Basilea Pharmaceutica has obtained FDA orphan drug status to isavuconazole, an investigational intravenous and oral broad-spectrum antifungal, for the treatment of invasive aspergillosis.
Isavuconazole is being investigated in phase 3 studies for the treatment of severe invasive fungal infections.
Basilea chief medical officer Prof. Achim Kaufhold said invasive aspergillosis affects patients with an impaired or weakened immune system and is associated with high mortality.
"The granting of orphan designation for isavuconazole in the U.S. reflects the high medical need and is an important regulatory milestone for Basilea and our partner Astellas," Kaufhold added.
Results from two phase 3 studies of isavuconazole, which include SECURE, VITAL and ACTIVE, are expected in the latter half of 2013.
SECURE phase 3 registration study will assess the safety and efficacy profile of isavuconazole administered once a day against voriconazole given twice a day for the primary treatment of invasive fungal disease caused by Aspergillus species.
The open-label VITAL study will evaluate isavuconazole as a treatment for patients with invasive fungal disease caused by emerging fungi and the treatment of aspergillosis patients with pre-existing renal impairment
Recruitment for isavuconazole ACTIVE phase 3 study, which is designed to assess isavuconazole i.v. and oral versus caspofungin i.v. followed by oral voriconazole against Candida infections, will carry on into 2014.
