Developing New Therapeutic Entities to Address Unmet Needs in Orphan Diseases

Student: Madeline Zupan, 2016-2017

Sponsor: Horizon, Chicago, IL

Over 7,000 distinct orphan (rare) diseases exist, affecting one in every ten Americans, but 95% of these orphan diseases are devoid of an FDA-approved therapy or treatment. This research analyzes the biochemical potential, commercial viability, and projected clinical trial outcome of RAVICTI (glycerol phenylbutyrate), an existing approved Urea Cycle Disorder therapy, as a potential New Therapeutic Entity in four other promising orphan diseases. This research prioritizes indicators based on the domains of science, market, and industry synergy. Science data is gathered from primary literature in PubMed, market forecasts are based on industry standards and internal report, and industry synergy is ascertained from key opinion leaders in pharmacology. The study concludes in a consummate recommendation for advancement to clinical trials and commercialization on the basis of scientific feasibility, unmet orphan disease need, patient payer price considerations, and corporate strategy fit. This research holistically assesses the likelihood of successful novel RAVICTI implementation and develops a commercialization market plan for clinical orphan use in one, some, or all of the new indicator areas.