Shire Gets Fast-Track Approval For Gaucher-Treating Drug
16 July 2009 - 5:25PM
Dow Jones News
Biopharmaceutical company Shire PLC (SHP.LN) said Thursday it
has received Fast Track designation from the U.S. Food and Drug
Administration for its drug velaglucerase alfa, an enzyme
replacement therapy that targets Type I Gaucher disease.
Fast track designation is used to speed through to market drugs
which are needed to treat a serious disease or unmet medical
need.
On July 6, the FDA asked that Shire file a treatment protocol
for velaglucerase alfa, which if accepted would allow physicians to
treat patients suffering from Gaucher - a genetic disease in which
a fatty substance accumulates in cells and certain organs - with
the drug before it was commercially available.
Shire said it would initially provide the drug free of charge to
speed up access for patients.
The total diagnosed population of Gaucher Disease patients is
around 7,000 worldwide, but it's thought that between 10,000 and
15,000 people have the disease.
The request came after production of Genzyme Corporation's
(GENZ) Cerezyme, the only other treatment for Gaucher I disease,
was halted due to manufacturing problems. Genzyme said production
would be resumed later this month.
At that time, Israel's Protalix BioTherapeutics Inc. (PLX) was
also approached by the FDA for a treatment protocol.
Velaglucerase alfa is made with Shire's proprietary technology,
the enzyme produced has the exact human amino acid sequence.
Shire focuses on attention deficit hyperactivity disorder, human
genetic therapies and gastrointestinal diseases.
Shire shares closed at 832 pence on Wednesday, having risen 12%
in the past year, giving the company a market capitalization of
GBP4.66 billion.
Company Web site: www.shire.com
-By Eric Jones, Dow Jones Newswires; 44-207-842-9295;
eric.jones@dowjones.com