Novartis has shared positive results from a phase 3b study of Fabhalta (iptacopan) in a new population of patients with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).
The APPULSE-PNH trial has been evaluating twice-daily Fabhalta in adults with PNH and higher haemoglobin levels of at least 10g/dl, expanding the clinical evidence base for the drug. Patients enrolled to the trial had also switched from the anti-C5 therapies eculizumab or ravulizumab.
In PNH, an acquired mutation causes patients to produce red blood cells susceptible to premature destruction by the complement system, potentially leading to anaemia, thrombosis, fatigue and other symptoms that can impact quality of life. The disorder affects approximately ten to 20 people per million worldwide.
Results presented at this year’s European Hematology Association (EHA) Congress showed that, after 24 weeks of treatment with Fabhalta monotherapy, haemoglobin levels improved on average by 2.01 g/dl and most patients achieved normal or near-normal levels.
No patients required transfusion during the study and Fabhalta was associated with clinically meaningful improvements in fatigue. Patients receiving Novartis’ drug also maintained intravascular haemolysis control and resolved extravascular haemolysis control.
“The positive results from APPULSE-PNH reinforce that Fabhalta can provide clinically meaningful improvements in haemoglobin among patients with higher baseline haemoglobin levels than those enrolled in previous trials, while offering an oral monotherapy for patients,” said Austin Kulasekararaj, consultant haematologist at King’s College Hospital and King’s College London.
Fabhalta, an oral Factor B inhibitor of the alternative complement pathway, already holds approvals to treat adults with PNH. The drug is also authorised to treat immunoglobulin A nephropathy and complement 3 glomerulopathy, two rare kidney diseases.
Shreeram Aradhye, president, development and chief medical officer, Novartis, said outlined that Fabhalta is the “first and only oral monotherapy currently available for the treatment of adults with PNH, regardless of previous treatment experience”.
Alongside results from APPULSE-PNH, longer-term data from patients initially included in the phase 3 APPLY-PNH and APPOINT-PNH trials of the drug were presented at EHA.
“New data from APPULSE-PNH, combined with findings from the phase 3 roll-over extension of the APPLY-PNH and APPOINT-PNH studies, reinforces the efficacy and safety profile of Fabhalta in delivering real benefits to patients,” Aradhye said.
