Friday, February 22, 2019

Mundipharma EDO GmbH: US FDA grants Orphan Drug Designation for etoposide toniribate in relapsed/refractory biliary tract cancer

BASEL, Switzerland -Friday 22 February 2019 [ AETOS Wire ]

    Biliary tract cancer or cholangiocarcinoma is rare, when patients fail first-line chemotherapy there are limited treatment options and in most cases the standard of care is palliative1,2
    Etoposide toniribate is to be accelerated through a Phase III trial, with the aim of providing a potential additional treatment option in this area of unmet patient need
    The decision by the US Food and Drug Administration (FDA) to grant Orphan Drug Status follows the precedent set by the European Medicines Agency (EMA) in 2014

(BUSINESS WIRE)-- Mundipharma EDO GmbH, part of the Mundipharma network of independent associated companies, 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 etoposide toniribate for the treatment of relapsed/refractory biliary tract cancer, also known as cholangiocarcinoma.3

This press release features multimedia. View the full release here:

Biliary tract cancer is a rare tumour with approximately 8,000 patients diagnosed in the US every year and 10,571 in Europe.4,5 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. Radical surgery is the only curative treatment for biliary tract cancer but, in most cases, the cancer is inoperable. Patients who fail first-line chemotherapy have limited treatment options and the standard of care is generally palliative.1,2 The five-year survival rates for patients with biliary tract cancer are very low, approximately 15%.6

Dr Thomas Mehrling, CEO of Mundipharma EDO added: “We are pleased that the FDA has recognised etoposide toniribate as a potential treatment for relapsed/refractory biliary tract cancer. As a company we are focused on developing treatments for rare and difficult-to-treat cancers and getting them to patients as rapidly as possible. We look forward to accelerating the development of etoposide toniribate, in conjunction with Imbrium Therapeutics, with a global Phase III trial with sites in EU, US, Australia and other countries.”

“We are pleased that the FDA has granted Orphan Drug Designation for etoposide toniribate in recognition of its potential as an important clinical advance for patients suffering from relapsed/refractory biliary tract cancer, a patient population that has limited treatment options,” said Paul Medeiros, President of Imbrium Therapeutics. “This designation represents Imbrium’s first milestone in oncology and underscores our commitment to advance the clinical development of oncology chemotherapeutics while actively collaborating to advance treatments across our therapeutic portfolio.”

Etoposide toniribate has shown encouraging data in Phase II trials and this data has been key in securing the ODD.7 This news is in addition to the European Medicines Agency (EMA) also granting orphan designation on 4 June 2014.8 As with the FDA, this designation allows protocol assistance, reduced fees and 10-years marketing exclusivity in that disease after approval. It can also lead to speedier reimbursement in many EU member states.

To find out more about this treatment or the Mundipharma EDO oncology clinical trials programme visit:

Notes to editors:

For further information please visit


1. Valle JW, et al. Ann Oncol. 2016;27(S5):v27–v37.
2. NCI. Accessed 14.02.19
3. US FDA. Accessed 14.02.19
4. ACS. Accessed 14.02.19
5. ECIS. Accessed 14.02.19
6. CCF. Accessed 14.02.19
7. Pape UF, et al. J Clin Oncol. 2019;3(S4):264.
8. EMA. Accessed 14.02.19

Date of Preparation: February 2019

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