Alexion Pharmaceuticals Inc.'s monoclonal antibody product Soliris won FDA approval in paroxysmal nocturnal hemoglobinuria (PNH), making it the first therapy indicated for the rare genetic disorder.

The Cheshire, Conn.-based firm, which already has recruited a 25-member sales team plus 15 medical science liaisons, will waste no time getting the drug to patients. Soliris' launch is expected "within two weeks," said Alexion CEO Leonard Bell.

The news boosted Alexion's shares (NASDAQ:ALXN) $2.78, or 7.4 percent, Friday, to close at $40.15, though investors still await the drug's pricing, which analysts have projected from $100,000 up to $300,000 in annual costs per patient.

Bell said disclosure of pricing and other marketing details will be announced by the company later this month during a conference call to outline its U.S. commercialization strategy. However, the company did introduce Soliris OneSource, a treatment support service to educate PHN patients and physicians on the diagnosis and treatment of the disease.

As the first PHN therapy to make it to market, Soliris (eculizumab) is indicated as a treatment for virtually all PNH patients. The disease, which is estimated to affect about 8,000 to 10,000 people across North America and Europe, is characterized by hemolysis, the chronic destruction of red blood cells. By selectively blocking terminal complement, the part of the immune system implicated in PNH red blood cell destruction, Soliris succeeded in reducing hemolysis in every patient treated in the clinic.

"One hundred percent of the patients receiving Soliris had objective responses," Bell told BioWorld Today, adding that the drug also was associated with improvement in PNH-associated symptoms, such as anemia, and that patients reported improvements "in their overall quality of life."

Prior to Soliris, the only treatment options for PNH patients have been palliative, such as vitamins, transfusions and blood-thinners. The disorder generally manifests in patients' early 30s, and, once diagnosed, median patient survival is 10 to 15 years. The most common cause of death for PNH patients is blood clots, another area in which Soliris demonstrated positive efficacy. Patients treated with the drug were less likely to suffer thrombosis compared to those on placebo, Bell said.

Data from a 26-week Phase III study, which formed the basis for Alexion's biologics license application submitted in September, showed that Soliris significantly reduced hemolysis in every treated patient, and that reductions occurred within one week of initiating therapy and were sustained for up to 54 months with continued dosing. Preliminary data, reported in January 2006, also showed that the median transfusion rate dropped from 10 units per patient with placebo to 0 units per patient with Soliris, and that hemoglobin stabilization was achieved by 49 percent of patients in the Soliris group compared to none receiving placebo. (See BioWorld Today, Jan. 27, 2006, and Sept. 22, 2006.)

According to study data, the most common adverse events were limited to headache, runny nose, back pain and nausea, though Soliris' product label includes a warning about a potential increase in meningococcal infections and recommends that all patients be vaccinated at least two weeks before starting treatment.

Outside of the U.S., Alexion has a pending marketing authorization application in Europe, and expects a regulatory decision "sometime in the summer," Bell said. The company already has started coordinating its commercialization activities there, and plans to handle all marketing itself on a worldwide basis.

"Our objective is to be on the market in 40 countries within three years of launching in the U.S., so around 2010," he said.

Though Alexion has an early stage pipeline, and has done preclinical work on Soliris in additional indications, those programs will take a backseat to Soliris' upcoming launch, Bell said. "We're a small company, so right now all our efforts are focused on that."