Mundipharma EDO GmbH: US FDA grants Orphan Drug Designation for tinostamustine in very rare blood cancer


Basel, Switzerland, 28 March 2019 – Mundipharma EDO GmbH, and Imbrium Therapeutics L.P., an operating subsidiary of Purdue Pharma L.P., today announced that the US FDA has granted Orphan Drug Designation (ODD) to tinostamustine, an alkylating deacetylase inhibiting molecule for the treatment of T-cell prolymphocytic leukaemia (T-PLL).3

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 US. 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 five to 10 years.4 Due to its rarity, T-PLL can be misdiagnosed, resulting in poor patient outcomes.4 Patients have a median survival of around seven months to one year, and the disease is typically resistant to conventional chemotherapy.4,5

Dr Thomas Mehrling, CEO of Mundipharma EDO added: “Currently there are no licensed treatment options for T-PLL, therefore, the development of new therapeutic approaches is essential for these patients.  We are pleased that the FDA has granted tinostamustine orphan drug designation in this area. At Mundipharma EDO, our focus is to develop treatments for rare and difficult-to-treat cancers, such as T-PLL, and we are progressing the development of tinostamustine in early phase clinical trials, in conjunction with Imbrium Therapeutics.”

To find out more about tinostamustine and the Mundipharma EDO oncology clinical trials programme visit:



Notes to editors:

About T-cell prolymphocytic leukaemia

T-cell prolymphocytic leukaemia (T-PLL) affects approximately 2% of all patients with mature lymphocytic leukaemias.1 It 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 T-PLL affects older adults with a median age at diagnosis of 61 years, and it is more common in men than in women.2 T-PLL typically has rapid progression and does not respond well to standard multi-agent chemotherapy and relapses are common.4

About tinostamustine

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 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.6-9 The preclinical data also suggest that these complementary and simultaneous modes of action have the potential to overcome resistance towards some other cancer treatments.6-9

Tinostamustine is currently being studied in multiple myeloma (MM), Hodgkin lymphoma (HL), peripheral T-cell lymphoma (PTCL), cutaneous T-cell lymphoma (CTCL), T-cell prolymphocytic leukaemia (T-PLL), soft tissue sarcoma (STS), small cell lung cancer (SCLC), triple-negative breast cancer (TNBC), ovarian cancer, endometrial cancer and MGMT-unmethylated glioblastoma.

About Mundipharma EDO

Mundipharma EDO is part of the Mundipharma global network of privately-owned independent associated companies, which operate in over 120 countries worldwide. We develop treatments for patients around the world with rare or R/R cancer, investigating smart approaches to new cancer treatments from concept through to clinical development and regulatory approval.

We operate a lean, agile research and development model, empowering the team to form conclusions and make quick decisions with the aim of getting new therapies to patients as rapidly as possible.

For more information please visit:

About Imbrium Therapeutics 

Imbrium is a clinical stage biopharmaceutical company dedicated to advancing medical science through the development of important new pharmacologic and biologic therapeutics. Imbrium is pursuing treatments for oncology chemotherapeutics, disorders of the central nervous system, and non-opioid approaches to the management of pain. As an operating subsidiary of Purdue Pharma L.P., Imbrium strives to develop and bring to market new medicines that serve the unmet needs of patients, physicians and health systems worldwide. The company has built a robust and diversified pipeline of investigational drug candidates, and actively collaborate with industry and academic partners to identify and advance future impactful medicines.

For more information, please visit:

About the Mundipharma network
Mundipharma is a global network of privately-owned independent associated companies whose purpose is to move medicine forward.

With a high performing and learning organization that strives for innovation and commercial excellence through partnerships, we successfully transformed and diversified our European portfolio of medicines to create value for patients, payers and wider healthcare systems across important therapeutic areas such as Diabetes, Respiratory, Oncology, Pain and Biosimilars.

For more information please visit:


For further information please contact:                                        


Tiffany Fretwell

Communications Lead, Mundipharma

[email protected]

+44 (0) 1223 393 361


Helen Rae

Makara Health

[email protected]

Tel: +44 (0) 23 81 247 327



[email protected]




  1. WHO Classification of Tumours of Haematopoietic and Lymphoid Tissues (Revised 4th edition) IARC: Lyon 2017 (pg 272)
  2. T-cell prolymphocytic leukemia (T-PLL) information page. Leukemia and Lymphoma Society. Available at: Last accessed 26 March 2019.
  3. US Food and Drug Administration. Last accessed 26 March 2019.
  4. Dearden C. How I treat prolymphocytic leukemia. Blood. 2012; 120(3):538–55.
  5. NIH NCI SEER Database. Available at: Last accessed 26 March 2019.
  6. López-Iglesias AA. The Alkylating Histone Deacetylase Inhibitor Fusion Molecule Edo-S101 Displays Full Bi-Functional Properties in Preclinical Models of Hematological Malignancies. Blood2014; 124: (21).
  7. López-Iglesias AA. preclinical antimyeloma activity of EDO-S101 (bendamustine-vorinostat fusion molecule) through DNA-damaging and HDACi effects. 15th International Myeloma Workshop. 23−26 September 2015. Rome, Italy.
  8. Di Filippi R, et al. The First-In-Class Alkylating Histone Deacetylase Inhibitor (HDACi) Fusion Molecule EDO-S101 Exerts Potent Preclinical Activity Against Tumor Cells of Hodgkin Lymphoma (HL) Including Bendamustine Resistant Clones. 57th Annual Meeting and Exposition of the American Society of Hematology (ASH), 6 December 2015.
  9. Yan S, Xu K, Lin J, et al. Synergistic inhibition of tumor growth and overcoming chemo-resistance by simultaneously targeting key components in DNA damage/repair, epigenetic, and putative cancer stem cell signaling pathways using novel dual-functional DNA-alkylating/HDAC inhibitor and tumor suppressor gene nanoparticles in lung cancer. Cancer Research 2012;72(Suppl 1): Abstract 2741.