Shots:

• The FDA has approved Nulibry based on three clinical trials compared to data from a natural history study in patients with MoCD Type A
• The results from three clinical trials showed improvement in overall survival and an 82% reduction in the risk of death- @3yrs. probability of survival in rcPMP-treated patients (84% vs 55%)- reduction of urine concentrations of SSC and reduction was sustained with long-term treatment over 48mos.- the high infant mortality rate
• Nulibry is a first-in-class cPMP substrate replacement therapy and reviewed under PR and has received FDA’s ODD- BTD and RPDD. With this approval- the FDA also issued an RPD PRV to Origin

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