Tigilanol Tiglate Wins FDA Orphan Drug Designation for Sarcoma


The FDA has granted an orphan drug designation to tigilanol tiglate for the treatment of soft tissue sarcoma.

Tumor Cell: ©nevio - stock.adobe.com

Tumor Cell: ©nevio - stock.adobe.com

  • Tigilanol tiglate (Stelfonta), an intratumoral drug, has received orphan drug designation from the FDA for the treatment of soft tissue sarcoma.
  • Orphan drug designation can be granted to agents that prevent, diagnose, or treat rare diseases.
  • The agent is being evaluated in an ongoing phase 2 trial (NCT05755113).

The FDA has awarded tigilanol tiglate orphan drug designation in soft tissue sarcoma, highlighting its potential as a treatment for the rare disease.1

"The prognosis of [patients with] advanced soft tissue sarcoma remains unfavorable and new treatments are urgently needed. The FDA orphan drug designation for tigilanol tiglate signals an important milestone for QBiotics, reflecting its recognition by the FDA as a potential new treatment option for this debilitating and life-threatening disease,” said Victoria Gordon, PhD, executive director of strategic alliances and investor relations at QBiotics, in a press release.

Orphan drug designation qualifies sponsors for certain incentives, including tax credits, user fee exemption, and a potential of 7 years of market exclusivity following approval.2

In 2020, the FDA approved tigilanol tiglate as a treatment for mast cell tumors in dogs.3

The agent is now being investigated in a phase 2 trial in patients with soft tissue sarcoma at Memorial Sloan Kettering Cancer Center, New York.4 The study’s primary end point is tumor response, and secondary end points are incidence of adverse events and systemic exposure. Investigators are also evaluating the tumor microenvironment, peripheral blood mononuclear cells, and local recurrence rate at injection site(s).

Patients in the single-arm, open-label study receive single or multiple injections of tigilanol tiglate at up to a fixed dose of 3.6 mg/m2.

Adult patients are eligible to participate in the trial if they have advanced and/or metastatic soft tissue sarcoma amenable to intratumoral injection, an ECOG performance status of 2 or lower, a life expectancy of more than 12 weeks, and adequate renal and hepatic function. Those of childbearing potential must agree to use a highly effective form of contraception.

Patients are ineligible to participate in the study if they have a tumor intended for injection that is adjacent to a major artery or vein or active brain metastases. Those who have received or are receiving other investigational agents or systemic cancer therapy must undergo an appropriate wash-out period.

1. QBiotics drug tigilanol tiglate awarded orphan drug designation for patients with soft tissue sarcoma. News release. QBiotics Group. February 16, 2024. Accessed February 16, 2024. http://tinyurl.com/mryt3vka
2. Designating an orphan product: drugs and biological products. U.S. Food & Drug Administration. Updated July 8, 2022. Accessed February 16, 2024. http://tinyurl.com/24muw8am
3. FDA approves first intratumoral injection to treat non-metastatic mast cell tumors in dogs. News release. FDA. November 16, 2020. Accessed February 16, 2024. http://tinyurl.com/4h9f87m4
4. A clinical study to investigate the efficacy of intratumoral tigilanol tiglate in soft tissue sarcoma. ClinicalTrials.gov. Updated May 31, 2023. Accessed February 16, 2024. https://clinicaltrials.gov/study/NCT05755113
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