Novartis’ Fabhalta (iptacopan) has been approved by the US Food and Drug Administration (FDA) as the first oral monotherapy for adults with paroxysmal nocturnal haemoglobinuria (PNH), a rare blood disease affecting approximately ten to 20 people per million worldwide.

PNH patients have an acquired mutation that causes them to produce red blood cells susceptible to premature destruction by the complement system, which can cause anaemia, thrombosis, fatigue and other symptoms that can impact quality of life.

The disease has a significant unmet need, Novartis reports, with a large proportion of patients on anti-C5 treatment remaining anaemic and dependent on blood transfusions.

Fabhalta is a factor B inhibitor of the immune system’s complement pathway and offers an alternative to therapies that need to be delivered by infusion or injection.

Victor Bultó, president US, Novartis, described the approval as an "extraordinary moment” for the PNH community.

"This new, effective oral medicine may mean that patients can reset their expectations of living with PNH,” he said.

The FDA’s decision on the drug, which applies to both previously treated and treatment-naïve patients, was based on the late-stage APPLY-PNH trial in adults with PNH and residual anaemia despite prior anti-C5 treatment.

Results showed that patients who switched to Fabhalta experienced superior increases of haemoglobin levels over those who continued on anti-C5 treatment.

The approval was also supported by positive results from the phase 3 APPOINT-PNH study in complement inhibitor-naïve patients, Novartis said.

“In clinical studies, [Fabhalta] was superior to anti-C5s in haemoglobin improvement in the absence of red blood cell transfusion and transfusion avoidance rate, and also effective in complement inhibitor-naïve individuals, by providing clinically meaningful haemoglobin-level increases without the need for blood transfusions,” said Vinod Pullarkat, clinical professor in the department of haematology and haematopoietic cell transplantation at City of Hope, US.

Bultó outlined that the company is exploring the potential of Fabhalta in other complement-mediated diseases “with an ultimate goal to drive meaningful change for patients”.

The approval comes just over a month after Novartis’ Cosentyx (secukinumab) was approved by the FDA to treat adults with moderate-to-severe hidradenitis suppurativa (HS).

The decision makes the IL-17A inhibitor, which is currently approved to treat a range of inflammatory conditions, the first biologic treatment approved for HS in nearly a decade.

https://www.pmlive.com/pharma_news/novartis_fabhalta_approved_by_fda_as_first_oral_monotherapy_for_rare_blood_disease_pnh_1504628