Replagel cleared for pediatric Fabry disease

The European Medicines Agency (EMEA) has announced a licence variation for Replagel (agalsidase alfa) to include children with Fabry disease from seven years of age. UK-based drugmaker Shire's product is now the only enzyme replacement therapy approved for use in this age group.

The EMEA's decision was based on new safety and exploratory efficacy data, which demonstrated that Replagel, delivered as a 40-minute intravenous infusion every other week, was well-tolerated. More than half of the patients on the drug being treated for symptoms of neuropathic pain, one of the most frequent, debilitating and early clinical manifestations of Fabry disease, were able to reduce or stop the use of pain medications. Shire added that all indices of heart rate variability that were decreased at baseline, improved significantly during treatment.

Shire noted that, as children often remain untreated, this news brings hope not only to Fabry children but also to their families. Uma Ramaswami, from the department of pediatric endocrinology, diabetes and metabolism, at Addenbrooke's Hospital, Cambridge, UK, added that "long-term studies are now needed to assess the hypothesis that early initiation of ERT in childhood offers the potential to change the natural course of the disease."