Post by Brenda Patterson

Securing FDA approval for both pediatric and adult populations in a single rare disease trial isn't common. When both children and adults are included, the regulatory complexity multiplies. Separate dosing strategies, enhanced safety monitoring, informed consent protocols, and heightened scrutiny at every approval stage. Companies like Apellis Pharmaceuticals and leaders Cedric Francois and Jeffrey Eisele recently achieved this with EMPAVELI, securing approval for both pediatric and adult patients with C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis in 2025. It's a huge accomplishment, the first-ever FDA-approved treatment for these ultra-rare kidney diseases. 👏 The regulatory teams that succeed in pediatric rare disease development don't just follow the standard playbook—they design protocols that generate robust data across age groups while working within extremely limited patient populations. When fewer than 3,000 people in the U.S. have a disease, there's no margin for error. Rare Disease Day is February 28th - I'm certain Apellis and the entire team will be celebrating! As Founder of JBK Search - Rare Disease Day is very important to us! Thank you Apellis and all the other people committed to working on rare diseases! #RegulatoryAffairs #RareDisease #Biopharma #Nephrology #JBKSearch #NoRest