FDA Grants Orphan Drug Designation to Tigilanol Tiglate for Soft Tissue Sarcoma


Tigilanol tiglate is a novel, small molecule drug for the intratumoral treatment of solid tumors, inducing tumor cell death and stimulating immune response.

Tigilanol tiglate (QBiotics Group), an intratumoral oncology asset, was granted Orphan Drug Designation by the FDA for the treatment of soft tissue sarcoma. According to a press release, Orphan Drug Designation is considered for drugs that show promise for treatment of rare diseases, defined as less than 200,000 cases per year in the United States.1

Cancer cells, 3D illustration | Image Credit: Dr_Microbe - stock.adobe.com

Dr_Microbe - stock.adobe.com

According to the American Cancer Society, the organization predicts approximately 13,590 new soft tissue sarcoma diagnoses in 2024, with approximately 5200 individuals dying from soft tissue sarcomas. The most common types include undifferentiated pleomorphic sarcoma, liposarcoma, and leiomyosarcoma, according to the organization.2

“Soft tissue sarcomas constitute a rare group of tumors comprising more than 80 subtypes that affect both adults and children. The prognosis of advanced soft tissue sarcoma patients remains unfavorable and new treatments are urgently needed,” Victoria Gordon, PhD, executive director of strategic alliances & investor relations at QBiotics, said in the press release. “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.”1

About The Trial

Trial Name: A Clinical Study to Investigate the Efficacy of Intratumoral Tigilanol Tiglate in Soft Tissue Sarcoma

ClinicalTrials.gov ID: NCT05755113

Sponsor: QBiotics Group Limited

Completion Date (Estimated): March 2025

Tigilanol tiglate is a novel small molecule drug for the intratumoral treatment of solid tumors. The drug induces tumor cell death and stimulates immune response, according to the press release.1

Currently, a phase II clinical trial, QB46C-H07, is being conducted at Memorial Sloan Kettering Cancer Center led by principal investigator Edmund Bartlett, MD. The trial will assess the preliminary efficacy and safety of the drug in those with a range of advanced and/or metastatic soft tissue sarcoma, according to the press release.1

The primary endpoint includes the ablation rate, defined as the proportion of individual achieving more than a 30% reduction in tumor value compared to baseline. Investigators will evaluated it via ultrasound. The secondary endpoint includes incidence of adverse events (AEs) and serious AEs as well as pharmacokinetics, according to the press release.1

Furthermore, the exploratory endpoints will include the local rate of recurrence at injection site at 6 months post-first injection and assessment of tumor response. Investigators plan biopsies at baseline and 14 days after injection. Also, investigators will use surgical specimens and blood samples to determine the changes in tumor biomarkers, according to the press release.1

In June 2023, the first patient was treated in the QB46C-H07 trial to investigate the drug. It was opened for recruitment until late April 2023, according to a press release.3 The trial is a single center, open-label, single arm study, including 10 individuals.

According to the press release, tigilanol tiglate (Stelfonta) is currently approved for veterinary usage by the European Medicines Agency, Swissmedic, the FDA’s Center for Veterinary Medicine, and more to treat canine mast cell tumors.3 In a veterinary study, the drug induced a complete response in 75% of mast cell tumors with a single injection and an 88% response with 2 injections. Furthermore, there was no tumor recurrence in 89% of cases 12 months after treatment.3


  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. https://qbiotics.com/investors/announcements/26-news/377-qbiotics-drug-tigilanol-tiglate-awarded-orphan-drug-designation-for-patients-with-soft-tissue-sarcoma
  2. American Cancer Society. Key Statistics for Soft Tissue Sarcomas. Updated January 12, 2023. Accessed February 16, 2024. https://www.cancer.org/cancer/types/soft-tissue-sarcoma/about/key-statistics.html
  3. First patient treated in QBiotics’ phase II clinical trial in patients with soft tissue sarcoma. News release. QBiotics Group. June 9, 2023. Accessed February 16, 2024. https://qbiotics.com/investors/announcements/36-news/human-oncology/367-first-patient-treated-in-qbiotics-phase-ii-clinical-trial-in-patients-with-soft-tissue-sarcoma
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