Idhifa

Application withdrawn

The application for this medicine has been withdrawn

Overview

Celgene Europe B.V. withdrew its application for a marketing authorisation of Idhifa for the treatment of adults with acute myeloid leukaemia (AML), a cancer of white blood cells.

The company withdrew the application on 6 December 2019.

What is Idhifa and what was it intended to be used for?

Idhifa was developed as a cancer medicine for the treatment of AML in adult patients whose cancer cells have a mutation (change) in the gene for a protein called IDH2 and who cannot receive intensive cancer treatment. Idhifa was to be used in patients whose disease did not respond to treatment (refractory) or had come back (relapsed) after previous treatments including a haematopoietic stem cell transplant (a transplant of cells that can develop into different types of blood cells).

Idihifa contains the active substance enasidenib and was to be available as tablets.

Idhifa was designated an ‘orphan medicine’ (a medicine used in rare diseases) for the treatment of AML on 28 April 2016.

How does Idhifa work?

The active substance in Idhifa, enasidenib, works by blocking the action of mutated forms of IDH2, a protein that plays an important role in generating energy for cells. Mutated IDH2 produces high levels of a substance called D-2-HG, which contributes to the growth of cancer cells. By blocking the action of mutated IDH2, enasidenib is expected to reduce production of D-2-HG and so slow down the progression of the disease.

What did the company present to support its application?

Idhifa was investigated in a main study involving 214 patients with AML carrying an IDH2 mutation. Idhifa was not compared with any other medicine and the main measure of effectiveness was the number of patients who did not show signs of the disease after treatment.

How far into the evaluation was the application when it was withdrawn?

The application was withdrawn after the European Medicines Agency had evaluated the information from the company and prepared questions for the company. After the Agency had assessed the company’s responses to the last round of questions, there were still some unresolved issues.

What did the Agency recommend at that time?

Based on the review of the data and the company’s response to the Agency’s questions, at the time of the withdrawal, the Agency had some concerns and its provisional opinion was that Idhifa could not have been authorised for the treatment of AML.

The Agency considered that the results of the study did not allow to conclude that Idhifa was sufficiently effective in the treatment of relapsed or refractory AML with an IDH2 mutation.

Therefore, at the time of the withdrawal, the Agency’s opinion was that the benefits of Idhifa did not outweigh its risks.

What were the reasons given by the company for withdrawing the application?

In its Withdrawal letter: Idhifa notifying the Agency of the withdrawal of the application, the company stated that it could not fully address the major objections raised by the Agency.

Does this withdrawal affect patients in clinical trials or compassionate use programmes?

The company informed the Agency that there are no consequences for patients in clinical trials or in compassionate use programmes using Idhifa.

If you are in a clinical trial or compassionate use programme and need more information about your treatment, speak with your doctor.

Questions and answers on the withdrawal of application for the marketing authorisation of Idhifa (enasidenib)

Reference Number: EMA/47029/2020

Key facts

• Name of medicine:
  Idhifa
• Therapeutic area (MeSH):
  Leukemia, Myeloid, Acute
• Anatomical therapeutic chemical (ATC) code:
  L01XX
• EMA product number:
  EMEA/H/C/004324
• Marketing authorisation applicant:
  Celgene Europe BV
• Withdrawal of application:
  06/12/2019

All Documents

Withdrawal letter: Idhifa

Withdrawal assessment report for Idhifa

AdoptedReference Number: EMA/CHMP/13746/2020