Notice of Intent to Award A Grant with Time Constraints

摘要

The Food and Drug Administration (FDA) is announcing its intention to accept and consider an application for the award of a grant with time constraints to the American Drug Development, Inc. (ADD). The purpose of the grant is to provide the orphan drug, Sodium Phenylbutyrate, to children with three urea cycle disorders (deficiencies of carbamyl phosphate synthetase, ornithine transcarbamylase (OTC), and argininosuccinic acid synthetase) which, if not treated, are fatal to the patient. Competition is limited to ADD because ADD is the entity currently able to manufacture and supply Sodium Phenylbutyrate for the treatment of these disorders. ADD is also in the process of compiling documentation which will be submitted to FDA for approval of a new drug application (NDA) for Sodium Phenylbutyrate. Until the NDA is approved, this drug is not available to patients by any other means other than through a grant to ADD.