FDA Grants ODD to Utidelone Capsules For Treatment of Gastric Cancer


The orphan drug designation (ODD) is a result of positive findings from a phase 2 clinical trial which demonstrated a 100% clinical benefit rate in patients.

X-ray of tumor in stomach -- Image credit: Sebastian Kaulitzki | stock.adobe.com

Image credit: Sebastian Kaulitzki | stock.adobe.com

About the Trial

Trial Name: Tolerability of Utidelone Capsule in Patients With Advanced Solid Tumors

ClinicalTrials.gov ID: NCT05681000

Sponsor: Biostar Pharma, Inc.

Completion Date (Estimated): December 31, 2024

The FDA has announced that utidelone capsules (UTD2, Biostar Pharmaceuticals) has been granted orphan drug designation (ODD) for the treatment of gastric cancer. The ODD comes after positive phase 2 trial data which evaluated the safety and efficacy of utidelone in patients with 2 different forms of gastric cancer.1

Utidelone is the only microtubule inhibitor that was developed using synthetic biology technology to be approved. Its mechanism of action is similar to taxanes while demonstrating multiple advantages, such as strong anti-tumor activity, broader anti-tumor spectrum, high oral bioavailability, stronger safety profile with a lower hematologic toxicity, and capability of crossing the blood-brain barrier for the treatment of brain tumors.1

Because of the failure of some oral taxanes—including Tesetaxel (Odonate Therapeutics) and paclitaxel plus encequidar (Oraxol; Kinex Pharmaceuticals, Hanmi Pharmaceuticals)—in clinical trials as a result of safety problems, there is a need for an approved oral microtubule inhibitor. The development of oral formulation of microtubule inhibitors presented to be a challenge due to susceptibility to P-glycoprotin-mediated efflux and low bioavailability, but UTD2 capsules have demonstrated advantages including improved administration convenience and patient compliance, reduced treatment cost, the acceleration of long-term therapy, and the promotion of combination therapy.1

The multicenter, phase 2 clinical trial, which assessed the safety and efficacy of utidelone capsules in patients with gastric cancer, evaluated participants across 2 stages. The first stage of the study enrolled 15 patients with advanced gastric cancer who received utidelone capsule monotherapy to evaluate the efficacy with an outcome of 3 partial response (PR) and 5 stable disease (SD), whereas the second stage examined 11 patients with metastatic and/or unresectable HER2 negative gastric cancer who previously had first-line treatment of utidelone with sintilimab and oxaliplatin, until a completed tumor assessment with an outcome of 8 PR and 3 SD.1

The results indicated that treatment with utidelone had resulted in a 100% clinical benefit rate in patients, and preliminary data showed a high bioavailability (57%) with a low effective dose, wide therapeutic window, and a positive safety profile.1

Previously, the open-label, dose escalation phase 1 study, BG02-2201 (NCT05681000), evaluated patients with advanced solid tumors aged 18 years and older for whom standard therapies are not available, no longer effective, or not tolerated. Patients were assigned to 1 of 6 potential cohorts that received oral doses of utidelone in a 21-day cycle starting as low as 25 mg that increased up to 120 mg. The study assessed the maximum tolerated dose and dose-limiting toxicity, as well as objective response rate, the pharmacokinetic profile of utidelona, and the recommended phase 2 dose.2,3

“We are confident that this breakthrough product will provide more benefits for numerous cancer patients and bring about a substantial change to microtubule inhibitors administration. [We are] fully committed to advancing this study in order to meet substantial clinical needs around the world,” said Li Tang, PhD, chairman of Biostar Pharma, in a press release.2

1. PR Newswire. 100% CBR! FDA Granted ODD to Biostar Pharma's Utidelone Capsule (UTD2) for the Treatment of Gastric Cancer. News release. March 21, 2024. Accessed March 21, 2024. https://prnmedia.prnewswire.com/news-releases/100-cbr-fda-granted-odd-to-biostar-pharmas-utidelone-capsule-utd2-for-the-treatment-of-gastric-cancer-302095896.html
2. BioSpace. Biostar Pharma Announces First Patient Enrolled for Phase 1 Study of Utidelone Capsule (UTD2)--The World's First Oral Epothilone Anti-Cancer Drug. News release. June 12, 2023. Accessed March 21, 2024. https://www.biospace.com/article/releases/biostar-pharma-announces-first-patient-enrolled-for-phase-1-study-of-utidelone-capsule-utd2-the-world-s-first-oral-epothilone-anti-cancer-drug/
3. Tolerability of Utidelone Capsule in Patients With Advanced Solid Tumors. ClinicalTrials.gov identifier: NCT05681000. Updated November 9, 2023. Accessed March 21, 2024. https://clinicaltrials.gov/study/NCT05681000
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