Shortage Could Cause Early Access To Developmental Gaucher Disease Drugs
This article was originally published in Pharmaceutical Approvals Monthly
Shire and Protalix BioTherapeutics' experimental Gaucher disease drugs could enter the market early to help offset likely treatment shortages created by manufacturing problems with the only enzyme replacement therapy currently approved for the orphan condition: Genzyme's Cerezyme
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The FDA approval of Shire's Vpriv (velaglucerase) means that Genzyme's Cerezyme (imiglucerase) finally has competition in the Gaucher disease space. But market analysts are strongly divided on whether velaglucerase or a pending Pfizer/Protalix drug will be better positioned to make a dent in Cerezyme's market share
Shire releases positive results from the first of three Phase III studies of its Type 1 Gaucher diseasedrug Vela (velaglucerase alfa) in conjunction with its Aug. 3 announcement that FDA has accepted the treatment protocol for the drug. Shire also has begun its rolling submission of the NDA under the Fast Track process. FDA asked Shire and Protalix BioTherapeutics to file treatment INDs to expand access of their Gaucher disease candidates after manufacturing problems halted production of Genzyme's Cerezyme (imiglucerase for injection), the only enzyme replacement therapy currently approved for the orphan condition (1Pharmaceutical Approvals Monthly July 2009). FDA has also approved Protalix's treatment protocol (2"The Pink Sheet" DAILY, Aug. 17, 2009). FDA's decision to grant early access bodes well for approval of both products. Shire's Phase III study evaluated the safety and efficacy of Vela in 25 Gaucher disease patients, meeting the primary endpoint of clinically important and statistically significant increases in mean hemoglobin concentration compared with baseline. Patients were evaluated after receiving velaglucerase at 60 U/kg I.V. every other week for 12 months and nominally significant improvements were observed in liver size at this dose. Statistically significant improvement was also seen in platelet and spleen sizes for both 60 U/kg I.V. and 45 U/kg I.V. doses. Vela was generally well tolerated with no drug-related serious adverse events reported in the trial
Plagued by a virus, the biotech hopes to have Allston Landing facility back in operation by late July, but warns of short-term drug shortages.