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Share this article Share this article CHARLOTTESVILLE, Va., Dec. 16, 2020 /PRNewswire/  HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to HemoShear s HST5040 oral small molecule for treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA).  HST5040 was granted Fast Track and Rare Pediatric Disease designations to treat MMA and PA earlier in 2020. This past 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. The diseases result in the buildup of toxic metabolites that can lead to frequent metabolic decompensations, severe organ damage, seizures, developmental deficits, and pr

United statesJim powersDrug administrationHemoshear therapeuticsOrphan drug designationsFast trackRare pediatric diseaseInvestigational new drugDrug designationShear therapeuticsஒன்றுபட்டது மாநிலங்களில்ஜிம் பவர்ஸ்வேகமாக டிராக்ரேர் குழந்தை நோய்மருந்து பதவிவெட்டு சிகிச்சை