Charlottesville, Va., July 28, 2020 – HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, has received Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for HST5040, a once-daily oral small molecule drug being developed to treat methylmalonic acidemia (MMA) and propionic acidemia (PA).
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 MMA and PA. MMA and PA are rare genetic disorders caused by the deficiency of certain enzymes required to metabolize amino acids.