Alexion Posts Data For Soliris In aHUS At ASH 2014

Alexion presented findings for its drug Soliris (eculizumab) in the treatment of atypical hemolytic uremic syndrome (aHUS), as well as new data regarding medical care for patients with aHUS and paroxysmal nocturnal hemoglobinuria (PNH) at the 56th Annual Meeting of the American Society of Hematology (ASH) held in San Francisco this month.

Soliris is a first-in-class terminal complement inhibitor indicated for the treatment of ultra-rare blood disorder PNH. The drug has also received approval in almost 40 countries to treat the ultra-rare genetic disease aHUS. Soliris was recently awarded orphan status in Japan for the treatment of neurological disorder neuromyelitis optica (NMO).

The company’s presentation featured the post-hoc analyses of Soliris treatment in adult and pediatric patients with aHUS who were with and without an identified genetic mutation. Alexion also included findings from its OPTIMA trial, which investigated high sensitivity flow cytometry in the detection of PNH cells in various patient populations.

Dr. Spero R. Cataland of Ohio State University Medical Center presented the results of two post-hoc sub-analyses from two trials investigating Soliris’ safety and efficacy in children and adult patients with aHUS with or without identified genetic mutations at baseline. Results show that platelet count normalization was achieved by 100 percent in both pediatric and adult patients with an identified mutation. Platelet count normalization was met by 91 percent of pediatric and 95 percent of adult patients without an identified mutation.

“Given the life-threatening nature of aHUS and the well-established clinical efficacy of Soliris, these data provide additional evidence for initiating treatment with Soliris immediately upon clinical diagnosis of aHUS. This is particularly important since genetic testing can take several months to complete and, to date, genetic complement mutations can only be identified in 50 percent to 70 percent of patients with aHUS,” said Dr. Cataland.

The findings suggest that treatment with Soliris led to clinically meaningful improvements in hematologic and renal parameters in both patient groups with aHUS whether or not an identified genetic mutation was present.

Leonard Bell, Chairman and CEO of Alexion, said, “We are pleased that the data presented at ASH continue to expand our understanding of aHUS and PNH… Importantly, significant improvements in hematologic and renal outcomes were observed in pediatric and adult patients with aHUS, both with and without identified genetic mutations, supporting the early initiation of Soliris treatment regardless of mutation status.”

Earlier this month, Soliris received the backing of the U.K.’s the National Institute for Health and Care Excellence (NICE) Highly Specialized Technologies Evaluation Committee (EC) as treatment for aHUS for patients in England.