Cystinosis is a rare, genetic metabolic condition that affects every cell in the body. There are just 500 diagnosed cases in the US and 2,000 worldwide. With a cost of $36,000 per year, there were significant hurdles to overcome at the launch of PROCYSBI.

The highly researched strategy was to engage with advocacy and key patient influencers to announce the FDA approval of the orphan drug. An insight-driven patient campaign and messaging resonated with the small patient population. The branded website and comprehensive support materials for patients and their extended-care teams were deployed. Key word, interest targeting, and ad messaging strategies for Search Engine Marketing and Facebook campaigns were key to launch success.