ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY TREATMENT MARKET ANALYSIS

Market News

Global Ornithine Transcarbamylase (OTC) Deficiency Treatment Market: Key Developments

Increasing number of initiatives for research and development activities for Ornithine Transcarbamylase (OTC) deficiency treatment market by market players is expected to propel the growth of the Global Ornithine Transcarbamylase (OTC) deficiency treatment market over the forecast period. For instance, in April 2021, Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, announced the successful completion of End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), for the DTX301 Ornithine Transcarbamylase (OTC) deficiency gene therapy program. The meeting focused on the discussion of the Phase 1/2 data and alignment on Phase 3 design and endpoints. Based on the outcome of this meeting, Ultragenyx has finalized the Phase 3 study design, which will include a 64-week primary efficacy analysis period and enroll approximately 50 patients 12 years of age and older, randomized 1:1 to DTX301 (1.7 x 10^13 GC/kg dose) or placebo.