Date: 2016-03-21
Type of information: Granting of the orphan status in the EU
Product name: ALN-GO1 - synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Compound: synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Therapeutic area: Rare diseases - Genetic diseases - Kidney diseases
Action mechanism: RNAi. ALN-GO1 is an investigational RNAi therapeutic targeting the enzyme glycolate oxidase (GO). Primary hyperoxaluria type 1 (PH1) is an autosomal recessive disorder of glyoxylate metabolism, where hepatic detoxification of glyoxylate is impaired due to mutation of the AGXT gene – which encodes the liver peroxisomal alanine-glyoxylate aminotransferase (AGT) enzyme – resulting in excessive oxalate production. Excess oxalate in PH1 patients is unable to be fully excreted by the kidneys leading to the formation of recurrent kidney stones and the deposition of calcium oxalate crystals in the kidneys and urinary tract. Renal damage is caused by a combination of tubular toxicity from oxalate, calcium deposition in the kidneys, and renal obstruction by calcium stones. Compromised kidney function exacerbates the disease as oxalate is released into systemic circulation potentially resulting in subsequent accumulation and crystallization in bones, eyes, skin, heart, and central nervous system, leading to severe illness and death. About 50% of patients will have kidney failure by age 15, and about 80% will have end stage renal disease by age 30. Current treatment options are very limited and although combined organ transplantation of liver and kidneys has been successful, it is a risky procedure and limited due to organ availability.
Company: Alnylam Therapeutics (USA - MA)
Disease: primary hyperoxaluria
Latest news: * On February 25, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues for treatment of primary hyperoxaluria. * On February 08, 2016, the FDA has granted orphan drug designation for synthetic double-stranded (hybridized duplex) ribonucleic acid oligonucleotide specific to hydroxyacid oxidase 1 gene (DCR-PH1) for the treatment of primary hyperoxaluria type 1.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2016-02-08
Orphan status UE: 2016-03-21
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
