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On 12 November 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion following a re-examination procedure,  recommending the granting of a marketing authorisation for the medicinal product Elzonris1, intended for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN).

The applicant for this medicinal product is Stemline Therapeutics B.V.

Elzonris will be available as a 1-mg/ml concentrate for solution for infusion. The active substance of Elzonris is tagraxofusp, an antineoplastic agent (ATC code: L01XX67). Tagraxofusp comprises a truncated diphtheria toxin (DT) fusion protein that is linked with recombinant human interleukin-3 (IL 3)  to target CD123-expressing cells. Tagraxofusp irreversibly inhibits protein synthesis of target cells by inactivating elongation factor 2 (EF2), resulting in apoptosis (cell death).

The benefits of Elzonris are that it can lead to complete response, with a rate (CR/Crc)[2] of 53.8% as shown in a study involving 13 previously untreated patients. The most common side effects are hypoalbuminaemia, increased transaminases, thrombocytopenia, nausea, fatigue and pyrexia observed in >20% pf patients. The most serious adverse reaction that may occur during treatment with Elzonris is capillary leak syndrome which was reported in 17% of patients with a median time to onset of 6 days.

The full indication is:

Elzonris is indicated as monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) (see section 5.1). Elzonris should be administered under the supervision of a physician experienced in the use of anti-cancer agents.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.

1 This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained.

2 complete resolution of the disease (CR) or CR with residual skin abnormality not indicative of active disease (CRc).

Key facts

Agency product number
International non-proprietary name (INN) or common name
  • tagraxofusp
Active substance
  • tagraxofusp
Therapeutic area

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.

Date opinion adopted
Company name
Stemline Therapeutics B.V.
Application type
Initial authorisation

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