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news.Novartis has received a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) to grant marketing authorisation for Fabhalta (iptacopan) to treat adults with paroxysmal nocturnal haemoglobinuria (PNH) who suffer from haemolytic anaemia.
The positive opinion is based on the findings from two Phase III trials in paroxysmal nocturnal haemoglobinuria patients. Credit: ANIRUDH on Unsplash.
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This endorsement is a significant step towards the availability of Fabhalta for PNH patients in Europe.
The positive opinion is based on data from two Phase III clinical trials: APPLY-PNH and APPOINT-PNH.
APPLY-PNH was a multinational, randomised, multicentre, active-comparator controlled, open-label trial involving 97 subjects who were previously treated with anti-C5 therapies.
The open-label, uncontrolled single-arm APPOINT-PNH study had 40 adult patients new to complement inhibitor therapy.
In the APPLY-PNH study, after 24 weeks, 82.3% of subjects who switched to Fabhalta from anti-C5 treatment showed a sustained increase in haemoglobin levels of at least 2g/dL without requiring transfusions. This was in stark contrast to just 2% of subjects who continued with anti-C5 treatment.
The APPOINT-PNH study reported a similar outcome, with 92.2% of complement inhibitor-naïve patients achieving the same haemoglobin increase on Fabhalta.
Moreover, the APPLY-PNH trial demonstrated a notable transfusion avoidance rate of 94.8% for patients treated with Fabhalta, versus 25.9% for those on anti-C5 therapy.
Both studies confirmed the potential of Fabhalta in controlling intravascular haemolysis (IVH), with patients maintaining mean lactate dehydrogenase (LDH) levels at less than 1.5 times the upper limit of normal.
Additionally, patients in the APPLY-PNH study reported improvements in fatigue symptoms.
The safety profile of iptacopan, the active ingredient in Fabhalta, remained consistent across both studies.
Based on the CHMP recommendation, the European Commission (EC) is expected to make a final decision on the marketing authorisation for Fabhalta within approximately two months.
Novartis International president Patrick Horber said: “If approved by the European Commission, Fabhalta would be the first oral monotherapy available to PNH patients in Europe.
“With current standard of care, PNH symptoms are often uncontrolled, while patients endure regular and time-consuming infusions. This oral therapy could provide a much-needed alternative to support many people living with PNH who often have to structure their lives around managing their condition.’’
