Servier’s Emi-Le Receives FDA Breakthrough Therapy Designation for Adenoid Cystic Carcinoma

Servier has announced that its investigational antibody-drug conjugate (ADC) therapy, emiltatug ledadotin (Emi-Le), has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of adenoid cystic carcinoma (ACC). The designation applies to patients with locally advanced, recurrent, or metastatic ACC with solid histology or high-grade transformation, both associated with more aggressive forms of the disease.

Adenoid cystic carcinoma (ACC) is a rare and aggressive cancer that develops in the secretory glands, most commonly in the head and neck region, but also in other parts of the body. Globally, more than 200,000 people are estimated to be living with ACC, with approximately four cases diagnosed per million people each year. No clear association with ethnicity or lifestyle factors has been established. Currently, there are no approved or preferred systemic therapies for advanced or metastatic ACC. Although many patients undergo surgery or radiation treatment, the disease recurs in nearly half of cases and often progresses to metastatic disease.

Emi-Le is currently being evaluated in a multicentre Phase 1 clinical trial (NCT05377996) assessing its safety, tolerability, and anti-tumour activity across several solid tumours, including ACC, breast, endometrial, and ovarian cancers. Initial Phase 1 findings showed manageable side effects, with confirmed objective responses observed across multiple tumour types.

Emi-Le is an investigational dolasynthen ADC targeting B7-H4, a protein associated with several cancers, including ACC. The therapy is designed with a target-optimised drug-to-antibody ratio (DAR 6) and incorporates a proprietary auristatin-F HPA payload intended to deliver tumour-killing activity with controlled bystander effect.

In addition to the newly granted Breakthrough Therapy Designation for ACC, Emi-Le has also received Fast Track Designations from the FDA for adult patients with advanced or metastatic triple-negative breast cancer and advanced or metastatic HER2-low/HER2-negative breast cancer following topo-1 ADC therapy, including TNBC and certain HR-positive breast cancers.

According to the company, the FDA’s Breakthrough Therapy Designation is intended to accelerate the development and review of investigational therapies that may demonstrate substantial improvement over existing treatments for serious diseases. Peter Adamson, Global Head of Oncology Clinical Development at Servier, stated that the designation reflects the company’s commitment to advancing first-in-class medicines for rare oncology indications and may help bring a new treatment option to patients with limited therapeutic choices.

The company also noted that the designation reinforces confidence in the oncology portfolio added through the acquisition of Day One Biopharmaceuticals and supports its broader strategy of developing targeted therapies and precision medicines for difficult-to-treat cancers.

Servier further highlighted that it reinvests nearly 20% of revenue generated from brand-name medicines into research and development activities annually. The company follows a One Innovation Engine strategy and continues to pursue collaborations, partnerships, and acquisitions across multiple stages of oncology drug development.

References

Emiltatug Ledadotin (Emi-Le) Granted Breakthrough Therapy Designation by the U.S. FDA for Adenoid Cystic Carcinoma (ACC) – May 12, 2026