FDA Approves Multi-Dose Thiotepa Vial for Breast and Ovarian Cancers

The FDA originally approved this thiotepa formulation as a therapy for patients with breast or ovarian cancers in June 2024.

The FDA has approved thiotepa (Tepylute; formerly SH-105) as a multi-dose vial at 100 mg/10 mL for patients with breast cancer or ovarian cancer, according to a press release from the developer, Shorla Oncology.¹

The regulatory decision for this ready-to-dilute formulation of thiotepa may decrease preparation time and errors while offering higher scheduling flexibility for patients.

“We are pleased to offer another viable treatment option for patients with breast and ovarian cancer,” Sharon Cunningham, chief executive officer and co-founder of Shorla Oncology, stated in the press release.¹ “Once opened, our 100 mg vial of [thiotepa] is stable for 14 days when properly stored, giving providers the flexibility they need when preparing and administering this very important treatment.”

The FDA originally approved this thiotepa formulation as a therapy for patients with breast or ovarian cancers in June 2024.²

“This is a huge win for providers because [thiotepa] avoids the need for complicated and time-consuming reconstitution,” Orlaith Ryan, chief technical officer and co-founder of Shorla Oncology, stated in the press release.¹

According to the agent’s prescribing information, the recommended dose of thiotepa for treating patients with adenocarcinoma of the breast or ovary is 0.3 mg/kg to 0.4 mg/kg intravenously.³ The labeling includes a warning for severe marrow suppression or ablation with consequent infection or bleeding as well as potential carcinogenicity in patients.

The most common adverse effects associated with the thiotepa formulation include neutropenia, anemia, thrombocytopenia, alanine aminotransferase elevation, aspartate aminotransferase elevation, bilirubin elevation, mucositis, and cytomegalovirus infection. The labeling advises against the agent’s administration in combination with live or attenuated viral or bacterial vaccines until any immunosuppressive effects subside. Additionally, providers should monitor for hepatic veno-occlusive disease via physical examination, serum transaminases, and bilirubin.

The labeling indicates that fatal encephalopathy has occurred in patients who have received high doses of thiotepa. Other dose-dependent central nervous system toxicities include headache, apathy, disorientation, confusion, amnesia, drowsiness, and seizures.

Thiotepa may cause fetal harm in patients who are pregnant. Those with reproductive potential are recommended to use highly effective contraception during treatment with thiotepa and for 6 months following therapy.

Administering thiotepa may cause skin discoloration, pruritus, blistering, desquamation, and peeling with higher severity in the groin, axillae, skin folds, and neck area. Patients who receive this agent are advised to shower or bathe with water at least twice each day for up to 48 hours following therapy.

According to the labeling, thiotepa contains a high concentration of polyethylene glycol (PEG) 400. Administering PEG 400 at higher doses than recommended may damage the kidneys and liver.

Regarding postmarketing experience with thiotepa, blood and lymphatic system disorders include febrile bone marrow aplasia. Additionally, cardiac disorders include bradycardia, cardiac failure congestive, cardiorespiratory arrest, pericardial effusion, pericarditis, and right ventricular hypertrophy. Other types of AEs identified in a postmarketing setting include congenital, familial, and genetic disorders; ear and labyrinth disorders; eye disorders; gastrointestinal disorders; general disorders and administration site conditions; hepatobiliary disorders; immune system disorders; and renal and urinary disorders.

Clinical studies for thiotepa in breast and ovarian cancer did not include enough patients who were 65 years or older to determine whether older patients respond differently compared with younger populations.

“We are excited to bring [thiotepa] to the US Market,” Rayna Herman, chief commercial officer of Shorla Oncology, said in the press release.¹ “It provides consistent dosing accuracy and allows for ‘just in time’ preparation, which benefits everyone, especially patients.”

References

1. Shorla Oncology announces FDA Approval of TEPYLUTE® 100mg, first and only ready-to-dilute multi-dose vial of thiotepa to treat breast and ovarian cancer and commercial launch of TEPYLUTE 15mg and 100mg vials in the U.S. News release. Shorla Oncology. April 29, 2025. Accessed April 30, 2025. https://tinyurl.com/ywmdhkp9
2. Shorla Oncology announces FDA approval for TEPYLUTE, a novel formulation to treat breast and ovarian cancer. News release. Shorla Oncology. June 28, 2024. Accessed April 30, 2025. https://shorturl.at/EBFEl
3. TEPYLUTE (thiotepa) injection, for intravenous use. Prescribing information. FDA. June 2024. Accessed April 30, 2025. https://tinyurl.com/23u6wnk8