US 505(b)(2) Regulatory Pathway and Strategies

Case Study: Ulesfia TM - Excipient• 5% benzyl alcohol lotion for treatment of head lice (Sciele Pharma, NDA22-129, April 9, 2009)• Widely-used chemical in cosmetic industry– Never approved as active ingredient so FDA classified it as NME• Development program:– Nonclinical• Literature for repeat dose and genetic toxicology• Literature for in vitro studies demonstrating mechanism of action• 2-year toxicology and carcinogenesis studies from the National Toxicology Program– Clinical Pharmacology/Biopharmaceutics• 2 PK studies (2nd was done because presence of API in the catheter wash wasconfounded results of the 1st study)– Efficacy– Safety• 2 Phase 3 trials, 615 subjects total (240 on treatment)• Relied on its database of 8 studies (2 Phase 3, 3 Phase 2, 2 Phase 1, 1 special safetystudy)• Extensive publication reviewReceived 5 years data exclusivity