NICE Issues Final Opinion For Alexion's Soliris For aHUS

Alexion announced that the National Institute for Health and Care Excellence (NICE) Highly Specialized Technologies Evaluation Committee (EC) has given its final positive recommendation for Soliris (eculizumab) as treatment for patients in England with atypical hemolytic uremic syndrome (aHUS).

Eculizumab is a first-in-class terminal complement inhibitor that works to reduce hemolysis or destruction of red blood cells. The drug is indicated for paroxysmal nocturnal hemoglobinuria (PNH) and is being investigated in other indications, including neuromyelitis optica (NMO) and Myasthenia Gravis (MG). Eculizumab has also received approvals as treatment for aHUS in the U.S., EU, Japan, and other countries.

Atypical hemolytic uremic syndrome is an ultra-rare, life-threatening, and debilitating genetic disorder marked by the formation of blood clots in small vessels. This could lead to sudden and major damage to the brain, heart, kidney, and other organs, which in turn could end in premature death. Indeed, 79 percent of all aHUS patients die or sustain permanent kidney damage within three years of diagnosis.

Leonard Bell, Chairman and CEO of Alexion, said, “Today's decision is an important victory for patients with aHUS and physicians who now have assurance that they will have access to the life-transforming efficacy of Soliris. We are pleased that Soliris will be made available on the NHS for patients with aHUS and we commend NHS England for its previous decision to provide interim funding to patients for the extended period it took for NICE to make this final determination.”

The company added that NICE’s final assessment reiterates the same conditions it gave in its previous draft recommendation, which required the company to establish an expert center and monitoring system for patients receiving eculizumab. This is due to the fact that some patients who received and later stopped treatment with Soliris experienced complications including end-stage renal failure and respiratory distress.

The news comes on the heels of Alexion’s announcement that it has received Orphan Drug Designation from Japan's Ministry of Health, Labor, and Welfare (MHLW) for the drug as treatment for neuromyelitis optica (NMO), a neurological disorder.