Manufactured by US biotechnology company Alexion Pharmaceuticals, the approval of Soliris means an estimated 8,000 to 10,000 people in Europe and North America suffering from paroxysmal nocturnal hemoglobinuria (PNH) will have a treatment for the rare blood disorder, which is defined by chronic red blood cell destruction. The EU approval follows US Food and Drug Administration (FDA) endorsement of the drug in March , which is also the first therapy for PNH in the US.
"As the only therapy approved in both Europe and the United States to treat PNH, EC approval marks another milestone for Soliris and brings hope to patients living with this debilitating disease," Alexion chief executive Leonard Bell said in a statement. "This approval support's Alexion's mission to prescribe Soliris to treat all patients with PNH, and we look forward to making Soliris available throughout Europe as soon as possible."
Additionally, the company has submitted an application for marketing authorisation in Australia. Soliris is a formulation of eculizumab, which is a recombinant humanised monoclonal antibody, which has been purified from murine myeloma cell culture by standard bioprocess technology.
The drug is administered by intravenous infusion, consisting of 600mg every seven days for the first four weeks, followed by 900mg for the fifth dose seven days later, and then 900mg every 14 days thereafter. Prior to Soliris, there were no drugs available for the treatment of PNH. Treatment was limited to symptom management through periodic blood transfusions, non-specific immunosuppressive therapy, and, infrequently, high-risk bone marrow transplantations.
While Soliris does not cure PNH, it works to stop the breakdown of red blood cells by the immune system, which is the most common characteristic of PNH. About one person out of a million will be diagnosed with PNH, which includes the symptoms of sever anaemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark coloured urine (hemoglobinuria), kidney disease, and blood clots.
While the drug will be directed at a small market, analysts have predicted large revenues from the drug. A Merrill Lynch analyst reported in Forbes.com, the sales potential for Soliris could be in the range of $550m (€409m) to $800m (€594m).
In January this year, prior to marketing approval, Alexion initiated sales through a named-patient programme in Europe. Net sales recognised under this were $1m for the three months ended March 31. Alexion expected to introduce Soliris in one of more major markets by the end of this year, with additional countries to follow in 2008.
The European Medicines Evaluation Agency (EMEA) evaluated Soliris under the European Accelerated Assessment Procedure, the fastest evaluation timeframe for approval awarded by the EMEA. According to EMEA, Soliris is the first medicinal product to receive a positive Committee for Human Medicinal Products (CHMP) opinion and approval with Accelerated Assessment Procedure. The EMEA approved Soliris based on clinical data from three multi-national clinical studies involving 195 patients. In these studies Soliris reduced haemolysis in every treated patient, thereby reducing symptoms, stabilizing haemoglobin and significantly reducing transfusions.
But Soliris' blockade of the body's natural immune system does increase the patient's susceptibility to certain serious infections, particularly meningococcal infections.
European labelling includes this warning and states all patients must be vaccinated at least two weeks prior to receiving Soliris.