Immutep secures FDA Orphan Drug Designation for eftilagimod alfa in soft tissue sarcoma, supported by Phase II data showing improved tumour response.
Written By: Nikita Jha, BPharm
Reviewed By: Pharmacally Editorial Team
Immutep Limited has earned Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for eftilagimod alfa (efti), its investigational immunotherapy, in treating soft tissue sarcoma (STS) a rare, heterogeneous cancer group with few effective options.
This designation targets diseases affecting fewer than 200,000 people in the U.S., accelerating development for high unmet needs. Key benefits include tailored regulatory guidance, tax credits, waived FDA fees, and up to seven years of market exclusivity upon approval.
Clinical Evidence Backing the Designation
The FDA’s decision draws from the investigator-initiated Phase II EFTISARC-NEO trial (NCT06128863), which assessed efti plus radiotherapy and pembrolizumab in the neoadjuvant setting for resectable STS.
In 38 evaluable patients, the trial hit its primary endpoint with a median tumor hyalinization and fibrosis rate of 51.5% surpassing the 35% target and historical radiotherapy-alone rates of approximately 15%. Responses held steady across sarcoma subtypes, with translational data showing immune activation via efti’s mechanism: boosting antigen-presenting cells and T-cell responses. The regimen also demonstrated a strong safety profile, with no surgery delays.
Strategic Implications and Path Forward
This milestone comes as Immutep reviews its pipeline after halting the Phase III TACTI-004 trial, reshaping efti’s clinical strategy.
CEO Marc Voigt highlighted the ODD as validation of efti’s promise in hard-to-treat STS, with EFTISARC-NEO data potentially paving the way for a late-stage neoadjuvant study subject to ongoing evaluations.
Future Implications
STS’s rarity and diversity demand innovative approaches. Efti’s ODD underscores immunotherapy’s role in neoadjuvant combos, offering a promising signal amid stagnant progress, though larger trials are needed for confirmation.
Reference
Immutep | LAG-3 Immunotherapy for Cancer & Autoimmune Disease
About the Writer
Nikita Jha BPharm is a pharmacy graduate with expertise in clinical research, pharmacovigilance, and medical writing. In her words, she is passionate about translating complex scientific data into clear, accurate healthcare communications that advance drug safety and patient care.