Fabhalta

Opinion

EMA has issued an opinion on this medicine

Iptacopan

Overview

On 21 March 2024, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Fabhalta¹, intended for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). The applicant for this medicinal product is Novartis Europharm Limited.

Fabhalta will be available as a 200 mg hard capsule. The active substance of Fabhalta is iptacopan, a complement inhibitor (ATC code: L04AJ08). Iptacopan targets Factor B to selectively inhibit the alternative complement pathway and control both C3-mediated extravascular haemolysis and terminal complement-mediated intravascular haemolysis.

The benefit of Fabhalta is its ability to prevent haemolysis to increase and maintain haemoglobin levels in patients with PNH, as shown in both an active comparator-controlled and single-arm phase 3 study. The most common side effects are upper respiratory tract infection, headache and diarrhoea.

The full indication is: 

Fabhalta is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia. 

Fabhalta should be prescribed by physicians experienced in the management of patients with haematological disorders.

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. 

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¹This 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

CHMP summary of positive opinion for Fabhalta

AdoptedReference Number: EMA/CHMP/54481/2024

Product details

• Name of medicine:
  Fabhalta
• Active substance:
  Iptacopan hydrochloride monohydrate
• International non-proprietary name (INN) or common name:
  Iptacopan
• Therapeutic area (MeSH):
  Hemoglobinuria, Paroxysmal
• Anatomical therapeutic chemical (ATC) code:
  L04A
• EMA product number:
  EMEA/H/C/005764
• 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:
  Novartis Europharm Limited
• Opinion adopted:
  21/03/2024
• Opinion status:
  Positive