TREATMENT OF UREA CYCLE DISORDERS

THE LONG-TERM GOAL OF THIS PHASE I PROJECT IS TO DETERMINE THE FEASIBILITY OF ASSEMBLING SUCCESSFUL NEW DRUG APPLICATIONS FOR TWO INVESTIGATION NEW DRUGS CURRENTLY UNDERGOING PHASE III FDA APPROVED STUDIES. THE DRUGS UNDER STUDY INCLUDE THE ORAL DISAGE FORM OF SODIUM PHENYLBUTYRATE AND THE INTREVENOUS DOSAGE FORM OF SODIUM BENZOATE AND PHENYLACETATE. BEACUSE THEIR INDICATED USE IS FOR A GROUP OF RARE UREA CYCLE DISORDERS (DEFICIENCIES OF CARBAMYL PHGOSPHATE SYNTHETASE, ORNITHINE TRANSCARBAMYLASE, AND ARGININOSUCCINICACID SYNTHETASE) WHICH HAVE AN ESTIMATED COMBINED INCIDENCE OF 1/25,000, IT HAS NOT BEEN POSSIBLE TO OBTAIN PHARMACEUTICAL INDUSTRY INTEREST IN THEM. THE SPECIFIC AIMS OF THIS PHASE I SBIR APPLICATION ARE TO DETERMINE IF THE CLINICAL AND LABORATORY DATA BSE CURRENTLY AVAILABLE IN FDA APPROVED PHASE III STUDIES ARE SUFFICIENT TO MEET THE FDA'S NEW DRUG APPLICAITON REQUIREMENTS AS WELL AS TO DETERMINE IF CONTRACT MANUFACTURING ARRANGEMENTS CAN BE MADE THAT MEET FDA'S GOOD MANUFACTURING PRACTICE REQUIREMENTS. IF THIS PHASE I SBIR APPLICAITON DEMONSTRATES SUCH FEASIBILITY, A PHASE II SBIR APPLICAITON WILL BE FILED, THE PRUPOSE OF WHICH WILL BE TO SUBMIT NEW DRUG APPLICAITONS TO THE FDA IN THE EXPECTATION OF MARKETING THESE DRUGS FOR THESE RARE DISEASES.