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

Basel, Switzerland, 22 February 2019 – 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

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 relative survival rate for patients diagnosed with early-stage biliary tract cancer is approximately 30%.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:


About Biliary Tract Cancer

Biliary tract cancer can develop in any part of the bile duct system. For those diagnosed with early-stage disease, the five-year relative survival is 30%; the prognosis is worse for patients with intra-hepatic cancer compared with those with extra-hepatic cancer. Nearly two out of three people with biliary tract cancer are age 65 or older at the time of diagnosis, and the average age is 70. Risk factors for biliary tract cancer include age older than 65 years, alcohol consumption, bile duct stones, cirrhosis, diabetes, a family history of cholangiocarcinoma, inflammatory bowel disease (ulcerative colitis and Crohn’s disease), obesity and long-term infection with hepatitis B or C virus. Surgery and chemotherapy are commonly used for early and locally advanced disease, but no treatments are indicated as second-line therapy. Clinical trials of targeted agents and immunotherapy are under way.6

About Etoposide Toniribate

Etoposide toniribate is a novel topoisomerase II inhibitor in clinical development for the treatment of relapsed/refractory biliary tract cancer. This small molecule drug is designed to work by metabolising into its active form through enzymes in the gastrointestinal tract that are active in cancer cells. Specifically, etoposide toniribate is hydrolysed at the site of the tumour by carboxylesterase, an enzyme expressed by some tumours, to produce activated etoposide. Activated etoposide binds to and inhibits topoisomerase II, which is often elevated in tumours, resulting in double-strand breaks in tumour DNA. Damage to the tumour DNA induces apoptosis (programmed cell death).

Results of a randomised Phase II trial of 23 patients with refractory, metastatic, unresectable biliary tract cancer who had relapsed following treatment with gemcitabine/cisplatin showed a one-year overall survival of 44% with etoposide toniribate versus 11.3% with best supportive care (BSC). Overall, 55.6% of patients met the primary endpoint of disease control compared with 20.0% who received BSC. In the trial, etoposide toniribate was generally well tolerated with a predictable toxicity profile. The most common drug-related adverse events were neutropenia, leukopenia, thrombocytopenia, anaemia, alopecia, fatigue and abdominal pain.7

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. We are 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. We have built a robust and diversified pipeline of investigational drug candidates, and we actively collaborate with industry and academic partners to identify and advance future impactful medicines.

For more information, please visit:

About the Mundipharma network

The Mundipharma global network of privately-owned independent associated companies was founded in 1956 by doctors, and now operates in over 120 countries worldwide. We are focused on developing business partnerships to identify and accelerate meaningful technology across an increasingly diverse portfolio of therapy areas including respiratory, oncology, pain, and biosimilars. Consistent with our entrepreneurial heritage, we like to think we see what others don’t by challenging conventional wisdom and asking different and challenging questions. By working in partnership with all our stakeholders, the Mundipharma network develops medicines that create value for patients, payers and wider healthcare systems.

For more information please visit:


For further information please contact:                                        



Tiffany Fretwell

Communications Lead, Mundipharma

[email protected]

+44 (0) 1223 393 361


Helen Laurence

Makara Health

[email protected]

Tel: +44 (0) 23 81 247 327



[email protected]




  1. Valle JW, et al. Annals of Oncology. 2016; 27(Supplement 5):v27–v37.
  2. National Cancer Institute. Last accessed 14 February 2019.
  3. US Food and Drug Administration. accessed 14 February 2019.
  4. American Cancer Society. Key Statistics for Bile Duct Cancer. accessed 14 February 2019.
  5. ECIS European Cancer Information Service. Last accessed 14 February 2019.
  6. Cholangiocarcinoma Defined Factsheet. Last accessed 14 February 2019.
  7. Pape UF, et al. Journal of Clinical Oncology. 2019; 3(Supplement 4):264.
  8. European Medicines Agency accessed 14 February 2019.