Delving into the Latest Updates on Alnylam Japan KK with Synapse

Overview

Tags

Congenital Disorders

Other Diseases

Endocrinology and Metabolic Disease

siRNA

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

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Targets

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Disease Domain	Count

Endocrinology and Metabolic Disease	2
Nervous System Diseases	1

Top 5 Drug Type	Count

siRNA	2

Top 5 Target	Count

TTR(Transthyretin)	1
ALAS1(5 aminolevulinate synthase 1)	1

In the treatment of transthyretin-type familial amyloid polyneuropathy (FAP), In addition to liver transplantation and tetramer-stabilizing preparationPachisilane sodium (trade name: Onbatro i.v. infusion 2 mg / mL) in Sept. 2019 (Hereinafter, Patisiran) has appeared.Patisiran is the world's first RNAi generator, Gene silencing suppresses the production of wild-type TTR as well as mutant TTR.In an international collaborative phase study in patients with transsilenatin-type familial amyloid polyneuropathy (FAP), improved mNIS + 7 scores in the patisiran group compared to the placebopo group at 18 mo.Due to these advances in treatment methods, the need for early diagnosis and early intervention for this disease is increasing.

Pharm Tech Japan

Coordinated activities of industry-government-academia for efficient development of oligonucleotide therapeutics Part 8. Platform advances in siRNA therapeutics at Alnylam

Author: Kamakura, Minoru

Evaluation of the safety and efficacy of givosiran in treating patients with acute hepatic porphyria and improving their quality of life: Clinical findings from the Expanded Access Protocol in Japan

Author: Fanelli, Mary Jean ; Ozaki, Nobuaki ; Adachi, Tomohide ; Fujii, Norihisa ; Goto, Yoshie ; Frascello, Karen ; Oyama, Takuro

AbstractBackground The givosiran Expanded Access Trial is an open-label, multicenter, single-arm study designed to provide access to investigational givosiran for patients with acute hepatic porphyria (AHP) who have limited or no available treatment options and for whom the treating physician believes the benefits outweigh the risks. The effects of givosiran were evaluated in an exploratory manner. Results Givosiran was administered to 10 patients: three patients who continued from the Phase III clinical study (the ENVISION study) and seven newly included patients from this study. Low aminolevulinic acid and porphobilinogen levels were maintained. Two porphyria attacks were reported by the patients; however, both were mild and did not require hemin or hospitalization. In the Givosiran Patient Experience Questionnaire, eight of the 10 patients reported symptom improvement. Adverse events occurred in eight cases, five of which were suspected to be causally associated with givosiran. All adverse events were non-serious, and there were no deaths; however, one patient discontinued treatment due to alopecia. Conclusion This study demonstrates that AHP patients on once-monthly administration of givosiran experienced clinical benefits, and there was an acceptable safety profile. Trial registration: jRCT2071200074. Registered Dec. 24, 2020, https://jrct.niph.go.jp/en-latest-detail/jRCT2071200074

100 Deals associated with Alnylam Japan KK

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100 Translational Medicine associated with Alnylam Japan KK

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Pipeline

Pipeline Snapshot as of 06 Nov 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

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2

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