Pfizer's Macugen approved by European Commission

The European Commission has granted Pfizer (New York, US) marketing authorisation for Macugen (pegaptanib sodium injection).

An ophthalmic drug for the treatment of neovascular (wet) age-related macular degeneration (AMD) - a chronic, progressive disease where abnormal blood vessel growth beneath the macula (the central portion of the retina) causes bleeding and other fluid accumulation in the retina and consequently impairs central vision - it targets vascular endothelial growth factor (VEGF 165), the protein that triggers the abnormal blood vessel growth.

Contraindicated in patients with active or suspected ocular or periocular infection, or with known hypersensitivity to the active or inactive substances, its approval was based on the results of two pivotal clinical trials involving 1,186 patients with all subtypes of wet AMD. The primary efficacy endpoint was set as the proportion of patients protected from a three-line loss of visual acuity on the eye chart by week 54. 70% of those who received 0.3 mg of Macugen every six weeks lost fewer than three lines of vision on the eye chart, compared with 55% in the control group.

After one year, patients were randomised to continue or discontinue treatment for another year, and two-year clinical data demonstrated a continued treatment benefit. The drug was well tolerated, and two-year patients received over 90% of possible injections, indicating strong compliance and acceptance to therapy. 'Most of the adverse events reported over the two years were mild in severity, transient and attributed by investigators to the injection procedure rather than the study drug', said Pfizer. The drug's safety and efficacy beyond two years have not been demonstrated.

'While the problem of wet AMD is a global one, it hits particularly hard in areas with rapidly aging populations, such as Europe,' said Steve Winyard, chairman of the AMD Alliance International. 'Each year there are an estimated 500,000 new cases of wet AMD worldwide, so new treatment options are crucially important.'

Macugen is approved in the US, Canada, Brazil, Argentina, Peru, Pakistan and the Philippines. It was used to treat more than 50,000 patients in the US in 2005.