Date: 2015-10-22
Type of information: Granting of the orphan status in the US
Product name: acalabrutinib
Compound: (S)-4-(8-amino-3-(1-but-2-ynoylpyrrolidin-2-yl)-imidazo[1,5-a]pyrazin-1-yl)-N-(pyridin-2-yl)-benzamide
Therapeutic area: Cancer - Oncology - Rare diseases
Action mechanism: kinase inhibitor/Bruton's tyrosine kinase inhibitor. Acalabrutinib (ACP-196) is a potential best-in-class irreversible oral Bruton's tyrosine kinase (BTK) inhibitor, currently in Phase III development for B-cell blood cancers and in Phase I/II clinical trials in multiple solid tumours.
Company: Acerta Pharma (USA - CA, The Netherlands), now AstraZeneca (UK)
Disease: Waldenstrom macroglobulinemia
Latest news: • On October 22, 2015, the FDA has granted orphan drug designation for (S)-4-(8-amino-3-(1-but-2-ynoylpyrrolidin-2-yl)-imidazo[1,5-a]pyrazin-1-yl)-N-(pyridin-2-yl)-benzamide for the treatment of Waldenstrom macroglobulinemia.
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: 2015-10-22
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
