Fabrazyme® exceptional circumstances lifted to receive full marketing approval
Fabrazyme® exceptional circumstances lifted
to receive full marketing approval
Brussels, Belgium -- Genzyme Corp. (Nasdaq: GENZ) announced today that the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMEA) has adopted a positive opinion to lift the exceptional circumstances and to grant full marketing authorization approval to Genzyme’s product Fabrazyme (agalsidase alfa for the treatment of Fabry disease) in the European Union. To date, Fabrazyme is one of only a few orphan medicinal products in the EU for which the exceptional circumstances have been lifted.
“We are thrilled by the CHMP’s decision to upgrade Fabrazyme to a full approval, based on the review of all the safety and efficacy data, and the completion of the post approval obligations,” said Carlo Incerti, M.D., Head of Research and Development in Europe at Genzyme. “Such a transition of an orphan drug from an approval under exceptional circumstances to a full marketing authorization is significant given the challenges in conducting drug development in small patient populations, like in Fabry disease.”
Orphan medicinal products are commonly granted approval under exceptional circumstances because of the difficulties in completing full requirements when applying for Marketing Authorisation, due largely to the rarity of the diseases, and in view of the lack of other available treatments. Companies then need to fulfill post-approval obligations and undergo an annual reassessment of the safety and efficacy of the product.
Fabrazyme was granted approval under exceptional circumstances in the European Union in August 2001 and, in agreement with the EMEA, a number of post-marketing obligations were undertaken which now have all been satisfactorily fulfilled. The European Commission decision following the CHMP positive opinion is expected within 2 months.
About Fabry Disease
Fabry disease is one of a group of rare diseases called lysosomal storage disorders. It is characterized by excessive accumulation of certain lipids in various organs and tissues, which overtime can cause renal, cardiac, and cerebrovascular events. As a result, patients with Fabry disease typically have a shortened life span, and children must often cope with significant pain and disability. Fabry is an inherited disorder linked to the X chromosome, but females may also develop moderate to severe pathology. It is estimated that 1 in 40,000 males has Fabry disease, whereas the prevalence in the general population is 1 in 117,000 people. The average life expectancy of patients with the disease is 43 years.
Fabrazyme was approved in the European Union in 2001, following completion of a 58-patient placebo-controlled Phase 3 trial that demonstrated the ability of Fabrazyme (at 1 mg/kg every other week) to clear globotriaosylceramide (GL-3) from the blood vessels of the major organs affected in Fabry disease, including kidney, heart and skin. The post-marketing commitments included submission of extension data from the Fabrazyme Phase 3 Extension Study (AGAL-005), the Phase 4 Study (AGAL-008), the Pediatric Study (AGAL-016), and the Fabrazyme Maintenance Dosing Study (AGAL-017). These controlled clinical trials that enrolled a total of 156 patients confirmed the ability of Fabrazyme to stabilize renal function and reduce the risk of serious clinical events. Better outcomes were seen with early treatment intervention. Administration in pediatric patients has been proven to be safe and clinically effective. Genzyme continues to make significant clinical investments in Fabry disease to improve the understanding of this progressive and life-threatening disease.
One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 10,000 employees in locations spanning the globe and 2006 revenues of $3.2 billion. In 2007, Genzyme was chosen to receive the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.
With many established products and services helping patients in nearly 90 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease and other areas of unmet medical need.