Lumasiran

Overview

Lumasiran is a small interfering RNA used in the treatment of primary hyperoxaluria type 1 (PH1). This condition, caused by a deficiency in the enzyme alanine-glyoxylate aminotransferase, leads to an accumulation of oxalate, causing calcium crystal formation. These patients experience frequent kidney stones, nephrocalcinosis, and renal failure. Oxlumo, producted by Alnylam Pharmaceuticals, represents the first approved treatment for PH1. Prior to this approval, therapy consisted of symptomatic treatment such as hyperhydration, inhibitors of crystallization, pyridoxine, and renal transplant. Lumasiran was granted FDA approval on 23 November 2020.

Background

Indication

Lumasiran is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients.

Associated Conditions

Primary Hyperoxaluria Type 1 (PH1)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Lumasiran in Hyperoxalaemic Patients on Haemodialysis	2024/01/26	NCT06225544	Phase 2	Recruiting	Charite University, Berlin, Germany
A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels	2021/12/17	NCT05161936	Phase 2	Terminated	Alnylam Pharmaceuticals
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1	2019/11/05	NCT04152200	Phase 3	Active, not recruiting	Alnylam Pharmaceuticals
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1	2019/10/14	NCT04125472	N/A	APPROVED_FOR_MARKETING	Alnylam Pharmaceuticals
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1	2019/04/05	NCT03905694	Phase 3	Completed	Alnylam Pharmaceuticals
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1	2018/09/21	NCT03681184	Phase 3	Completed	Alnylam Pharmaceuticals
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1	2017/11/22	NCT03350451	Phase 2	Completed	Alnylam Pharmaceuticals
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1	2016/03/11	NCT02706886	Phase 1	Completed	Alnylam Pharmaceuticals

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
OXLUMO	Alnylam Pharmaceuticals, Inc.	71336-1002	SUBCUTANEOUS	94.5 mg in 0.5 mL	9/27/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Oxlumo	Alnylam Netherlands B.V.,Antonio Vivaldistraat 150,Cross Towers, 20th floor,1083 HP Amsterdam,The Netherlands	Authorised	11/19/2020
Oxlumo	Alnylam Netherlands B.V.,Antonio Vivaldistraat 150,Cross Towers, 20th floor,1083 HP Amsterdam,The Netherlands	Authorised	11/19/2020

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
OXLUMO lumasiran (as sodium) 94.5 mg/0.5 mL injection vial.	421050	Medison Pharma Australia Pty Ltd	Medicine	A	6/24/2024

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