With a potential shortfall in the only marketed treatment, the agency is looking for alternatives in development.
Shire filed a treatment protocol, upon FDA’s request, for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Gaucher disease. The agency is looking for alternatives to the currently marketed treatment, which may have restricted availability in the coming months.
Genzyme offers Cerezyme for Gaucher’s but recently reported viral contamination at its plant. The firm was forced to shut down the facility temporarily, and hence Cerezyme’s supply will take a hit.
Yesterday FDA asked Protalix to submit a treatment protocol for its candidate as well. Both Shire’s and Protalix’ treatments are in Phase III development. Shire’s is made using human cell lines, while Protalix’ is a plant-produced therapy.
If either drug is approved by the FDA, the treatment protocol would allow physicians to treat Gaucher disease patients with the therapy ahead of commercial availability in the U.S. Under the conditions of the treatment protocol, the medications would be supplied free of charge initially to provide access to patients as quickly as possible.