Hold on Alnylam’s Hemophilia Drug Candidate Fitusiran
CAMBRIDGE, Mass. & PARIS–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including the Phase 2 open-label extension (OLE) study …
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11 December
FDA Expands Orphan Drug Designation for Alnylam’s Patisiran to Treatment of Transthyretin-Mediated Amyloidosis
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today the U.S. Food and Drug Administration (FDA) has granted a request to amend the orphan drug designation for patisiran to the treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis). This is an expansion to patisiran’s prior designation which was for …
September, 2017
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20 September
Alnylam’s Rare Disease Drug Meets Endpoints in Late-Stage hATTR Amyloidosis Study
CAMBRIDGE, Mass. & PARIS–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy, met its primary efficacy endpoint …
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8 September
Patient Death Forces Alnylam to Suspend Dosing in All Ongoing Fitusiran Studies in Hemophilia Patients
CAMBRIDGE, Mass.–(BUSINESS WIRE)– Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), the leading RNAi therapeutics company, announced today an update on the company’s fitusiran and givosiran investigational RNAi therapeutic programs. With fitusiran, an RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors, Alnylam is reporting a fatal thrombotic …
May, 2017
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31 May
FDA Grants Breakthrough Therapy Designation to Alnylam’s Acute Hepatic Porphyria Drug
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the prophylaxis of attacks in patients with acute hepatic porphyria (AHP). …
April, 2017
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26 April
The Medicines Company and Alnylam Announce Agreement with FDA on Phase 3 Program for Inclisiran
PARSIPPANY, N.J. & CAMBRIDGE, Mass.–(BUSINESS WIRE)–The Medicines Company (NASDAQ:MDCO) and Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) today announced that The Medicines Company has agreed with the U.S. Food and Drug Administration (FDA) on plans for the Phase III clinical program for inclisiran, which is designed to support the submission of a New …