Posted by Michael Wonder on 28 Jul 2020
HemoShear Therapeutics receives FDA fast track and rare paediatric disease designations for HST5040 to treat methylmalonic acidaemia and propionic acidaemia
28 July 2020 - HemoShear Therapeutics has received fast track and rare paediatric disease designations from the U.S. FDA for HST5040, a once-daily oral small molecule drug being developed to treat methylmalonic acidaemia and propionic acidaemia.
In June, HemoShear received clearance from the FDA for its Investigational New Drug application to conduct a phase 2 clinical study of HST5040 in patients with methylmalonic acidaemia and propionic acidaemia.
Methylmalonic acidaemia and propionic acidaemia are rare genetic disorders caused by the deficiency of certain enzymes required to metabolise amino acids. The diseases result in the rapid buildup of life-threatening metabolites that can lead to severe organ damage, seizures, developmental deficits, and premature death. HemoShear's phase 2 clinical study, HERO (HElp Reduce Organic Acids), is designed to enrol at least 12 patients with MMA and PA at select children's hospitals in the United States.
