Mundipharma has announced that the European Commission (EC) has now adopted the European Medicines Agency (EMA) Committee for Orphan Medicinal Products recommendation to grant Orphan Drug Designation to tinostamustine, an alkylating histone-deacetylase inhibiting molecule, for the treatment of T-cell prolymphocytic leukaemia (T-PLL).3
The EMA considers Orphan Drug Designation (ODD) status for medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than five per 10,000 people in the EU.4
T-PLL is an extremely rare and typically aggressive leukaemia.5,6 It affects approximately 2% of all patients with mature lymphocytic leukaemias1 and affects older adults with a median age at diagnosis of 61 years. It is more common in men than in women.2 T-PLL is characterised by the out-of-control growth of mature T-cells (T-lymphocytes). T-cells are a type of white blood cell that protects the body from infections.2 Due to its rarity, T-PLL can be misdiagnosed, resulting in poor patient outcomes.5 The condition is life-limiting and chronically debilitating, with fewer than 5% of patients surviving to five years from diagnosis.7 T-PLL generally progresses rapidly and does not respond well to standard multi-agent chemotherapy, with relapses being a common occurrence.5,6
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.
The EC decision adopting the EMA opinion follows that of the FDA, which granted tinostamustine ODD status for the treatment of T-PLL in March 2019.
Tinostamustine, is an alkylating histone-deacetylase inhibiting molecule in early phase clinical development for a range of rare and difficult-to-treat blood cancers and advanced solid tumours.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.8-11 The preclinical data also suggest that these complementary and simultaneous modes of action have the potential to overcome resistance towards some other cancer treatments.8-11