First oral monotherapy for patients with PNH

April 8, 2024

First oral monotherapy for patients with paroxysmal nocturnal haemoglobinuria


EMA has recommended granting a marketing authorisation in the European Union (EU) for Fabhalta (iptacopan), an oral treatment for adults with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

PNH is a rare genetic disorder that causes the premature breakdown of red blood cells (haemolytic anaemia) by the immune system, and is potentially life-threatening. Disease symptoms include fatigue, body pain, blood clots, bleeding and shortness of breath. PNH generally worsens over time and patients often require red blood cell transfusions.

The standard treatment for PNH is anti-C5 monoclonal antibodies (eculizumab or ravulizumab), known as complement inhibitors, administered via subcutaneous or intravenous infusion. A minority of PNH patients treated with complement inhibitors suffer from residual haemolytic anaemia and require blood transfusions, The active substance of Fabhalta is iptacopan, a proximal complement inhibitor. Complement inhibitors are a type of immunotherapy used in the treatment of many inflammatory conditions that could be caused by deficiencies of the complement system, a part of the body's immune system. Iptacopan targets Factor B to selectively inhibit the alternative complement pathway and prevent the destruction of red blood cells within blood vessels (intravascular haemolysis) and in the liver and spleen (extravascular haemolysis).

EMA’s recommendation is based on the results of two phase III trials.

Fabhalta’s most frequent side effects in patients were upper respiratory tract infection, headache and diarrhea.

Fabhalta was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs, The opinion adopted by the CHMP is an intermediary step on Fabhalta’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.

Check the link for the full article: First oral monotherapy for patients with paroxysmal nocturnal haemoglobinuria | European Medicines Agency (europa.eu)

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