PNH treatment Fabhalta recommended for EU approval

Therapy would be 1st oral stand-alone treatment available in Europe

Marisa Wexler MS avatar

by Marisa Wexler MS |

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A European Medicines Agency committee has recommended that Novartis’ twice-daily oral therapy Fabhalta (iptacopan) be approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

The recommendation from the Committee for Medicinal Products for Human Use (CHMP) supports Fabhalta’s use as a monotherapy, or stand-alone treatment, in adults with PNH who have hemolytic anemia, a condition that occurs when red blood cells are destroyed faster than they are made.

The European Commission, which has final say in approvals of treatments in the European Union, will review the committee’s recommendation. The commission is not required to abide by the CHMP’s recommendations, but it usually does. A final decision is expected within two months.

“If approved by the European Commission, Fabhalta would be the first oral monotherapy available to PNH patients in Europe,” Patrick Horber, MD, president of the international unit for Novartis, said in a company press release.

“With current standard of care, PNH symptoms are often uncontrolled, while patients endure regular and time-consuming infusions,” Horber said. “This oral therapy could provide a much-needed alternative to support many people living with PNH who often have to structure their lives around managing their condition.”

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Targeting a different protein

In PNH, a part of the immune system called the complement cascade is activated to destroy blood cells, particularly red blood cells. Fabhalta, which was approved in the U.S. in December 2023, works to stop red blood cell destruction by blocking a complement protein called factor B.

The CHMP’s recommendation was based on data from two Phase 3 clinical trials, APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530).

The APPLY-PNH study tested the therapy against Soliris (eculizumab) and Ultomiris (ravulizumab), the first two treatments approved for PNH in the U.S. Both are given by infusion and target a complement protein called C5.

APPLY-PNH enrolled 97 patients who continued to experience substantial disease symptoms despite being on those older treatments. Results showed that Fabhalta was better at preventing anemia in these patients, normalizing the levels of hemoglobin, the protein that carries oxygen in red blood cells, and reducing fatigue. Most trial participants didn’t require blood transfusions over the course of a year on the oral therapy.

The APPOINT-PNH trial tested Fabhalta in 40 PNH patients who hadn’t previously received other treatments. The findings were consistent with results from the other study, indicating that Fabhalta could reduce anemia. As in APPLY-PNH, most patients didn’t require blood transfusions.

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Potential ‘practice-changing’ PNH treatment

“With a robust body of evidence and a demonstrated safety profile, Fabhalta could be practice-changing for patients, helping relieve burdens experienced by people living with PNH,” said Antonio Risitano, MD, PhD, president of the International PNH Interest Group, a coalition of researchers and clinicians dedicated to advancing and improving PNH treatments.

“In clinical studies, oral [Fabhalta] demonstrated superior hemoglobin improvement without the need for red blood cell transfusions compared to anti-C5 therapies, leading to normalization of hemoglobin in the majority of patients — a potentially groundbreaking benefit for those living with this chronic blood disorder,” Risitano said.

A report from the Institute for Clinical and Economic Review, an independent nonprofit group that assesses the efficacy and value of medicines, found that Fabhalta’s U.S. list price of $550,377 represented “low” long-term cost-effectiveness.