Medicus Pharma CEO meets Energy & Commerce leaders to advance SkinJect® pathway
Medicus Pharma CEO Raza Bokhari met four senior Energy & Commerce Committee members and rare disease caucus chairs to push for Orphan Drug Designation, IND approval and pediatric voucher access for SkinJect® in Gorlin Syndrome patients. The company plans a registrational pathway to produce decision-grade data for localized non-surgical therapy.
1. Capitol Hill Engagements
Medicus Pharma CEO Raza Bokhari and senior leadership held meetings on Capitol Hill with key lawmakers including Chair Brett Guthrie, Member John Joyce, Vice Chair Diana Harshbarger and Co-Chair Gus Bilirakis to discuss healthcare legislation impacting rare disease therapies.
2. Regulatory Support Goals
Discussions centered on securing Orphan Drug Designation for SkinJect® in Gorlin Syndrome, obtaining Investigational New Drug approval and leveraging the rare pediatric disease priority review voucher program to expedite development.
3. SkinJect Development Strategy
The company is advancing a registrational development pathway focused on generating decision-grade clinical data for SkinJect® as a localized, repeatable non-surgical treatment option to address the high recurrence burden in Gorlin Syndrome patients.