Orphan Drug Designation granted to tinostamustine for rare form of leukaemia

Tinostamustine is in early phase clinical trials (Phase I) to investigate its use as a potential future treatment option in this area of significant unmet patient need.

Mundipharma has revealed  that the European Commission (EC) has adopted the European Medicines Agency (EMA) Committee for Orphan Medicinal Products recommendation to grant Orphan Drug Designation (ODD) to tinostamustine, an alkylating histone-deacetylase inhibiting molecule, for the treatment of T-cell prolymphocytic leukaemia (T-PLL).

The EMA considers ODD status for medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 5 per 10,000 people in the EU.

T-PLL is an extremely rare and typically aggressive leukaemia. Due to its rarity, T-PLL can be misdiagnosed, resulting in poor patient outcomes. The condition is life-limiting and chronically debilitating, with fewer than 5% of patients surviving to five years from diagnosis. T-PLL generally progresses rapidly and does not respond well to standard multi-agent chemotherapy, with relapses being a common occurrence.

Brian Sheehan, Chief Scientific Officer, Mundipharma Research added: “Orphan drug designation is an important milestone in the development of tinostamustine, which is currently in early phase clinical trials. We are proud of our commitment to helping patients with rare and difficult-to-treat cancers, such as T-PLL, where therapeutic options are so limited and patients have a clear need for new therapies.”

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