FDA Grants Priority Review for Shire's velaglucerase alfa for Type 1 Gaucher Disease

The US Food and Drug Administration has granted priority review for Shire plc’s (Cambridge, MA) New Drug Application (NDA) for velaglucerase alfa, the company's enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease.

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 10 to six months. The FDA has issued an action date for the NDA of February 28, 2010, under the Prescription Drug User Fee Act (PDUFA).

In the US, patients continue to be enrolled in an FDA-approved treatment protocol, under which Gaucher patients receive velaglucerase alfa before commercialization. Shire also has engaged with national and regional authorities outside the US and patients are receiving velaglucerase alfa through pre-approval access programs. Shire confirms it is on track with its filing of the marketing authorization application in the EU for 2009.

Previous coverage:
Shire Completes NDA for Gaucher’s Disease TreatmentGaucher Patients Can Be Treated with Protalix's Drug During Cerezyme ShortageProtalix's Gaucher Drug May Fill Gap Left from Genzyme Plant ClosureGenzyme Detects Virus Contamination of Bioreactor, Halts Production