Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company presented updated results from the ongoing Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic, targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP). The new …
Tag Archives: Alnylam
November, 2018
October, 2018
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15 October
Alnylam to Initiate Rolling Submission of NDA and Pursue Full Approval for Givosiran for Acute Hepatic Porphyria
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that in consultation with the U.S. Food and Drug Administration (FDA), the Company plans to pursue a full approval based on the complete results of the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting …
August, 2018
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10 August
FDA Approves Alnylam’s RNAi Therapeutic Onpattro for Treatment of Polyneuropathy of Hereditary 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) 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 …
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 …