Mundipharma EDO GmbH: US FDA Grants Orphan Drug Designation for Tinostamustine in Very Rare Blood Cancer

FOR EX-US MEDICAL AND TRADE MEDIA ONLY

- T-cell prolymphocytic leukaemia is an extremely rare and
aggressive T-cell leukaemia, with very limited effective treatment
options1,2

- Tinostamustine is in very early phase clinical trials to
investigate it as a potential future treatment option in this area of
significant unmet patient need

Basel, Switzerland (ots/PRNewswire) - 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 and view the full press release
visit: www.edoncology.com

References:

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:
https://www.lls.org/leukemia/t-cell-prolymphocytic-leukemia-t-pll.
Last accessed 26 March 2019.
3. US Food and Drug Administration. https://www.accessdata.fda.gov/sc
ripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=636418. Last
accessed 26 March 2019.
4. Dearden C. Blood. 2012; 120(3):538-55.
5. NIH NCI SEER Database. Available at: https://seer.cancer.gov/seert
ools/hemelymph/51f6cf58e3e27c3994bd53f3/. Last accessed 26 March
2019.

ots Originaltext: Mundipharma EDO GmbH
Im Internet recherchierbar: http://www.presseportal.de

Contact:
+44 (0) 23 81 247 327
USA
media@imbriumthera.com

Original-Content von: Mundipharma EDO GmbH, übermittelt durch news aktuell