Shire has submitted a Biologics License Application to the US Food and Drug Administration (FDA) for Replagal, an enzyme replacement therapy for Fabry disease.
Replagal first received marketing authorisation in the EU in 2001, and is approved for the treatment of Fabry disease in 45 countries.
Replagal is available to US Fabry patients under an FDA-approved treatment protocol, and the company is also supporting emergency IND requests.
Fabry disease is a lysosomal storage disorder that interferes with the body's ability to break down a specific fatty substance, resulting in symptoms such as cardiovascular and/or renal dysfunction, intense or burning pain, heat intolerance, skin lesions, gastrointestinal complaints, hearing loss and ocular problems.
