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

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

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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: www.edoncology.com/clinical-trials

-Ends-

Notes to editors:

For further information please visit https://www.edoncology.com/latest-news/

References:

1. Valle JW, et al. Ann Oncol. 2016;27(S5):v27–v37.
2.
NCI. https://www.cancer.gov/types/liver/hp/bile-duct-treatment-pdq.
Accessed 14.02.19
3. US FDA. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=671318
Accessed 14.02.19
4. ACS. https://www.cancer.org/cancer/bile-duct-cancer/about/key-statistics.html.
Accessed 14.02.19
5. ECIS. https://ecis.jrc.ec.europa.eu/.
Accessed 14.02.19
6. CCF. https://cholangiocarcinoma.org/.
Accessed 14.02.19
7. Pape UF, et al. J Clin Oncol.
2019;3(S4):264.
8. EMA. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3141270
Accessed 14.02.19

Date of Preparation: February 2019

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