New drugs and biosimilars to compete in the PNH market

Samsung Bioepis’s ‘Epysqli’ was granted approval in Korea, following its approval in Europe

Novartis' Fabhalta and AstraZeneca’s Voydeya are next in line for release in Korea

New drugs and biosimilars have shifted the competitive landscape of the treatment market for paroxysmal nocturnal hemoglobinuria (PNH), which Soliris and Ultomiris have previously dominated.

 

PNH is a rare, life-threatening disorder characterized by the destruction of red blood cells in the blood, leading to symptoms such as dark-colored urine and acute renal failure.

 

According to industry sources on the 30th, Samsung Bioepis’s Epysqli, a biosimilar version of Soliris, has recently received approval in Korea.

 

This marks the first domestic approval of a Soliris biosimilar.

 

Following the European approval of Epysqli in May of last year, Samsung Bioepis has now secured approval in Korea.

 

Samsung Bioepis’s Epysqli.

AstraZeneca owns Soliris, an inhibitor of complement component 5 (C5), with global sales amounting to 5 trillion won (about $3.7 billion won).

 

Soliris works by binding to the C5 protein, inhibiting complement activity and thereby preventing the destruction of blood cells.

 

Samsung Bioepis conducted a global Phase 3 clinical trial from August 2019 to October 2021.

 

The trial demonstrated the clinical bioequivalence of Epysqli to the original medicines.

 

AstraZeneca and Samsung Bioepis form the competitive landscape of the Soliris market.

 

However, Soliris distributor AstraZeneca is switching to the C5 complement inhibitor Ultomiris.

 

AstraZeneca introduced Ultomiris as a replacement for Soliris, as it expects the European patent to expire in 2023 and the U.S.

 

patent to expire in 2027.

 

Ultomiris offers an extended dosing interval of once every 8 weeks, in contrast to the intravenous administration of Soliris, which requires dosing every 2 weeks.

 

According to the drug market research company IQVIA, Soliris, which once had sales of 44 billion won in 2018, has experienced a steep decline in sales, with figures dropping to 31 billion won in 2020 and further down to 10.1 billion won in 2022.

 

In Q3 last year, Soliris net sales saw a year-on-year drop of 23.7%.

 

In the same period, Ultomiris sales showed rapid growth.

 

Released in Q3 2021, Ultomiris topped sales of 43.2 billion won in 2022.

 

In Q3 last year, Ultomiris sales saw a year-on-year increase of 14.2%.

 

New drugs, beyond biosimilars, are waiting to be released in Korea A competitive landscape is expected in the PNH market as drugs other than Soliris and Ultomiris await release.

 

One of the major pharmaceutical industry competitors is Novartis.

 

Novartis' oral PNH treatment, Fabhalta, was recently approved in the United States.

 

Fabhalta is a B-factor inhibitor that controls the destruction of red blood cells in the complement alternative pathway.

 

Fabhalta's advantage is its formulation.

 

Unlike existing intravenous formulations like Soliris and Ultomiris, Fabhalta is an oral medication that provides greater convenience.

 

Novartis is currently conducting five Phase 3 clinical trials in Korea to evaluate the efficacy and safety profile of Fabhalta.

 

The efficacy of Fabhalta was confirmed in patients who did not respond to C5 complement inhibitors or had not receive previous treatment.

 

In clinical trials, Fabhalta treatment resulted in a hemoglobin level increase of more than 2 g/dL from baseline in the absence of red blood cell transfusions in 82% of the patients at 24 weeks.

 

AstraZeneca has also achieved success in developing an oral treatment.

 

On the 19th, AstraZeneca stated that their orally available inhibitor of D factor Voydeya received approval in Japan.

 

Voydeya can be administered in combination with C5 inhibitors to adult patients with PNH who did not respond well to C5 complement inhibitors.

 

Voydeya is assessed to help mitigate the side effects of C5 complement inhibitors, which can lead to extravascular hemolysis (EVH) and subsequent anemia in certain patients.

 

The approval of Voydeya was based on a multi-national Phase 3 ALPHA clinical trial.

 

In clinical trials, Voydeya met key assessment criteria, including hemoglobin levels, in patients who exhibited EVH when administered with C5 complement inhibitors.

 

The safety profile evaluation has shown common side effects, including headaches and diarrhea.

 

Voydeya has been granted Breakthrough Therapy designation by the US Food and Drug Administration (FDA) and PRIority MEdicines (PRIME) status by the European Medicines Agency.

 

In addition to Voydeya, Roche is developing a new C5 complement inhibitor called crovalimab.

 

Crovalimab is currently undergoing review for approval in the United States, Japan, and Europe.

 

Under development as a subcutaneous (SC) delivery, crovalimab’s efficacy has been demonstrated in a once-every-four-week treatment.