FDA Approves Add-on Danicopan In Patients With PNH, EVH

The US Food and Drug Administration (FDA) has approved danicopan (VOYDEYA™; Alexion, AstraZeneca Rare Disease) as an add-on therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH) with clinically significant extravascular hemolysis (EVH), according to a press release.

Danicopan is a first-in-class Factor D inhibitor developed to improve treatment outcomes in the 10% to 20% of patients with PNH who experience EVH while undergoing standard-of-care therapy with ravulizumab (ULTOMIRIS®) or eculizumab (SOLIRIS®).

“The approval of VOYDEYA offers this small subset of PNH patients an add-on therapy designed to address EVH, while maintaining disease control with ULTOMIRIS or SOLIRIS,” stated Bart Scott, MD, of University of Washington Medical Center and Fred Hutchinson Cancer Center. “Terminal complement inhibition with ULTOMIRIS can address the life-threatening complications of PNH, building on the efficacy and safety of SOLIRIS established over nearly 20 years.”

PNH Treatment Approved by FDA

The approval by the FDA is supported by primary and long-term findings from the pivotal phase III ALPHA trial. ALPHA data showed that danicopan improved hemoglobin, fatigue score, and transfusion avoidance in patients with PNH and EVH.

“As the ALPHA trial suggests, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for this subset of patients with EVH, enabling them to continue with proven standard-of-care therapy.” wrote Marc Dunoyer, Chief Executive Office of Alexion.

Previously, danicopan was granted the Breakthrough Therapy designation by the FDA and the Priority Medicines status by the European Medicines Agency. Danicopan has also received Orphan Drug Designation in the United States, European Union, and Japan for PNH.

Related: Adjunct Danicopan for PNH with EVH Maintains Superiority Versus Placebo in ALPHA