Aplidin
Application withdrawn
The application for this medicine has been withdrawn
plitidepsin
Medicine
Human
Application withdrawn
- Application under evaluation
- CHMP opinion
- Withdrawal of application
Overview
PharmaMar withdrew its application for a marketing authorisation of Aplidin for the treatment of multiple myeloma.
The company withdrew the application on 23 July 2025 during a re-examination.
Aplidin was developed as a medicine for treating adults with multiple myeloma (a cancer of the bone marrow) who have received at least three prior cancer treatments (including bortezomib and either lenalidomide or thalidomide). Aplidin was to be used in combination with dexamethasone (another medicine used to treat multiple myeloma).
Aplidin contains the active substance plitidepsin. It was to be available as powder and solvent to be made up into a solution for infusion (drip) into a vein.
Aplidin was designated an ‘orphan medicine’ (a medicine to be used in rare diseases) on 16 November 2004 for the treatment of multiple myeloma.
The active substance in Aplidin, plitidepsin, blocks a protein called eEF1A2. eEF1A2 is involved in breaking down wrongly folded proteins, which are toxic to myeloma cells. By blocking eEF1A2, plitidepsin causes the accumulation of these proteins in multiple myeloma cells, damaging them and ultimately leading to their death.
The company presented the results of one main study involving 255 patients with multiple myeloma who had been treated with at least 3 other cancer medicines. In this study, Aplidin plus dexamethasone was compared with dexamethasone on its own, and the main measure of effectiveness was progression-free survival (how long patients lived without their disease getting worse).
The evaluation had finished and the European Medicines Agency had recommended refusing marketing authorisation. The company had requested a re-examination of the Agency’s recommendation, but it withdrew the application before this re-examination had finished.
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 recommended refusing marketing authorisation for Aplidin for the treatment of multiple myeloma.
The Agency was concerned that the data from the main study showed only a modest increase of around one month in the time patients given Aplidin lived without their disease getting worse, compared with those treated with dexamethasone alone. In addition, improvement in overall survival (how long patients lived overall) was not sufficiently demonstrated. Regarding safety, severe side effects were reported more frequently with the combination of Aplidin and dexamethasone than with dexamethasone alone.
In its letter notifying the Agency of the withdrawal of the application, the company stated that their decision was based on a change in their marketing strategy.
The company informed the Agency that there are no consequences for patients in clinical trials with Aplidin. If you are in a clinical trial and need more information about your treatment, speak with your clinical trial doctor.
Key facts
- Name of medicine
- Aplidin
- Active substance
- plitidepsin
- International non-proprietary name (INN) or common name
- plitidepsin
- Therapeutic area (MeSH)
- Multiple Myeloma
- Anatomical therapeutic chemical (ATC) code
- L01
- EMA product number
- EMEA/H/C/004354
Orphan
This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.
- Marketing authorisation applicant
- Pharma Mar S.A.
News on Aplidin
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