News

Title
Date
Statement
October 4, 2018

IntraBio Inc today announced that the US Food and Drug Administration has granted Orphan Drug Designation to IB1000s for the treatment of Ataxia-Telangiectasia (A-T). A-T, alternatively known as Louis-Barr disease, refers to an autosomal-recessive cerebellar ataxia disorder caused by mutations in the ataxia telangiectasia mutated (ATM) gene. Mutations in the ATM gene cause progressive degeneration to the […]

October 2, 2018

IntraBio is pleased to share an exciting media advisory shared by US Senator Rand Paul’s office regarding a US Senate Congressional Hearing that will be by the Children and Families Subcommittee of the Senate Health, Education, Labor, and Pensions Committee on the 3rd of October at 2:30 PM Eastern Time. The hearing will discuss the […]

October 1, 2018

IntraBio Inc is pleased to announce that the US Food and Drug Administration has granted a Rare Pediatric Disease Designation for their lead compound series, IB1000s, for the treatment of GM2 Gangliosidoses (Tay-Sachs and Sandhoff diseases). GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and is caused by mutations in the HEXA gene, which disrupt […]

August 1, 2018

IntraBio Inc is pleased to announce that it has been granted orphan designation by the US Food and Drug Administration, and orphan medicinal drug designation by the European Commission, for its lead drug series, IB1000s, for the treatment of Spinocerebellar Ataxias, of which there are currently over 40 known subtypes. Spinocerebellar Ataxias (SCAs) refers to […]

April 30, 2018

IntraBio Inc today announced that the US Food and Drug Administration has granted Orphan Drug Designation to Ursodeoxycholic Acid (UDCA) for the treatment of Niemann-Pick disease type C. Niemann-Pick Disease Type C affects 1:100,000 live births, and is most commonly caused by dysfunction of the NPC1 protein leading to the accumulation of lipids in lysosomes, […]

April 12, 2018

IntraBio Inc is pleased to announce that the US Food and Drug Administration has granted a Rare Pediatric Disease Designation for their lead compound series, IB1000s, for the treatment of Niemann-Pick disease type C. Niemann-Pick Disease Type C (NPC) is a rare, devastating, neurovisceral autosomal-recessive inherited metabolic, lysosomal storage disorder (LSD) that predominantly affects pediatric […]

March 28, 2018

IntraBio Inc has announced that it has been granted orphan designation by the US Food and Drug Administration for its lead drug series, IB1000s, for therapeutic use in the rare lysosomal storage disorders GM2 Gangliosidoses (Sandhoff and Tay-Sachs Disease). GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and is caused by mutations in the HEXA […]

February 25, 2018

IntraBio Inc has announced that it has been granted orphan designation by the US Food and Drug Administration for its lead drug series, IB1000s, for therapeutic use in the rare lysosomal storage disorders Niemann-Pick disease type C. Niemann-Pick Disease Type C affects 1:100,000 live births and is most commonly caused by dysfunction of the NPC1 […]

October 30, 2017

IntraBio Ltd is pleased to announce that it has been granted orphan medicinal drug designation by the European Commission for its lead drug series, IB1000s, for therapeutic use in the rare lysosomal storage disorders GM2 Gangliosidoses (Sandhoff and Tay-Sachs Disease). GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and is caused by mutations in the […]

September 15, 2017

IntraBio Inc. is pleased to announce significant progress in the treatment of rare and neurodegenerative diseases with IB1000, a well-tolerated, modified amino acid that is administered orally. Most notably, IB1000 is showing promising effects in Phase 2 observational clinical studies in patients with Niemann-Pick Type C (NPC) disease, Late-Onset Tay Sachs (LOTS) disease, and different […]

June 1, 2017

IntraBio Ltd, a clinical stage biopharmacuetical company developing treatments for rare lysosomal storage disorders and neurodegenerative diseases, today announced that the European Commission has granted Orphan Drug Designation (EU/3/17/1878) to Ursodeoxycholic Acid (UDCA) for the treatment of Niemann-Pick disease (NPC) Type A, B, and C. “We are pleased to receive orphan designation for UDCA, which […]

April 1, 2017

IntraBio Ltd has announced that it has been granted orphan designation (EU/3/17/1848) by the European Commission on its amino acid analogue-based drug product (IB 1000), for therapeutic use in the rare lysosomal storage disorders Niemann-Pick disease type C and also disease Types A & B. IntraBio, with its collaborators, have completed multiple observational clinical studies […]