Date: 2015-06-19
Type of information: Granting of the orphan status in the EU
Product name: UX007 - triheptanoin
Compound: triheptanoin
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism:
- triglyceride. Triheptanoin is a purified, pharmaceutical-grade, specially designed synthetic triglyceride compound created via a multi-step chemical process. Triheptanoin is metabolized to and intended to provide patients with heptanoate, which can diffuse across the blood-brain barrier and be converted into glucose. Heptanoate can also be further metabolized to four- and five-carbon ketone bodies in the liver that also cross the blood-brain-barrier and provide an additional energy source to the brain. Heptanoate and five-carbon ketone bodies can also regenerate new glucose in the brain, which is deficient in these patients.
Company: Ultragenyx Pharmaceutical (USA - CA)
Disease: very long-chain acyl-CoA dehydrogenase deficiency
Latest news:
- • On 12-13 May 2015, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for triheptanoin for treatment of very long-chain acyl-CoA dehydrogenase 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:
Orphan status UE: 2015-06-19
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
