Shire plc (SHPGY) Submits License Application for REPLAGAL®

In the company’s news yesterday,

Shire plc announced that it has submitted a Biologics License Application (BLA) with the FDA for REPLAGAL®, the company’s enzyme replacement therapy for Fabry disease. REPLAGAL first received marketing authorization in the EU in 2001, and is approved for the treatment of Fabry disease in 45 countries.

Fabry disease is a rare X-linked recessive (inherited) lysosomal storage disease that can cause a wide range of systemic symptoms. The disease interferes with the body’s ability to break down a specific fatty substance (globotriaosylceramide or Gb3) which accumulates within the body due to deficiency of a specific enzyme (alpha-galactosidase A). It affects both males and females and can present a number of signs or symptoms of variable degree, such as cardiovascular and/or renal dysfunction, intense or burning pain, heat intolerance, skin lesions, gastrointestinal complaints, hearing loss, and ocular problems. Lifespan is typically reduced in patients with Fabry disease by approximately 20 years in men and 15 years in women, compared with the general population. The principal causes of death are renal failure, cardiomyopathy and cerebrovascular events (e.g. stroke). Fabry disease is rare, affecting an estimated 8,000 to 10,000 people worldwide.

REPLAGAL is available to U.S. patients under an FDA approved treatment protocol. Shire is also supporting emergency IND (Investigational New Drug) requests (an application to use an unapproved product for a single patient). Shire worked closely with the FDA to establish an early access program in response to the ongoing shortage of the currently marketed treatment for Fabry disease in the U.S.

Shire Human Genetic Therapies president, Sylvie Gregoire, said, “We continue to deliver on our commitment to the Fabry community by filing a BLA to support long-term access to REPLAGAL in the United States. 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.”

Shire expects its REPLAGAL supply to be adequate to meet anticipated global demand.

About QualityStocks

QualityStocks, based in Scottsdale, Arizona is a free service that collects data from hundreds Small-Cap and Micro-Cap online Investment Newsletters into one Free Daily Newsletter Report. QualityStocks is dedicated to assisting emerging public companies with their advertising efforts and now has over 750,000+ subscribers that receive The Daily Stock Newsletters.

To sign up for “The QualityStocks Daily Newsletter” please visit www.QualityStocks.net

Please see disclaimer on QualityStocks website: http://Disclaimer.QualityStocks.net 

Forward-Looking Statement:

This release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All forward-looking statements are inherently uncertain as they are based on current expectations and assumptions concerning future events or future performance of the company. Readers are cautioned not to place undue reliance on these forward-looking statements, which are only predictions and speak only as of the date hereof. Risks and uncertainties applicable to the company and its business could cause the company's actual results to differ materially from those indicated in any forward-looking statements.