(RTTNews) - Biopharmaceutical company Shire plc (SHPGY:
News , SHP.L) said Wednesday that the New Drug Application of its Gaucher drug, velaglucerase alfa , was granted priority review status by the United States Food and Drug Administration. The FDA has issued action date of February 28, 2010 under the Prescription Drug User Free Act, or PDUFA.
Priority Review designation is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists, and accelerates the target review timing from ten to six months.
The FDA has also approved treatment protocol, under which Gaucher patients can be administered velaglucerase alfa prior to commercialization.
Shire noted that it is on track with its filing of the Marketing Authorization Application in the EU for 2009.
Gaucher disease, with an incidence of about 1 in 20,000 live births, is a lysosomal storage disorder resulting from a deficiency of glucocerebrosidase enzyme in the body, caused by a genetic mutation. The enzymes break down specific fat molecules, lack of which results in the accumulation of fat in liver, spleen and bone marrow.
Velaglucerase alfa is Shire's enzyme replacement therapy in the human cell line. The enzyme produced has the exact human amino acid sequence and has a human glycosylation pattern. Shire's velaglucerase alfa program enrolled over 100 patients at 24 sites in 10 countries around the world.
The only enzyme replacement therapy currently approved for Gaucher disease is Genzyme's Cerezyme, an intravenous, freeze-dried medicine containing imiglucerase. In order to clean up viral contamination in one of its plants, Genzyme has temporarily stopped production and shipments of Cerezyme. The supply shortfall is expected to last through the end of this year.
Genzyme is also in the process of evolving GENZ-112638 an investigational oral Gaucher treatment, which has met primary endpoints in Phase 2 clinical trials.
Protalix BioTherapeutics Inc's. (PLX:
News ) proprietary plant cell expressed recombinant form of GCD, prGCD, is under development, for the treatment of Gaucher disease. Earlier, prGCD was assigned orphan drug status, fast track designation and also approved for treatment protocol, by the FDA. Protalix expects to file an NDA for prGCD with the FDA by the end of this year.
SHPGY is currently trading up 0.32% at $52.95, on the Nasdaq.
 | | To receive FREE breaking news email alerts for Shire Pharmaceuticals Group PLC and others in your portfolio |
|
by RTT Staff Writer
For comments and feedback: contact editorial@rttnews.com