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Basel, Switzerland — Novartis said Friday it received a positive Committee for Medicinal Products for Human Use (CHMP) opinion for Fabhalta as first oral monotherapy for PNH in adults, showing superior hemoglobin improvement without transfusions compared to anti-C5 therapy.

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, life-threatening blood disorder characterized by the destruction of red blood cells, leading to anemia and severe fatigue. It occurs when mutations in blood cell genes cause vulnerability to immune attacks.

This condition can lead to various complications, including blood clots and impaired bone marrow function. Due to its rarity and serious nature, PNH requires specialized medical management, often involving medications that help regulate the immune system’s response and reduce the risk of further complications.

Fabhalta may offer a practice-changing treatment option. The decision is based on Phase III data showing significant efficacy and safety.

If approved by the EC, Fabhalta would be the first oral monotherapy for PNH in Europe.

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By Lisa Luckas

Lisa Luckas is a Sr. Business News Editor at Nobot.News.

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