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OXLUMO (lumasiran) Structural Formula - Illustration

The molecular formula of lumasiran sodium is C530H669F10N173O320P43S6Na43 and the molecular weight is 17,286 Da.


CAS 1834610-13-7

FDA APPROVED, 11/23/2020, Oxlumo

To treat hyperoxaluria type 1
Press Release
Drug Trials Snapshot

RNA, (Gm-​sp-​Am-​sp-​Cm-​Um-​Um-​Um-​(2′-​deoxy-​2′-​fluoro)​C-​Am-​(2′-​deoxy-​2′-​fluoro)​U-​(2′-​deoxy-​2′-​fluoro)​C-​(2′-​deoxy-​2′-​fluoro)​C-​Um-​Gm-​Gm-​Am-​Am-​Am-​Um-​Am-​Um-​Am)​, 3′-​[[(2S,​4R)​-​1-​[29-​[[2-​(acetylamino)​-​2-​deoxy-​β-​D-​galactopyranosyl]​oxy]​-​14,​14-​bis[[3-​[[3-​[[5-​[[2-​(acetylamino)​-​2-​deoxy-​β-​D-​galactopyranosyl]​oxy]​-​1-​oxopentyl]​amino]​propyl]​amino]​-​3-​oxopropoxy]​methyl]​-​1,​12,​19,​25-​tetraoxo-​16-​oxa-​13,​20,​24-​triazanonacos-​1-​yl]​-​4-​hydroxy-​2-​pyrrolidinyl]​methyl hydrogen phosphate]​, complex with RNA (Um-​sp-​(2′-​deoxy-​2′-​fluoro)​A-​sp-​Um-​Am-​Um-​(2′-​deoxy-​2′-​fluoro)​U-​Um-​(2′-​deoxy-​2′-​fluoro)​C-​(2′-​deoxy-​2′-​fluoro)​C-​Am-​Gm-​Gm-​Am-​(2′-​deoxy-​2′-​fluoro)​U-​Gm-​(2′-​deoxy-​2′-​fluoro)​A-​Am-​Am-​Gm-​Um-​Cm-​sp-​Cm-​sp-​Am) (1:1)

Nucleic Acid Sequence

Sequence Length: 44, 23, 2115 a 8 c 7 g 14 umultistranded (2); modified

OXLUMO is supplied as a sterile, preservative-free, clear, colorless-to-yellow solution for subcutaneous administration containing the equivalent of 94.5 mg of lumasiran (provided as lumasiran sodium) in 0.5 Ml of water for injection and sodium hydroxide and/or phosphoric acid to adjust the pH to ~7.0.

Lumasiran An investigational RNAi Therapeutic for Primary Hyperoxaluria Type 1 (PH1)

Overview • Lumasiran (ALN-GO1) is an investigational, subcutaneously administered (under the skin) RNA interference (RNAi) therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1).

• PH1 is a rare, life-threatening disease that can cause serious damage to kidneys and progressively to other organs.1

• PH1 is characterized by the pathologic overproduction of oxalate by the liver. Oxalate is an end product of metabolism that, when in excess, is toxic and accumulates in the kidneys forming calcium oxalate crystals.1,2

• Symptoms of PH1 are often associated with recurrent kidney stones and include flank pain, urinary tract infections, painful urination, and blood in the urine.2,3

• Currently, the only curative treatment is a liver transplant, to correct the metabolic defect, combined with a kidney transplant, to replace the terminally damaged kidneys.1,3 Clinical Development

• The safety and efficacy of lumasiran are being evaluated in a randomized, double-blind, placebo-controlled, global, multicenter Phase 3 study of approximately 30 PH1 patients, called ILLUMINATE-A (NCT03681184).

• The primary endpoint is percent change in 24-hour urinary oxalate excretion from baseline to Month 6.

• Key secondary and exploratory endpoints in ILLUMINATE-A will evaluate additional measures of urinary oxalate, estimated glomerular filtration rate (eGFR), safety, and tolerability. 

Regulatory Designations • Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) • Priority Medicines (PRIME) Designation from the European Medicines Agency (EMA) • Orphan Drug Designations in both the U.S. and the European Union

Alnylam Announces U.S. Food and Drug Administration Has Granted Priority  Review of the Lumasiran New Drug Application for the Treatment of Primary  Hyperoxaluria Type 1 | Business Wire

/////////lumasiran, fda 2020, 2020 approvals, Oxlumo, Breakthrough Therapy Designation, Orphan Drug, Priority Medicines (PRIME) Designation

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DR ANTHONY MELVIN CRASTO, Born in Mumbai in 1964 and graduated from Mumbai University, Completed his Ph.D from ICT, 1991,Matunga, Mumbai, India, in Organic Chemistry, The thesis topic was Synthesis of Novel Pyrethroid Analogues, Currently he is working with AFRICURE PHARMA, ROW2TECH, CLEANCHEM LABS as ADVISOR, earlier assignment was with GLENMARK LIFE SCIENCES LTD, as CONSUlTANT, Retired from GLENMARK in Jan2022 Research Centre as Principal Scientist, Process Research (bulk actives) at Mahape, Navi Mumbai, India. Total Industry exp 32 plus yrs, Prior to joining Glenmark, he has worked with major multinationals like Hoechst Marion Roussel, now Sanofi, Searle India Ltd, now RPG lifesciences, etc. He has worked with notable scientists like Dr K Nagarajan, Dr Ralph Stapel, Prof S Seshadri, etc, He did custom synthesis for major multinationals in his career like BASF, Novartis, Sanofi, etc., He has worked in Discovery, Natural products, Bulk drugs, Generics, Intermediates, Fine chemicals, Neutraceuticals, GMP, Scaleups, etc, he is now helping millions, has 9 million plus hits on Google on all Organic chemistry websites. His friends call him Open superstar worlddrugtracker. His New Drug Approvals, Green Chemistry International, All about drugs, Eurekamoments, Organic spectroscopy international, etc in organic chemistry are some most read blogs He has hands on experience in initiation and developing novel routes for drug molecules and implementation them on commercial scale over a 32 PLUS year tenure till date Feb 2023, Around 35 plus products in his career. He has good knowledge of IPM, GMP, Regulatory aspects, he has several International patents published worldwide . He has good proficiency in Technology transfer, Spectroscopy, Stereochemistry, Synthesis, Polymorphism etc., He suffered a paralytic stroke/ Acute Transverse mylitis in Dec 2007 and is 90 %Paralysed, He is bound to a wheelchair, this seems to have injected feul in him to help chemists all around the world, he is more active than before and is pushing boundaries, He has 100 million plus hits on Google, 2.5 lakh plus connections on all networking sites, 100 Lakh plus views on dozen plus blogs, 227 countries, 7 continents, He makes himself available to all, contact him on +91 9323115463, email, Twitter, @amcrasto , He lives and will die for his family, 90% paralysis cannot kill his soul., Notably he has 38 lakh plus views on New Drug Approvals Blog in 227 countries...... , He appreciates the help he gets from one and all, Friends, Family, Glenmark, Readers, Wellwishers, Doctors, Drug authorities, His Contacts, Physiotherapist, etc He has total of 32 International and Indian awards

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