Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that Soliris
(eculizumab), the company’s first-in-class terminal complement inhibitor, has received a positive opinion for orphan medicinal product designation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) for the treatment of neuromyelitis optica (NMO), a life-threatening, ultra-rare neurological disorder. The positive opinion of the COMP has now been forwarded to the European Commission for final approval and publication in the community register. Soliris is not approved in any country for the treatment of patients with NMO.
“We are pleased that the COMP has provided a positive opinion regarding orphan medicinal product status for Soliris for the treatment of patients with NMO, particularly after having been recently granted orphan-drug designation by the FDA,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “NMO is an extremely rare, chronic and debilitating disease that can lead to paralysis, blindness and death, and there are no approved therapies for it. Therefore, this news represents another positive step toward meeting the needs of this underserved patient population in the EU.”
The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product status designation would provide Alexion with certain benefits and incentives, including a period of marketing exclusivity.
In patients with NMO, uncontrolled complement activation causes destruction of myelin-producing cells, leading to severe damage to the central nervous system (CNS), including the spinal cord and optic nerve.
The disease leads to severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death.
Patients with NMO have a life-long exposure to the uncontrolled complement activation due to chronic autoimmune attack, and most patients experience an unpredictable, relapsing course of disease with cumulative disability, as each attack adds to the neurologic disability.
Fifty percent of relapsing NMO patients have been reported to sustain permanent severe disability, including paralysis and blindness, within five years of disease onset.
Most NMO-related deaths result from respiratory complications from NMO attacks.
The disease primarily affects women, with a female to male ratio as high as a 9:1.
Phase 2 Data in Patients with NMO
Phase 2 data presented at the 2012 annual meeting of the American Neurological Association (ANA) and subsequently published in
The Lancet Neurology
showed Soliris treatment was associated with a significant reduction in the frequency of relapses (recurring attacks) in patients with severe, relapsing NMO.
The study met its primary efficacy endpoint, reduction in annualized relapse rate, with high degrees of clinical and statistical significance. Additionally, Soliris was associated with significant improvements in key secondary endpoints.
Soliris was generally well-tolerated, with the three most common adverse events being headache, nausea, and dizziness.
One case of meningococcal sepsis occurred. The patient made an uneventful recovery and restarted treatment with eculizumab to complete the study.
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the United States, European Union (EU) and other countries as the first and only treatment for aHUS patients. Soliris is indicated to inhibit complement-mediated TMA. Soliris is not indicated for the treatment of patients with Shiga toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). Alexion is evaluating the safety and efficacy of Soliris for the treatment of patients with STEC-HUS.