FDA grants orphan drug status to NovelMed’s PNH treatment

2024-04-16

临床1期孤儿药

来源: Pharmaceutical Technology

Breakdown of PNH RBCs within and outside the blood vessels. Credit: Phonlamai Photo / Shutterstock.com.

NovelMed has announced the receipt of orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug, NM5072, for treating paroxysmal nocturnal hemoglobinuria (PNH).

An alternative pathway blocker anti-properdin antibody, NM5072 selectively hinders the immune system components responsible for the disease, inhibiting alternative pathway cascades and preventing the lysis of PNH red blood cells (RBCs).

By blocking the function of properdin, NM5072 prevents the breakdown of PNH RBCs, which is central to the anaemia associated with PNH.

The drug has concluded a Phase I clinical trial in 48 healthy volunteers with a good safety profile.

It is currently being reviewed for multiple indications by regulators in the US and internationally.

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PNH is a rare disease characterised by chronic inflammation and haemolysis, in which PNH RBCs break down within and outside blood vessels, leading to anaemia and severe health implications.

NM5072, with its longer half-life, represents a significant advancement in the biologic treatment of PNH to improve patient outcomes.

NovelMed CEO Dr Rekha Bansal stated: “PNH is a rare disease that involves a range of blood cells that contribute to debilitating symptoms for patients, including anaemia, fatigue and severe pain with a shorter life span if it remains untreated.

“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.”

The FDA’s ODD provides benefits including seven years of market exclusivity upon the receipt of approval, tax credits for clinical trial costs, fee waivers, reduced annual product fees, assistance for clinical protocol development and the possibility of expedited development programmes.

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