Date: 2016-03-21
Type of information: Granting of the orphan status in the EU
Product name: DTX301 - recombinant adeno-associated virus serotype AAV8 vector encoding human ornithine transcarbamylase
Compound: recombinant adeno-associated virus serotype AAV8 vector encoding human ornithine transcarbamylase
Therapeutic area: Rare diseases - Metabolic diseases
Action mechanism: gene therapy. Designed to address the underlying genetic defect, DTX301 delivers stable expression and activity of ornithine transcarbamylase. DTX301 has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism, which if elevated, can cause serious neurological deficiencies in patients.
Company: Dimension Therapeutics (USA - MA)/Pharma Gateway (Sweden)
Disease: ornithine transcarbamylase deficiency
Latest news: * On February 25, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase for treatment of ornithine transcarbamylase deficiency. Dimension has completed candidate selection in its DTX301 program and plans to submit an investigational new drug (IND) application with the FDA in the second half of 2016. * On December 29, 2015, the FDA has granted orphan drug designation for recombinant adeno-associated virus serotype AAV8 vector encoding human ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency.
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: 2015-12-29
Orphan status UE: 2016-03-21
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
