Novartis, PNH drug 'Fabhalta' enters drug pricing nego.

The NHIS process began after passing the Drug Reimbursement Evaluation Committee (DREC) in March

It is expected to overcome limitations related to conventional C5 inhibiotrs

Product photo of Fabhalta

The new oral drug 'Fabhalta' has entered the last phase of receiving approval for insurance reimbursement.

 

According to industry sources, Novartis Korea’s ’Fabhalta (iptacopan),’ a treatment for paroxysmal nocturnal hemoglobinuria (PNH), is under negotiations for drug pricing.

 

Consequently, attention is gaining to whether another treatment option for PNH would emerge.

 

PNH is a rare disease estimated to occur in approximately 1.5 individuals per 1 million globally.

 

Until now, the treatment for PNH has relied on C5 inhibitors.

 

In 2010, ’Soliris (eculizumab)’ was first approved in South Korea, and 'Ultomiris (ravulizumab)' has been used for PNH treatment since its approval in 2022.

 

Both treatment options are C5 inhibitors.

 

C5 inhibitors inhibit C5, the terminal component within the complement system's alternative pathway involved in the body's immune response, and are administered via intravenous injection.

 

In April last year, the subcutaneous injection product 'Empaveli (pegcetacoplan),' which works by binding to C3 and C3b to inhibit the complement cascade, was approved.

 

The oral drug Fabhalta, which operates by inhibiting factor B, was introduced in August.

 

Due to the mechanistic limitations of C5 inhibitors, there are still unmet needs for patients with PNH.

 

'Extravascular hemolysis (EVH)' PNH arises from a genetic deficiency in red blood cells and leads to both intravascular hemolysis (IVH) and extravascular hemolysis (EVH).

 

Such hemolysis subsequently triggers thrombosis and bone marrow failure, thereby endangering life.

 

Therefore, controlling hemolysis is critical for treating PNH.

 

However, the current standard treatment for PNH, a C5 inhibitor, effectively manages IVH but is inherently limited in its mechanism to control EVH.

 

This is why there is significant interest in the reimbursement status of the factor B inhibitor, Fabhalta.

 

Factor B is located higher in the alternative pathway than C5, C3, and C3b, and by inhibiting it, one can comprehensively regulate both IVH and EVH.

 

In fact, the efficacy of Fabhalta has been demonstrated in patients with no prior treatment experience.

 

According to the APPOINT-PNH study, conducted in treatment-naïve PNH patients, 19 out of 33 patients achieved a hemoglobin level of at least 12 g/dL without the need for red blood cell transfusions.

 

Furthermore, 92% of the patients showed a clinically significant increase in hemoglobin of at least 2 g/dL, and 63% maintained a hemoglobin level of 12 g/dL or higher without transfusions.

 

During the 24‑week study period, hemoglobin levels continued to rise steadily, reaching normalized levels by week 20 and remaining at that level through week 24.

 

Additionally, 98% of the patients overcame transfusion dependency.

 

Professor Junho Jang, Department of Hematology at Samsung Medical Center, said, "When a C5 inhibitor first emerged, experts stated that the PNH treatment paradigm has shifted.

 

However, C5 inhibitors are still limited in managing EVH." "Fabhalta is a new drug that is likely to bring a paradigm shift for PNH treatment.

 

This drug is involved in regulating factor B located in the upper alternative pathway, thus inhibiting factor B.

 

It can comprehensively control both IVH and EVH.

 

A favorable result has been demonstrated through clinical trials," Professor Jang emphasized.