Date: 2015-09-29
Type of information: Granting of the orphan status in the US
Product name: RGX-111
Compound: adeno-associated virus vector serotype 9 expressing human a-L-iduronidase
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: gene therapy. RGX-111 uses the AAV9 vector to deliver the human α-l-iduronidase (IDUA) gene to the central nervous system (CNS). Mucopolysaccharidosis Type I (MPS I) is caused by deficiency of IDUA, an enzyme required for the breakdown of polysaccharides heparan sulfate and dermatan sulfate in the lysosomes of cells. Many patients develop symptoms related to glycosaminoglycan storage in the CNS, which can include excessive accumulation of fluid in the brain (hydrocephalus), spinal cord compression and cognitive impairment. Delivery of the enzyme within cells in the CNS could provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occur in MPS I patients following bone marrow transplant.
Company: RegenXBio (USA - MD)
Disease: mucopolysaccharidosis type I (MPS I)
Latest news: * On December 30, 2015, RegenX Bio announced that the FDA)has granted Rare Pediatric Disease Designation to RGX-111, the Company’s investigational gene therapy product candidate for the treatment of mucopolysaccharidosis Type I (MPS I). "The Rare Pediatric Disease Designation builds upon the Orphan Drug Designation granted earlier this year by the FDA to RGX-111 for MPS I, underscoring the therapy’s potential to provide meaningful benefit to children struggling with this severely debilitating disease,” said Kenneth T. Mills, President and Chief Executive Officer of RegenX Bio. * On September 29, 2015, the FDA has granted orphan drug designation to RegenXBio\'s investigational gene therapy product candidate RGX-111 for the treatment of mucopolysaccharidosis Type I (MPS I). RegenXBio intends to file an Investigational New Drug Application (IND) with the FDA in the first half of 2016 to support the initiation of a dose-escalation Phase I/II clinical trial of RGX-111 beginning in the first half of 2016.
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: 2015-09-29
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
