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news.Alexion Pharmaceuticals has received orphan drug designation (ODD) from Japan’s Ministry of Health, Labour and Welfare (MHLW) for Soliris (eculizumab) to treat patients with neuromyelitis optica (NMO), a life-threatening, ultra-rare neurologic disorder.
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In NMO patients, chronic, uncontrolled complement activation results in severe damage to the central nervous system (CNS), mainly impacting the optic nerve and spinal cord.
This disease is characterized by severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death.
Alexion R&D executive vice-president and global head Martin Mackay said: "The orphan drug designation for eculizumab for NMO highlights the significant need for an effective and innovative treatment option for patients in Japan suffering from this debilitating and life-threatening disease.
"By specifically inhibiting the terminal complement pathway, eculizumab has the potential to improve outcomes for patients with NMO.
"We look forward to evaluating the clinical benefits of eculizumab in NMO in our registration study, known as PREVENT, which is currently enrolling patients."
Currently, the company is enrolling patients in a multinational, placebo-controlled registration trial (PREVENT) of eculizumab in patients with relapsing NMO.
Soliris is a first-in-class terminal complement inhibitor and is currently approved in the US, EU, Japan and other countries to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
PNH and aHUS are two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation.
The company said that Soliris is not approved in any country for the treatment of NMO.