Lumasiran: First Approval.

Abstract:

:Lumasiran (Oxlumo™) is a subcutaneously administered small interfering RNA (siRNA) targeting the mRNA for hydroxyacid oxidase 1 gene (HAO1; encodes glycolate oxidase) and was developed by Alnylam Pharmaceuticals for the treatment of primary hyperoxaluria type 1 (PH1). By silencing the gene encoding glycolate oxidase, lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of PH1. On 19 November 2020, lumasiran received its first global approval in the EU for the treatment of PH1 in all age groups. On 23 November 2020, lumasiran was approved in the USA for the treatment of adult and paediatric patients with PH1. This article summarizes the milestones in the development of lumasiran leading to this first approval.

journal_name

Drugs

journal_title

Drugs

authors

Scott LJ,Keam SJ

doi

10.1007/s40265-020-01463-0

subject

Has Abstract

pub_date

2021-01-06 00:00:00

eissn

0012-6667

issn

1179-1950

pii

10.1007/s40265-020-01463-0

pub_type

杂志文章,评审

相关文献

DRUGS文献大全
  • Management of recurrent head and neck cancer: recent progress and future directions.

    abstract::The incidence of squamous cell carcinoma of the head and neck (SCCHN) is on the rise in the US despite a drop in cigarette smoking rates. Much of this rise is due to the increasing incidence of SCCHN attributable to human papillomavirus (HPV). HPV-related SCCHN has a high cure rate, which contributes to the stable dea...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/11592540-000000000-00000

    authors: Brockstein BE

    更新日期:2011-08-20 00:00:00

  • Tenoxicam. A preliminary review of its pharmacodynamic and pharmacokinetic properties, and therapeutic efficacy.

    abstract::Tenoxicam is a new non-steroidal anti-inflammatory and analgesic agent of the oxicam class, and therefore closely related to piroxicam. It possesses a long half-life which enables it to be administered once daily. Clinical trials in patients with rheumatoid arthritis, osteoarthritis, ankylosing spondylitis, gout and n...

    journal_title:Drugs

    pub_type: 临床试验,杂志文章,评审

    doi:10.2165/00003495-198734030-00001

    authors: Gonzalez JP,Todd PA

    更新日期:1987-09-01 00:00:00

  • Drug-induced hypertension: pathogenesis and management.

    abstract::Estrogenic compounds are the most important group of drugs that can induce hypertension. Studies have shown an incidence of significant hypertension amounting to less than 1% after 1 year of taking oral contraceptives and about 2% after 5 years. The ratio of the incidence of hypertension among ''takers'' to that of ...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-197612030-00005

    authors: Stokes GS

    更新日期:1976-01-01 00:00:00

  • Ambulatory blood pressure monitoring.

    abstract::This paper reviews the evidence that, in patients with hypertension, end-organ damage correlates more closely with blood pressure values obtained by ambulatory blood pressure monitoring than with those obtained by conventional sphygmomanometry. However, ambulatory blood pressure monitoring is not suitable for routine ...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199200441-00004

    authors: Mancia G,Omboni S,Ravogli A,Frattola A,Villani A

    更新日期:1992-01-01 00:00:00

  • Antirheumatic drugs: clinical pharmacological and therapeutic aspects.

    abstract::There are many current concepts of the pathogenesis of rheumatic diseases which incorporate immunological, infectious and hereditary factors. Rheumatic diseases may sometimes become apparent after trauma, be associated with certain diseases and may be induced by nerve damage and serum sickness. Systemic lupuserythemat...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-197510050-00008

    authors: Smyth CJ,Bravo JF

    更新日期:1975-01-01 00:00:00

  • HDL-cholesterol as a risk factor in coronary heart disease. An update of the Helsinki Heart Study.

    abstract::The aim of the Helsinki Heart Study, a 5-year primary prevention placebo-controlled study involving 4081 dyslipidaemic men (aged 40 to 55 years), was to investigate if increasing high density lipoprotein (HDL)-cholesterol plasma levels and decreasing low density lipoprotein (LDL)-cholesterol levels would reduce the in...

    journal_title:Drugs

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.2165/00003495-199000401-00004

    authors: Frick MH,Manninen V,Huttunen JK,Heinonen OP,Tenkanen L,Mänttäri M

    更新日期:1990-01-01 00:00:00

  • Tipranavir: a ritonavir-boosted protease inhibitor.

    abstract::Tipranavir is a non-peptidic HIV-1 protease inhibitor. It binds strongly and selectively, has a favourable resistance profile, and is administered orally twice daily with a subtherapeutic dosage of ritonavir in a 'boosted' regimen (TPV/r) in order to increase its bioavailability. Analysis of clinical isolates from tre...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200565120-00005

    authors: Croom KF,Keam SJ

    更新日期:2005-01-01 00:00:00

  • Pancreatic islet cell transplantation: likely impact on current therapeutics for type 1 diabetes mellitus.

    abstract::Pancreatic islet transplantation as a cell-based treatment for diabetes mellitus has been pursued for over quarter of a century, ever since the first successful use of this approach to cure diabetes mellitus in rodents in the 1970s. However, even though autoislet intrahepatic transplantation in patients with chronic p...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-200161140-00001

    authors: Robertson RP

    更新日期:2001-01-01 00:00:00

  • Current treatment recommendations for topical burn therapy.

    abstract::Infections in burn patients continue to be the primary source of morbidity and mortality. Topical antimicrobial therapy remains the single most important component of wound care in hospitalised burn patients. The goal of prophylactic topical antimicrobial therapy is to control microbial colonisation and prevent burn w...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199040030-00004

    authors: Monafo WW,West MA

    更新日期:1990-09-01 00:00:00

  • Prevention of transplant rejection: current treatment guidelines and future developments.

    abstract::In the past 2 decades, progressive improvements in the results of organ transplantation as a therapeutic strategy for patients with end-stage organ disease have been achieved due to greater insight into the immunobiology of graft rejection and better measures for surgical and medical management. It is now known that T...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199754040-00003

    authors: Perico N,Remuzzi G

    更新日期:1997-10-01 00:00:00

  • Sacituzumab Govitecan: First Approval.

    abstract::Sacituzumab govitecan (sacituzumab govitecan-hziy; Trodelvy™) is a Trop-2-directed antibody conjugated to a topoisomerase I inhibitor (SN-38) that is being developed by Immunomedics for the treatment of solid tumours, including breast cancer. In April 2020, sacituzumab govitecan received accelerated approval in the US...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-020-01337-5

    authors: Syed YY

    更新日期:2020-07-01 00:00:00

  • Postanaesthetic shivering: epidemiology, pathophysiology, and approaches to prevention and management.

    abstract::Along with nausea and vomiting, postanaesthetic shivering is one of the leading causes of discomfort for patients recovering from general anaesthesia. The distinguishing factor during electromyogram recordings between patients with postanaesthetic shivering and shivering in fully awake patients is the existence of clo...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200161150-00004

    authors: Alfonsi P

    更新日期:2001-01-01 00:00:00

  • General considerations when treating hypertension.

    abstract::Numerous intervention studies have clearly demonstrated the value of antihypertensive treatment. By lowering diastolic blood pressure 5 to 6mm Hg in middle-aged hypertensive patients, a 42% reduction in stroke morbidity and a 14% reduction in coronary heart disease morbidity can be obtained. Recently published large i...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199300462-00008

    authors: Hansson L

    更新日期:1993-01-01 00:00:00

  • Influence of carvedilol on blood glucose and glycohaemoglobin A1 in non-insulin-dependent diabetics.

    abstract::Pharmacological treatment of hypertension can, cause clinically significant alterations in endocrine function and glucose homeostasis. The aim of this study was to investigate the antihypertensive efficacy and the influence on carbohydrate metabolism of carvedilol and metoprolol in non-insulin-dependent diabetics with...

    journal_title:Drugs

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.2165/00003495-198800366-00023

    authors: Ehmer B,van der Does R,Rudorf J

    更新日期:1988-01-01 00:00:00

  • Clinical pharmacology, therapeutic use and potential of COMT inhibitors in Parkinson's disease.

    abstract::When peripheral decarboxylation is blocked by carbidopa or benserazide, the main metabolic pathway of levodopa is O-methylation by catechol-O-methyltransferase (COMT). Entacapone and tolcapone are new potent, selective and reversible nitrocatechol-type COMT inhibitors. Animal studies have demonstrated that entacapone ...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200059060-00004

    authors: Kaakkola S

    更新日期:2000-06-01 00:00:00

  • Steroid-refractory severe ulcerative colitis: what are the available treatment options?

    abstract::Approximately 15% of patients with ulcerative colitis will experience a severe episode requiring hospitalization. Although intravenous corticosteroids are the current first-line therapy for these patients, about 30% of patients do not respond to corticosteroids and require either an alternative anti-inflammatory agent...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200868090-00001

    authors: Moss AC,Peppercorn MA

    更新日期:2008-01-01 00:00:00

  • The cost of treating systemic fungal infections.

    abstract::The increasing incidence of systemic fungal infections and rising medical costs have highlighted the need for an economic appraisal of antifungal agents to determine the most cost-effective therapeutic option. Cost savings derived from the prophylactic or empirical use of antifungal agents have been difficult to estim...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200161001-00005

    authors: van Gool R

    更新日期:2001-01-01 00:00:00

  • The challenge of changing healthcare systems.

    abstract::Healthcare systems are in flux throughout the world. Traditional structures and attitudes are changing. The balance of power between political bodies, payers, providers and patients is being destabilised. New approaches by governments and forward integration by drug companies and payers into care management are all ma...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-199600522-00014

    authors: Weber F

    更新日期:1996-01-01 00:00:00

  • Fluticasone furoate: intranasal use in allergic rhinitis.

    abstract::Fluticasone furoate nasal spray is a new topical intranasal corticosteroid with enhanced affinity for the glucocorticoid receptor and low systemic exposure, which was recently approved in the US for the treatment of seasonal or perennial allergic rhinitis in adults and in children aged >or=2 years. Fluticasone furoate...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200767130-00010

    authors: McCormack PL,Scott LJ

    更新日期:2007-01-01 00:00:00

  • Postoperative ileus: progress towards effective management.

    abstract::The pathogenesis of postoperative ileus (PI) is multifactorial, and includes activation of inhibitory reflexes, inflammatory mediators and opioids (endogenous and exogenous). Accordingly, various strategies have been employed to prevent PI. As single-modality treatment, continuous postoperative epidural analgesia incl...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200262180-00004

    authors: Holte K,Kehlet H

    更新日期:2002-01-01 00:00:00

  • Torasemide. A review of its pharmacological properties and therapeutic potential.

    abstract::Torasemide (torsemide) is a high-ceiling loop diuretic which acts on the thick ascending limb of the loop of Henle to promote rapid and marked excretion of water, sodium and chloride. Like furosemide (frusemide), its major site of action is from the luminal side of the cell. Torasemide is at least twice as potent as f...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199141010-00008

    authors: Friedel HA,Buckley MM

    更新日期:1991-01-01 00:00:00

  • Effect of felodipine and metoprolol on muscle and skin arteries in hypertensive patients.

    abstract::The antihypertensive and vasodilator effects of felodipine, a new calcium antagonist of the dihydropyridine group, were examined in 15 patients with moderate to severe hypertension. Flow was measured simultaneously at the calf and finger using a venous occlusion ECG-triggered plethysmograph. Measurements were made at ...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-198500292-00024

    authors: Clement DL,De Pue NY

    更新日期:1985-01-01 00:00:00

  • Optimal pharmacological therapy for community-acquired pneumonia: the role of dual antibacterial therapy.

    abstract::The optimal pharmacological therapy of community-acquired pneumonia (CAP) is one of the most ardently debated issues in medicine. Presently, most guidelines recommend either a fluoroquinolone alone or dual therapy with a third-generation cephalosporin plus a macrolide in patients hospitalised with CAP, but few provide...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-200565140-00004

    authors: Epstein BJ,Gums JG

    更新日期:2005-01-01 00:00:00

  • The use of fluoroquinolones in sexually transmitted diseases in Southeast Asia.

    abstract::This paper reviews the use of the fluoroquinolone antibiotics in the management of sexually transmitted diseases (STD) in Southeast Asia. Numerous clinical trials performed in the region have shown that the quinolones are highly effective for treating uncomplicated gonorrhoea. Norfloxacin 800 mg, ofloxacin 400 mg, cip...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.2165/00003495-199500492-00019

    authors: Sivayathorn A

    更新日期:1995-01-01 00:00:00

  • A comparison of roxatidine acetate and ranitidine in gastric ulcer healing.

    abstract::A randomised multicentre double-blind study was conducted to compare the efficacy and safety of roxatidine acetate 75 mg twice daily and ranitidine 150 mg twice daily in 295 patients with endoscopically confirmed gastric ulcers. Substantial reductions in ulcer diameters and healing rates of 85.6 and 88.2% for roxatidi...

    journal_title:Drugs

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.2165/00003495-198800353-00021

    authors: Judmaier G

    更新日期:1988-01-01 00:00:00

  • Is tiaprofenic acid different from other NSAIDs with regard to effects on renal function in the elderly?

    abstract::In a study of the effects of various NSAIDs on renal function in the elderly, indomethacin, tiaprofenic acid and sulindac were administered to patients ranging in age from 54 to 97 years for a period of 3 months. Indomethacin sustained release (25 mg tid) was administered to 66 patients, tiaprofenic acid (200 mg tid) ...

    journal_title:Drugs

    pub_type: 杂志文章

    doi:10.2165/00003495-198800351-00022

    authors: Ishioka T

    更新日期:1988-01-01 00:00:00

  • Elbasvir/Grazoprevir: First Global Approval.

    abstract::A fixed-dose combination tablet of the hepatitis C virus (HCV) NS5A inhibitor elbasvir and the HCV NS3/4A protease inhibitor grazoprevir (elbasvir/grazoprevir; Zepatier™) is under development by Merck. Oral elbasvir/grazoprevir 50/100 mg once daily has been approved in the USA for the treatment of adults with chronic ...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-016-0558-3

    authors: Keating GM

    更新日期:2016-04-01 00:00:00

  • Hepatic Encephalopathy: Definition, Clinical Grading and Diagnostic Principles.

    abstract::In general, hepatic encephalopathy (HE) is defined as a brain dysfunction caused by liver insufficiency and/or portal-systemic blood shunting. This article relates to the so-called type C HE: that is, HE in patients with liver cirrhosis. It manifests as a wide spectrum of neurological or psychiatric abnormalities, ran...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-018-1018-z

    authors: Weissenborn K

    更新日期:2019-02-01 00:00:00

  • Are All Dopamine Agonists Essentially the Same?

    abstract::Dopamine agonists (DAs) represent an excellent treatment option for patients with Parkinson's disease, in both the early and advanced stages of the disease, improving motor symptoms, lowering the incidence of motor complications, and addressing several non-motor symptoms. Indeed, each of these compounds have different...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-019-01103-2

    authors: Torti M,Bravi D,Vacca L,Stocchi F

    更新日期:2019-05-01 00:00:00

  • Peramivir: A Review in Uncomplicated Influenza.

    abstract::Intravenous peramivir (Alpivab™; Rapivab®; Rapiacta®; PeramiFlu®), the most recent globally approved inhibitor of influenza neuraminidase, is indicated for the treatment of uncomplicated influenza in adults and children from the age of 2 years. This article, written from an EU perspective, reviews the clinical use of ...

    journal_title:Drugs

    pub_type: 杂志文章,评审

    doi:10.1007/s40265-018-0981-8

    authors: Scott LJ

    更新日期:2018-09-01 00:00:00