EU/3/15/1463: Orphan designation for the treatment of plasma cell myeloma

Melphalan flufenamide


On 19 March 2015, orphan designation (EU/3/15/1463) was granted by the European Commission to Oncopeptides AB, Sweden, for melphalan flufenamide for the treatment of plasma cell myeloma.

Please note that this product (marketed as Pepaxti) was withdrawn from the Union Register of orphan medicinal products by the European Commission in July 2022 at the time of the granting of a marketing authorisation.

Key facts

Active substance
Melphalan flufenamide
Intended use
Treatment of plasma cell myeloma
Orphan designation status
EU designation number
Date of designation
Oncopeptides AB
Västra Trädgårdsgatan 15
111 53 Stockholm
Tel. +46 709 957 220

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

The Committee for Orphan Medicinal Products reviewed the orphan designation of melphalan flufenamide at the time of marketing authorisation. The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the withdrawal assessment report – orphan maintenance

Update history

Date Update
July 2022 Product withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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