US biotech company Alexion has been given the green light by European regulators to market its new orphan drug Soliris (eculizumab) - the first therapy to be approved under the EU's new fast-track review process.
Soliris is the only drug approved in Europe to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) - a rare blood disorder defined by chronic red blood cell destruction, or hemolysis.
The nod from the European Medicines Agency (EMEA) comes just two months after Soliris became the first drug endorsed by the EMEA's committee and approved under the Accelerated Assessment Procedure - a review system implemented by the agency last July to speed up the approval of innovate drugs that have "major therapeutic interest".
Drug makers can apply for accelerated assessment if they are able to demonstrate that their product responds to unmet medical needs or constitutes a significant improvement over the available methods of prevention, diagnosis or treatment of a disease.
Under the accelerated route, the standard time period of seven months to give an opinion for a marketing authorisation is reduced to five months.
Last year, the EMEA's Committee for Human Medicinal Products (CHMP) received 13 applications for accelerated assessment, 4 of which were accepted.
Alexion's drug, which reduces hemolysis in patients with PNH, was approved by the US Food and Drug Administration (FDA) last March and has been marketed in the US since April. Alexion has set the price for Soliris at more than $389,000 (€289,000) a year. However it is still unsure what the figure will be for the European market.
PNH affects an estimated 8,000 to 10,000 people in North America and Europe. Patients with PNH can suffer from severe anaemia, chronic fatigue and pulmonary hypertension, and are at increased risk of forming life-threatening blood clots.
Alexion said it is ready to start reimbursement negotiations with healthcare systems in major European countries right now, and expects to launch Soliris - its first marketed drug - in at least one major EU market by the end of the year, with additional countries to follow in 2008.
"The launch order is dictated by the timing of each country's completion of the reimbursement process. Typically, one expects the UK and Germany to be earlier countries, followed by France, Italy, and Spain," the company said.
Currently, Soliris is being made by contract manufacturer Lonza but David Keiser, Alexion's CEO, told Biopharma-Reporter.com that commercial supplies of the drug will eventually be manufactured at the Smithfield, Rhode Island biomanufacturing facility the company bought almost a year ago from Dow for $13m. The firm spent more than $50m to refurbish the 55,000-square-foot plant which now includes two 10,000-liter bioreactors.
"Once the facility is certified and validated by the regulators, we will move the production to the Smithfield site," said Keiser, adding that he expected this to happen at the end of next year.
Alexion's shares fell 2 per cent after the announcement but Keiser said the event was not connected with the approval of Soliris.