This was a long-term, open-label study of the safety, tolerability and effectiveness of RP103
in cystinosis patients who were naïve to any form of cysteamine treatment. Participants
received RP103 treatment for at least 12 months. U.S. participants transitioned to the
commercially approved drug PROCYSBI®. In Brazil, after at least 12 months of study
participation and upon approval by the Brazilian regulatory authorities, participants were
eligible to transition to a post-study drug supply program, and continue to receive the drug
at no personal cost.