Orphan drug designation for SiSaf's osteopetrosis ADO2

UK-based RNA delivery and therapeutics company SiSaf has announced that SIS-101-ADO, its siRNA therapeutic for patients with autosomal dominant osteopetrosis type 2 (ADO2), has been granted orphan drug designation by the US Food and Drug Administration (FDA).

Due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has also been granted rare pediatric disease designation for the treatment of autosomal dominant osteopetrosis.

As a result of the orphan drug designation, SiSaf will benefit from incentives such as tax credits for clinical trials, exemption from user fees, and expanded marketplace exclusivity.