Paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder, shows a triad of features: intravascular hemolysis, thromboembolic events, and cytopenia. Manifestations vary among patients, making classification based on typical presentations challenging due to the disease's unpredictable nature.

In 2023, the Paroxysmal Nocturnal Hemoglobinuria diagnosed prevalent cases were ~12,000 cases in the 7MM, which might reach ~13,000 cases by 2034. In the 7MM, the highest number of paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases were observed in the US.

Until 2007, PNH was a devastating disease without treatment for hemolysis and thrombosis, the leading cause of death in patients. However, the last decade saw a revolutionary shift with the advent of the anti-C5 agent eculizumab. It significantly reduced hemolysis, decreased transfusion dependency, and, crucially, lowered the thrombosis rate.

The disease-modifying therapeutic strategy for paroxysmal nocturnal hemoglobinuria includes complement inhibition therapy, with drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI approved by the FDA, and considered the gold standard. SOLIRIS, the pioneering therapy for PNH, was succeeded by ULTOMIRIS, both developed by Alexion Pharmaceuticals, offering C5 inhibitors as essential therapeutic options. 

Recent approvals of factor B and D inhibitors like FABHALTA (iptacopan) in the US and VOYDEYA (danicopan) in Japan aim to offer improved treatment with fewer side effects.

Learn more about the FDA-approved paroxysmal nocturnal hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal Hemoglobinuria Treatment

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended crovalimab for treating PNH in adults and adolescents aged 12 and above in June 2024, based on findings from the global Phase III COMMODORE 2 clinical trial.

The FDA accepted a biologics license application for crovalimab in September 2023 and subsequently approved it in June 2024 for treating PNH in adults and adolescents aged 13 years and older. Genentech, a subsidiary of Roche, has made the drug available in the US.

In Japan, Chugai Pharmaceutical submitted a new drug application for PiaSky to the Japan Ministry of Health, Labour, and Welfare (MHLW) in June 2023. The drug was approved in March 2024 and launched in May 2024 for patients with PNH aged 12 years and above.

In February 2024, China's National Medical Products Administration (NMPA) approved crovalimab for treating PNH in adults and adolescents aged 12 years and older who have not previously received complement inhibitor treatment, following a priority review that began in August 2022.

PiaSky is also undergoing review by other regulatory authorities, including those in Europe and Taiwan.

 

Source: prnewswire.com

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