Date: 2018-08-29
Type of information: Submission of an NDA
Product name: UX007 - triheptanoin
Compound: triheptanoin
Therapeutic area: Rare diseases - Metabolic 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: long-chain 3-hydroxyacyl-coA dehydrogenase deficiency
Latest news:
- • On August 29, 2018, Ultragenyx Pharmaceutical announced that the FDA has accepted Ultragenyx’s most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data. Further details regarding timing will be forthcoming following a pre-NDA meeting, which is being scheduled for the second half of 2018. The data submitted to the FDA for evaluation included the recently published 78-week Phase 2 study results in 29 patients, a now published retrospective medical record review of 20 patients, and 56 emergency IND cardiomyopathy and other patients. In addition, these data were supported by results of a published randomized controlled investigator study of 32 patients showing an effect of triheptanoin on cardiac function. In the 78-week sponsored Phase 2 study, the data showed a 48.1 percent reduction in the mean annualized rate of major clinical events (MCEs) and a 50.6 percent reduction in the median annualized rate of MCEs after 78 weeks of treatment with UX007 compared to an annualized rate of MCEs in the 18 months prior to treatment with UX007. There was also a 50.3 percent reduction in the mean annualized duration of MCEs and a 76.7 percent reduction in the median annualized duration of MCEs following 78 weeks of UX007 treatment. The safety profile was consistent with what has been previously observed with UX007.
- In the EU, Ultragenyx will discuss these data with the European Medicines Agency (EMA) and expects to have an update in the second half of 2018.
- • On 16-18 June, 2015, the Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending triheptanoin for designation as orphan medicinal product for treatment of long-chain 3-hydroxyacyl-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: 2015-07-28
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
