The European Medicines Agency Committee for Medicinal Products for Human Use has recommended approval of Alexion Europe’s Soliris (eculizumab), the first human medicine successfully to complete the European Union’s accelerated assessment procedure.
Soliris, a designated orphan drug for reducing haemolysis (destruction of red blood cells) in patients with the rare blood disorder paroxysmal nocturnal haemoglobinuria, was reviewed in 147 days, within the time limit of 150 days set by the EU legislation for accelerated assessment. The CHMP’s positive opinion was also the first for a product filed under the accelerated-assessment procedure by a company that has benefited from specific EU incentives for small and medium-sized enterprises.
Accelerated assessment was introduced by the revised EU pharmaceutical legislation (Article 14 (9) of Regulation (EC) No 726/2004) in November 2005, with the aim of speeding up patient access to new medicines of major public-health interest. Companies using the procedure need to demonstrate that their product addresses an unmet medical need or presents a significant improvement over available methods of preventing, diagnosing or treating a condition.
As the CHMP’s guideline on accelerated assessment notes, there is no single definition of what constitutes a major public health interest in terms of the procedure. Applicants must justify their eligibility for accelerated assessment on a case-by-case basis. Last year the CHMP received 13 requests for accelerated assessment, four of which were accepted.
The US-based Alexion Pharmaceuticals expects to receive final EU approval for Soliris within the next two to three months. The product is already on the US market, following approval by the Food and Drug Administration for the treatment of PHN to reduce haemolysis on 16 March. Currently there are no therapies available specifically for the treatment of PHN in Europe, Alexion said.