Celgene has asked European regulators to re-evaluate licensing application for lymphoma treatment Istodax (romidepsin) after EMA scientific advisors decided not to recommend the drug for use in Europe.
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) gave Istodax a 'negative opinion', citing problems with the study design that meant it was not possible to assess the medicine's effect on overall survival or progression free survival in comparison with treatments.
The CHMP also said that Celgene failed to provide an adequate certificate of Good Manufacturing Practice for the site where the medicine is manufactured - a legal requirement for any drug sold in the EU.
Responding to the announcement, Celgene said the CHMP acknowledged Istodax showed anti-tumour activity in trials, and the company remained convinced the drug has a favourable enough benefit/risk profile for it to be approved for use in patients with relapsed or refractory peripheral t-cell lymphoma (PTCL) who had failed two prior treatments.
“The company will therefore, in accordance with European regulations, request a re-examination of the CHMP opinion,” the company said in a statement.
PTCL is a group of rare types of lymphoma that develop from mature T-cells that are thought to account for between 10 and 15 per cent of all non-Hodgkin lymphomas.
Current treatment options include generic chemotherapies vincristine and doxorubicin as part of multi-drug regimens, although relapse rates are common, creating a demand for improved treatments.
Despite this unmet medical need, Istodax is the second new drug to be turned down for the treatment of relapsed PTCL by the CHMP this year following the Committee's negative opinion on Allos Therapeutics' Folotyn in February 2012.
In its Folotyn opinion, the CHMP also highlighted the trial design as a problem, with a lack of comparator product again meaning overall survival or progression free survival rates could not be measured.
Like Celgene, Allos also asked for a re-examination, although this did not resolve any of the CHMP's concerns, and the Committed confirmed its negative opinion in April, 2012.
Despite their struggles in Europe, both drugs have been approved stateside by US regulator the Food and Drug Administration (FDA).