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FDA Actions & Updates

The latest approvals, designations, and new indications from the U.S. Food and Drug Administration for oncology drugs.

Friday, March 29, 2019

Tinostamustine Granted Orphan Drug Designation for T-Cell Prolymphocytic Leukemia

The FDA has granted Orphan Drug Designation (ODD) to tinostamustine, an alkylating deacetylase inhibiting molecule for the treatment of T-cell prolymphocytic leukemia (T-PLL).

The FDA grants ODD status to medicines intended for the treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. T-PLL is an extremely rare and typically aggressive blood cancer.  It is so rare that healthcare professionals may only see one case of T-PLL every 5-10 years. Due to its rarity, T-PLL can be misdiagnosed, resulting in poor patient outcomes. Patients have a median survival of around 7 months to 1 year, and the disease is typically resistant to conventional chemotherapy.

T-cell prolymphocytic leukemia (T-PLL) affects approximately 2 percent of all patients with mature lymphocytic leukemias. It is characterized by the out of control growth of mature T-cells (T-lymphocytes). T-PLL affects older adults with a median age at diagnosis of 61 years, and it is more common in men than in women. T-PLL typically has rapid progression and does not respond well to standard multi-agent chemotherapy and relapses are common.

Tinostamustine (EDO-S101), is an alkylating deacetylase inhibiting molecule in early phase clinical development for a range of rare and difficult-to-treat blood cancers and advanced solid tumors.

Preclinical studies have shown that tinostamustine has the potential to improve access to the DNA strands within cancer cells, break them, and counteract damage repair. The preclinical data also suggest that these complementary and simultaneous modes of action have the potential to overcome resistance towards some other cancer treatments.

Tinostamustine is currently being studied in multiple myeloma, Hodgkin lymphoma, peripheral T-cell lymphoma, cutaneous T-cell lymphoma, T-PLL, soft tissue sarcoma, small cell lung cancer, triple-negative breast cancer, ovarian cancer, endometrial cancer, and MGMT-unmethylated glioblastoma.