Shire submits regulatory approval application for Fabry disease treatment

CAMBRIDGE, Mass. Shire has submitted a regulatory approval application to the Food and Drug Administration for a biotech drug for treating a fatal genetic disease that affects approximately 10,000 people in the world, the company announced Tuesday.

The British drug maker is seeking approval for Replagal (agalsidase alfa), designed to treat Fabry disease. Fabry is a lysosomal storage disorder that causes deficiency of the enzyme alpha-galactosidase A, causing accumulation of the fatty substance Gb3 and resulting in symptoms such as cardiovascular and kidney dysfunction, pain, skin lesions and hearing and eye problems. The disease reduces the average patient’s lifespan by 15 to 20 years.

“We continue to deliver on our commitment to the Fabry community by filing a [biologics license application] to support long-term access to Replagal in the United States,” Shire Human Genetic Therapies president Sylvie Gregoire said. “We understand that this has been a difficult time for patients, and we remain committed to doing all we can to support the Fabry community during the supply shortage and for the long term.”

Replagal won approval in the European Union in 2001 and is available to Fabry patients in the United States under a limited FDA-approved treatment protocol.

In other news, Shire has also submitted approval applications in the United States, Canada and the European Union for velaglucerase alfa, a treatment for Gaucher disease, also a lysosomal disorder.