Iptacopan

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Novartis announces positive results for Fabhalta in Phase 3B APPULSE-PNH study, showing improved haemoglobin levels and consistent safety profile. Scemblix also shows superior major molecular response rates in Phase 3 ASC4FIRST study for CML patients.

Novartis reports positive outcomes from Phase IIIB APPULSE-PNH trial of Fabhalta for treating adults with paroxysmal nocturnal haemoglobinuria, showing improved haemoglobin levels and consistent safety profile.

Novartis announced positive Phase IIIB results for Fabhalta in PNH patients, showing improved hemoglobin levels. Fabhalta, approved by FDA and EMA, targets rare kidney diseases. Novartis also reported long-term success with Scemblix in leukemia, aiming to strengthen its pipeline through strategic acquisitions and collaborations.

Novartis' Fabhalta (iptacopan) demonstrated improved Hb levels in PNH patients switched from anti-C5 therapies in the APPULSE-PNH phase IIIB study. Fabhalta's safety profile was consistent with previous data, reinforcing its potential as an oral monotherapy for PNH treatment.

Novartis announced positive results from the phase 3B APPULSE-PNH study, showing iptacopan's efficacy as a twice-daily oral monotherapy for PNH patients transitioning from anti-C5 therapies, with significant Hb level improvements over 24 weeks. The study enrolled 52 participants stabilized on anti-C5 therapies for at least 6 months, with baseline Hb levels of at least 10 g/dL. Iptacopan demonstrated an average increase in Hb levels, addressing anemia in PNH management. The safety profile remained consistent with previous trials, and detailed data will be presented at a 2025 medical meeting. Additionally, the Max Foundation expanded its collaboration with Novartis to provide iptacopan access for PNH treatment in low-resource countries.

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APPULSE-PNH Phase IIIB study shows oral Fabhalta® (iptacopan) improves hemoglobin levels in adults with PNH switched from anti-C5 therapies, with consistent safety profile. Fabhalta is approved in the US, EU, Japan, and China for PNH treatment.