Immutep Earns FDA Orphan Drug Status for Efti After 51.5% Tumor Hyalinization
Immutep’s eftilagimod alfa received FDA Orphan Drug Designation for soft tissue sarcoma, offering potential seven years of market exclusivity and regulatory incentives. The Phase II EFTISARC-NEO trial achieved median tumor hyalinization of 51.5% in 38 patients, surpassing the 35% endpoint and historical 15% radiotherapy benchmark.
1. FDA Designation
Immutep’s eftilagimod alfa has received FDA Orphan Drug Designation for soft tissue sarcoma, qualifying for incentives such as seven years of market exclusivity, tax credits, and fee waivers designed to support development of therapies for diseases affecting fewer than 200,000 US patients.
2. Phase II Trial Results
The investigator-initiated Phase II EFTISARC-NEO trial evaluated efti with radiotherapy and pembrolizumab in 38 patients with resectable soft tissue sarcoma. The study surpassed its primary endpoint with a median tumor hyalinization/fibrosis of 51.5%, exceeding the 35% target and the ~15% benchmark seen with radiotherapy alone, while maintaining a favorable safety profile.
3. Next Steps
Following discontinuation of the Phase III TACTI-004 trial, Immutep is conducting a comprehensive review of clinical data to determine the optimal pathway for late-stage development of efti in the neoadjuvant setting for soft tissue sarcoma.