Core Insights - Medicus Pharma is expanding compassionate access to its lead treatment Skinject for non-melanoma skin diseases through a partnership with the Gorlin Syndrome Alliance, targeting unmet needs for patients with Gorlin syndrome [1][5][6] Company Initiatives - The company is pursuing FDA compassionate use approval for Skinject, which is a non-invasive treatment specifically for basal cell carcinoma, a common issue for Gorlin syndrome patients [2][6] - The partnership with the Gorlin Syndrome Alliance aims to provide treatment options for approximately 11,000 individuals living with Gorlin syndrome, who face a lifelong burden of basal cell carcinoma [5][6] Regulatory Environment - The FDA is currently open to advancing treatments for rare diseases, which aligns with Medicus Pharma's goals and regulatory progress [3][7] - The company is engaging key supporters in Washington, including board member Cathy McMorris Rodgers, to facilitate the application process for compassionate use [3][8] Patient Advocacy - The collaboration with the Gorlin Syndrome Alliance is seen as a strategic move to enhance patient advocacy and support for those affected by Gorlin syndrome, which currently lacks available treatments [7][10] - The alliance will help coordinate with patients and maintain a registry, ensuring that safety data is collected during the compassionate use of Skinject [9][10]
Medicus Pharma eyes compassionate use approval of Skinject for Gorlin Syndrome - ICYMI