NovelMed’s Blood Disorder Candidate Receives Orphan Drug Designation

The US FDA has granted NovelMed’s drug candidate for the treatment of paroxysmal nocturnal hemoglobinuria an orphan drug designation.  

The candidate has completed early-stage studies on just under 50 individuals, showcasing a low-risk profile.  

Regulators in the US and globally are currently assessing it for multiple indications.  

Paroxysmal nocturnal hemoglobinuria is a rare, acquired disorder characterised by the abnormal breakdown of red blood cells, leading to symptoms such as hemolytic anaemia, thrombosis, and bone marrow failure.  

Dr Rekha Bansal, the head executive at NovelMed, commented, “We are hopeful that NM5072, with its unique mechanism of action that targets the top of the complement cascade, could become a promising treatment to improve outcomes in these patients.” 

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