Hot Rare Blood Disorder Treatment Market... 'Ultomiris' High-Concentration Domestic Approval

Alexion Leads Market with 'Soliris' and 'Ultomiris'
Competition Continues in Soliris Biosimilar Development

Rare blood disorder treatment 'Ultomiris' developed by Alexion in the United States (Photo by Handok)

[Asia Economy Reporter Lee Chun-hee] Competition is intensifying to replace the rare blood disorder treatment 'Soliris' (generic name eculizumab), which costs 400 to 500 million KRW annually. While biosimilar development to replace the original continues, strategies to protect the market are also emerging.

On the 28th, the Ministry of Food and Drug Safety announced the approval of a high-concentration (100 mg/mL) treatment of 'Ultomiris' (generic name ravulizumab), a rare blood disorder treatment for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) by Handok.

Ultomiris is a treatment developed by Alexion in the U.S. for intractable rare blood disorders. It is used to treat PNH, which causes hemoglobin to escape from red blood cells leading to hemoglobinuria at night, and aHUS, which causes thrombi in blood vessels due to complement abnormalities, resulting in hemolytic anemia.

Ultomiris is a humanized monoclonal antibody that specifically binds to complement component 5 (C5), inhibiting complement-mediated inflammatory and hemolytic reactions. Alexion's previously developed 'Soliris' is also a C5 inhibitor with the same indications.

However, neither is a fundamental cure, requiring continuous administration, which places a heavy burden on patients. According to domestic standards, the drug ceiling price is about 5.13 million KRW for Soliris and about 5.6 million KRW for Ultomiris per vial, making Ultomiris more expensive per vial. However, the dosing interval differs significantly: Soliris is administered every 2 weeks, while Ultomiris is every 8 weeks, resulting in lower annual treatment costs for Ultomiris. Additionally, the recent approval of the high-dose formulation allows for a reduction in intravenous infusion time needed for patient injections of Ultomiris.

Alexion has dominated the PNH and aHUS treatment markets through Soliris. As of last year, Soliris's global sales reached $1.874 billion (approximately 2.38 trillion KRW), classifying it as a blockbuster drug. This was a key reason why global big pharma AstraZeneca acquired Alexion in 2020 for a massive $39 billion (about 49 trillion KRW).

With patent expiration approaching, companies like Samsung Bioepis and Amgen have begun developing biosimilars. Samsung Bioepis has demonstrated clinical equivalence to the original in a Phase 3 trial for PNH patients with 'SB12' and has applied for approval from the Korean Ministry of Food and Drug Safety and the European Medicines Agency (EMA). Amgen has also completed Phase 3 trials for 'ABP959', showing no clinically meaningful differences in safety, efficacy, or immunogenicity compared to the original. However, no biosimilar approvals for Soliris have been granted worldwide yet.

In response, Alexion has sought to defend its market share with the more convenient Ultomiris. Since its reimbursement in Korea in June last year, Ultomiris's domestic sales rapidly increased to 19.6 billion KRW, more than half of Soliris's 33 billion KRW sales.

However, the sales rights for Ultomiris and Soliris, previously held by Handok, are expected to transfer to Korea AstraZeneca starting next year. Handok has effectively led the domestic PNH market by selling Soliris since 2012 and Ultomiris since last year after reimbursement, but following Alexion's acquisition by AstraZeneca, all related sales rights will be reclaimed.

Alexion is also developing a PNH treatment called 'Danicopan (ALXN2040)'. In recent Phase 3 trials, it demonstrated statistically significant improvement in hemoglobin levels compared to placebo after 12 weeks of treatment. Unlike the C5 inhibitors Ultomiris and Soliris, Danicopan is an oral factor D inhibitor. It has also shown clinically meaningful improvements in combination therapy with Ultomiris, and Alexion is pursuing related combination therapy studies.

This content was produced with the assistance of AI translation services.