The FDA on Friday announced that it expanded approval of Alexion Pharmaceuticals’ Soliris (eculizumab) to include the treatment of atypical Hemolytic Uremic Syndrome (aHUS). “This is the first approval of a drug for treating this life-threatening disease, and the first approval for use of Soliris in children,” commented Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.
The decision, which was made under the agency’s priority review and accelerated approval programmes, was based on data from two single-arm trials in 37 adults and adolescent patients with aHUS and one retrospective study in 19 paediatric patients and 11 adult patients with aHUS. Patients treated with Soliris in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients with aHUS that did not respond to plasma therapy. In addition, patients treated with Soliris experienced improvements in platelet counts and other blood parameters that relate to aHUS disease activity.
The drug was approved with an extension of the existing Risk Evaluation and Mitigation Strategy to inform health care professionals and patients about the risk of meningococcal infections. In addition, the drug will continue to be available only through a restricted access programme. Soliris previously garnered FDA approval in 2007 for the treatment of paroxysmal nocturnal haemoglobinuria.
Also Friday, the European Medicine’s Agency’s Committee for Medicinal Products for Human Use recommended that approval of Soliris be extended to include the treatment of paediatric and adult patients with aHUS. A final decision on this indication is expected in approximately two months.