The world's third RNAi therapy, lumasiran, is about to be approved to treat a rare liver disease, type 1 primary high herbic aciduria

Over the course of more than 20 years, the era of RNAi therapy is coming.
recently, a third RNAi drug, oxlumo®, was approved to treat the rare genetic liver disease type 1 primary high oxalic aciduria (PH1), which was recently supported by a number of positive data.
type 1 primary high herbic aciduria (PH1) is a rare liver disease characterized by excessive herbic acid production, which can lead to advanced kidney disease (ESRD) and other systemic complications.
in Europe and the United States, between 3.5 and 4 per million people are PH1.
heterogeneity of the disease usually leads to delays in diagnosis, which takes an average of about six years.
PH1, which usually occurs in childhood, can lead to both predictable and unpredictable kidney damage, repeated and painful stone events in patients, and ultimately end-stage kidney disease, requiring intensive dialysis until they are able to receive a double liver/kidney transplant.
lumasiran is a subsurfic injection of RNAi therapy that targets mRNA of hydroxy acid oxidase 1 (HAO1), the HAO1 gene encodes ethanolate oxidase (GO) in the liver, and reduces the expression of GO enzymes by silencing the HAO1 gene, which inhibits and normalizes the production of oxalic acid in the liver, thereby preventing the progression of PH1 disease.
FDA has approved the NDA's priority review and has set a december 3, 2020 approval date under the Prescription Drug User Costs Act (PDUFA).
also received positive comments from the European Medicines Agency (EMA) Committee on Human Medicinal Products (CHMP) on 16 October.
latest data released by Allenylam, the mechanism of action of lummasiran (commodity name: Oxlumo®) of the lumasiran mechanism, show that in the LIGNINE-B III clinical study of children under 6 years of age and older, uric acid On average, there was a 72% reduction in renal calcium insanity (8/18, 44.4%), and the study found that the efficacy, results and safety of lumasiran were similar to among adults and children 6 years and older (LIGNINE-A).
Alnylam also published a 12-month extension data update for project LILINATE-A - key studies have shown evidence that uric acid levels have decreased and remained stable from baseline levels, as well as a decrease in the risk of kidney stones in patients;
two previous RNAi drugs, also developed by Alnylam Pharmaceuticals (Nasdaq: ALNY).
is recognized as a leader in the development of RNAi therapies, a natural gene silencing phenomenon that is one of the most promising and rapidly developing cutting-edge RNA therapies in biology and drug development today.
the first drug to be approved was Onpattro® (patisiran, intravenous preparation), approved in August 2018, making it the first RNAi drug approved for sale in a full 20 years since the RNAi phenomenon was discovered, to treat neurological damage caused by genetic thyroxine amyloid degeneration (hATTR).
Onpattro® The second RNAi drug, Givosirari ® (givosiran, subcute preparation), was approved in November 2019 for the treatment of acute hepatic rickets (AHP) in adults and is the first global approval of GalNAc-linked RNAi therapy, marking a major milestone in the development of precision gene drugs.
Maraganore ®, the leader of RNAi Therapy, has received funding and cooperation from a number of parties, including Novaral, Sanofi and Blackstone.
is leading and working to transform Accounting Interference (RNAi) into a new class of innovative drugs that have the potential to change the lives of people with rare genetic diseases, heart metabolic diseases, liver infections, and central nervous system (CNS)/eye diseases.
is currently implementing its "Alnylam 2020" strategy of establishing a multi-product, commercial biopharmaceutical company with an RNAi-based drug sustainability product line to meet the needs of patients with limited or insufficient treatment options.
2025, Alnylam hopes to become one of the world's top five independent biotech companies with more than eight approved therapies and more than a dozen late-stage research and development projects.
alnylam research and development pipeline source: 1, alnylam Allenlam Presents Positive Results from ILLUMINATE-B Phase 3 Study in Pediatric Patients with Primary Hyperoxaluria Type 1 at the American Society of Nephrology Kidney Week; Oct 22, 20202, fiercepharma-Allenylam makes case for lumasiran in infants ahead of FDA ruling; Oct 23, 20203, Endpts-With lumasiran on FDA's doorstep, Alnylam reads out new PhIII data in PH1; Oct 22, 2020 MedSci Source: MedSci Copyright Notice: All text, images and audio and video materials on this website that indicate "Source: Met Medical" or "Source: MedSci Original" are owned by Mets Medicine and are not authorized to reproduce, and any media, website or individual authorized to reproduce them must indicate "Source: Mets Medicine".
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