Nedosiran (Rivfloza) - primary hyperoxaluria type 1 Skip to main contentSkip to FDA SearchSkip to footer links An official website of the United States government Here's how you know U.S. Food and Drug Administration Search MenuSearch FDASubmit search Home Drugs Drug Approvals and Databases Drugs@FDADrug Approval Package: RIVFLOZAShareTweetLinkedinEmailPrintCompany: Novo Nordisk
Lumasiran (Oxlumo) - for the treatment of primary hyperoxaluria type 1 (PH1) Search Page - Drug and Health Product Register * Skip to main content * Skip to "About this site"Language selection * FrançaisGovernment of CanadaSearch and menus * Search and menusSearchSearch websiteSearchTopics menu * Jobs * Immigration * Travel * Business * Benefits * Health * Taxes * More servicesYou are here: 1
Lumasiran (Oxlumo) - primary hyperoxaluria type 1 (PH1) Published 11 December 2023 Statement of advice SMC2639 lumasiran solution for injection (Oxlumo®) Alnylam UK Ltd 10 November 2023 ADVICE: in the absence of a submission from the holder of the marketing authorisation lumasiran (Oxlumo®) is not recommended for use within NHSScotland. Indication under review: Treatment of primary hyperoxaluria type 1 (PH1) in all age groups. The holder of the marketing authorisation has not made a submission to SMC regarding this product in this indication. As a result we cannot recommend its use within NHSScotland. Advice context: No part of this advice may be used without the whole of the advice being quoted in full. This advice represents the view of the Scottish Medicines
Lumasiran (Oxlumo) - To treat hyperoxaluria type 1 Drug Approval Package: OXLUMO * Skip to main page content * Skip to search * Skip to topics menu * Skip to common linksHHS U.S. Department of Health and Human Services U.S. Food and Drug Administration * Follow FDA * En EspañolSearch FDASubmit search * Popular Content * Home * Food * Drugs * Medical Devices * Radiation-Emitting Products
Lumasiran (Oxlumo) - treating primary hyperoxaluria type 1 1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 for how to report adverse reactions. 1. NAME ; osmolality 210 to 390 mOsm/kg). 4. CLINICAL PARTICULARS 4.1 Therapeutic indications Oxlumo is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups. 4.2 Posology and method of administration Therapy should be initiated and supervised by a physician experienced in the management of hyperoxaluria. Posology Oxlumo is administered by subcutaneous injection
Characterization of CHK-336, A First-in-Class, Liver-Targeted, Small Molecule Lactate Dehydrogenase Inhibitor for Hyperoxaluria Treatment. Primary hyperoxalurias 1-3 (PH1-3) are genetic diseases defined by elevated hepatic oxalate production and increased incidence of calcium oxalate kidney stones and potentially kidney failure. There are two approved agents available for PH1, and no approved therapies for PH2 or PH3. Lactate dehydrogenase A (LDHA) catalyzes the final step in hepatic oxalate synthesis and represents a potential therapeutic target for PH and other forms of hyperoxaluria associated with increased oxalate production. Potent and selective LDH inhibitors with liver-targeted tissue distribution were identified and characterized in enzymatic, cellular, and in vivo models. We
A molecular journey on the pathogenesis of primary hyperoxaluria. Primary hyperoxalurias (PHs) are rare disorders caused by the deficit of liver enzymes involved in glyoxylate metabolism. Their main hallmark is the increased excretion of oxalate leading to the deposition of calcium oxalate stones in the urinary tract. This review describes the molecular aspects of PHs and their relevance
Resolution of Crystalline Retinopathy After Kidney Transplant for Hyperoxaluria. This case report discusses the evolution of crystalline retinopathy secondary to systemic hyperoxalosis after kidney transplant for hyperoxaluria was performed.
Lumasiran: A Review in Primary Hyperoxaluria Type 1. Lumasiran (Oxlumo), a first-in-class synthetic, double-stranded, ribonucleic acid (RNA) interference molecule targeting glycolate oxidase through silencing HAO1 mRNA, is approved in several countries for patients of any age and stage of kidney function with primary hyperoxaluria type 1 (PH1). Approval was based on results from the phase III
The Evolving Role of Genetic Testing in Monogenic Kidney Stone Disease: Spotlight on Primary Hyperoxaluria. Multiple factors are thought to give rise to common, recurrent kidney stone disease, but for monogenic stone disorders a firm diagnosis is possible through genetic testing. The autosomal recessive primary hyperoxalurias (PH) are rare but important forms of monogenic kidney stone disease and mortality, occurs in the setting of CKD when oxalate clearance by the kidneys declines. Novel small interfering RNA-based therapeutics targeting the liver to reduce urinary oxalate excretion have been approved, introducing precision medicine to treat primary hyperoxaluria type 1 (PH1). Increased access to genetic testing facilitates early detection of PH and other monogenic causes of kidney stone disease
Primary hyperoxaluria: Long-term outcomes of isolated kidney versus simultaneous liver/kidney transplant. To compare long-term transplant outcomes (organ rejection and retransplant) of simultaneous liver/kidney transplant (SLK) versus isolated kidney transplant (IK) for patients with primary hyperoxaluria (PH). The Rare Kidney Stone Consortium PH registry was queried to identify patients with PH
Efficient and Safe In Vivo Treatment of Primary Hyperoxaluria Type 1 via LNP-CRISPR/Cas9-mediated glycolate oxidase disruption. Primary hyperoxaluria type 1 (PH1) is a severe genetic metabolic disorder caused by mutations in the AGXT gene, leading to defects in enzymes crucial for glyoxylate metabolism. PH1 is characterized by severe, potentially life-threatening manifestations due to excessive
Effect of the allelic background on the phenotype of primary hyperoxaluria type I. Primary hyperoxaluria type 1 (PH1) is an autosomal recessive disorder of hepatic glyoxylate metabolism leading to nephrolithiasis and kidney failure. PH1 is caused by mutations on the AGXT gene encoding alanine:glyoxylate aminotransferase (AGT). The AGXT gene has two haplotypes, the major (Ma) and the minor (mi
A Targeted Release Capsule of Lanthanum Carbonate: a New Efficient Cheap Treatment for Primary Hyperoxalurias. Primary hyperoxaluria (PH) is a devastating disease in children and adults. Recently, a substantial progress in the treatment of this deadly disease has been made that consists of the introduction of the RNA inhibitors, lumasiran and nedosiran, which deplete the substrate for oxalate
Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. Lumasiran reduces urinary and plasma oxalate (POx) in patients with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function. ILLUMINATE-C evaluates the efficacy, safety, pharmacokinetics, and pharmacodynamics of lumasiran in patients with PH1 and advanced kidney disease. Phase 3, open-label reductions in POx with acceptable safety in patients with PH1 who have advanced kidney disease, supporting its efficacy and safety in this patient population. Alnylam Pharmaceuticals. Registered at ClinicalTrials.gov with study number NCT04152200 and at EudraCT with study number 2019-001346-17. Primary hyperoxaluria type 1 (PH1) is a rare genetic disease characterized by excessive hepatic oxalate
Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that causes progressive kidney damage and systemic oxalosis due to hepatic overproduction of oxalate. Lumasiran demonstrated efficacy and safety in the 6-month primary analysis period
ePHex: a phase 3, double-blind, placebo-controlled, randomized study to evaluate long-term efficacy and safety of Oxalobacter formigenes in patients with primary hyperoxaluria. Primary hyperoxalurias (PHs) are rare genetic diseases that increase the endogenous level of oxalate, a waste metabolite excreted predominantly by the kidneys and also the gut. Treatments aim to improve oxalate excretion