NICE Recommends Alexion's Eculizumab For aHUS

Alexion Pharmaceuticals announced that it has received positive recommendation from the National Institute for Health and Clinical Excellence (NICE) for its drug eculizumab as treatment for the rare disease atypical hemolytic uremic syndrome (aHUS) in the U.K.

Eculizumab (brand name Soliris) is a terminal complement inhibitor that works to reduce hemolysis or destruction of red blood cells. The drug is also indicated for the inhibition of complement-mediated thrombotic microangiopathy, or TMA (blood clots in small vessels). Soliris is approved in the U.S., EU, and Japan, among other countries for the treatment of ultra-rare disease paroxysmal nocturnal hemoglobinuria (PNH) and aHUS. In August, the drug was also granted Orphan Drug Designation by the European Commission for the treatment of rare neurologic disorder Myasthenia Gravis (MG).

Atypical hemolytic uremic syndrome is characterized by the formation of blood clots in small vessels throughout the body. This blockage results in life-long risk for TMA, which can erupt in sudden and deadly damage to the kidney, heart, and brain, and even cause premature death.

The NICE’s Highly Specialized Technologies Evaluation Committee (EC) gave its positive opinion for the commissioning of eculizumab subject to certain conditions. While acknowledging its efficacy, the Committee cited the drug’s high price as a concern. NICE Chief Executive, Sir Andrew Dillon, said, “Eculizumab radically improves the quality of life of the small number of people with aHUS… The drug is, however, very expensive. In making its decision, the independent Evaluation Committee needed to take into account the total fixed budget for highly specialized services as a whole and how it is allocated.”

Alexion said it looks forward to NICE’s confirmation of the final policy for eculizumab. Leonard Bell, CEO of Alexion, said, “We are pleased that NICE has once again confirmed that… eculizumab is a significant breakthrough for patients with this devastating disorder. Alexion supports the use of eculizumab consistent with the EMA-approved label, which specifically directs that treatment is recommended to continue for the patient's lifetime, unless the discontinuation of eculizumab is clinically indicated.” Bell said that the company will provide scientific comments to NICE about its conditions for commissioning the drug in order to bring Soliris to aHUS patients in England.