Date: 2015-12-29
Type of information: Granting of the orphan status in the US
Product name: GBT440 - 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl) methoxy)benzaldehyde
Compound: 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl) methoxy)benzaldehyde
Therapeutic area: Genetic diseases - Hematological diseases - Rare diseases
Action mechanism: GBT440 is being developed as an oral, once-daily therapy for patients with sickle cell disease. GBT440 works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, Global Blood Therapeutics believes GBT440 blocks polymerization and the resultant sickling of red blood cells (RBCs). With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT440 may be capable of modifying the progression of sickle cell disease.
Company: Global Blood Therapeutics (USA - CA)
Disease: sickle cell disease
Latest news: * On December 29,2015, the FDA has granted orphan drug designation for 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl) methoxy)benzaldehyde for the treatment of sickle cell disease. Currently, GBT440 is being investigated in an ongoing Phase 1/2 clinical trial in both healthy subjects and SCD patients.
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:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
