Posted by Michael Wonder on 24 Sep 2020
FDA grants Spirovant Sciences orphan drug and rare paediatric disease designations for SPIRO-2101 for treatment of cystic fibrosis
24 September 2020 - Spirovant Sciences today announced that the U.S. FDA has granted orphan drug and rare paediatric disease designations to its lead product candidate, SPIRO-2101, for the treatment of cystic fibrosis.
SPIRO-2101, an inhaled adeno-associated virus (AAV) gene therapy, is designed to replace a defective cystic fibrosis transmembrane conductance regulator gene in patients with class 1 mutations or in those who are unable to tolerate an existing CFTR modulator.
SPIRO-2101 contains an evolved AAV capsid engineered to have high tropism to human airway epithelia.
