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Novartis’s Fabhalta Reaches First Stop On Regulatory Journey With PNH Nod

 
• By Alaric DeArment

Novartis envisions the drug, the first oral monotherapy for PNH, as a “pipeline in a pill” that could see $3bn in peak sales, with a Phase III readout in IgAN in October and one in C3G expected this month.

Novartis won FDA approval for Fabhalta in PNH • Source: Shutterstock

Novartis AG’s Fabhalta (iptacopan) reached its first market milestone with US Food and Drug Administration approval for paroxysmal nocturnal hemoglobinuria (PNH) as the first oral monotherapy for patients with the disease. But PNH is just the first of several development milestones Novartis has planned for the drug.

The Swiss drug maker has called Fabhalta a potential pipeline-in-a-pill, and it is in the clinic for additional disease states, which could help it reach peak annual

Key Takeaways

  • Novartis’s Fabhalta won FDA approval as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria.

  • The drug maker envisions it as a “pipeline-in-a-pill” and is developing it for a wide range of complement-mediated diseases, with potential peak sales of more than $3bn

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