This press release is issued in order to provide information to the media, shareholders, and investors; the information contained herein does not constitute product promotion or medical advice.
This press release concerns a regulatory approval in Japan. The information contained herein is specific to Japan and may differ from information approved in other countries or regions.
Swedish Orphan Biovitrum Japan Co., Ltd. (Sobi Japan) and Asahi Kasei Therapeutics Corp. announced that Empaveli™ 1080 mg Solution for Subcutaneous Injection (pegcetacoplan; hereinafter, “Empaveli”) has received approval in Japan for additional indications: C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis. With this approval, Empaveli will provide a new treatment option for patients, including pediatric patients for whom treatment options have been limited. It is the first complement inhibitor in Japan approved for patients aged 12 years and older with C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis.
Empaveli is a PEGylated peptide that binds to complement proteins C3 and C3b, inhibiting the upstream portion of the complement cascade. The drug was approved in Japan in 2023 as a treatment for paroxysmal nocturnal hemoglobinuria and is supplied by Sobi Japan, the marketing authorization holder, and by Asahi Kasei Therapeutics as the distributor.
C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis are rare, serious, and progressive kidney diseases whose pathophysiology involves abnormal activation of the complement pathway. In these diseases, C3 deposition in the renal glomeruli is considered an important indicator of disease activity. Excessive complement activation may lead to kidney inflammation, tissue damage, and a decline in renal function. Patients present with persistent proteinuria, hematuria, edema, and reduced renal function. Furthermore, disease progression may result in end-stage renal failure requiring dialysis or kidney transplantation.
Although C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis are rare diseases with extremely small patient populations, they occur across a wide range of age groups. The disease poses a significant long-term burden on patients and their families. The diseases may also recur after kidney transplantation, highlighting the need for treatment options that address the underlying pathophysiology of these diseases. To date, disease-specific therapies have been limited, especially among pediatric patients. The majority of treatment options have not been aimed at preserving renal function and suppressing disease progression.
This approval is based on the results of the global Phase III VALIANT study, among other data. This study evaluated the efficacy and safety of Empaveli in patients with C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis. VALIANT was a randomized, placebo-controlled, double-blind, multicenter study in patients aged 12 years and older with C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis. The study enrolled 124 patients.
In the VALIANT study, the Empaveli group achieved the primary endpoint by reducing the urine protein-to-creatinine ratio by 68.1% compared with the placebo group at Week 26. Renal function, as assessed by estimated glomerular filtration rate (eGFR), was +6.3 mL/min/1.73 m² versus the placebo group, indicating stabilization of renal function. In addition, the proportion of subjects with a reduction of two or more grades in C3c staining from baseline in the kidney biopsy was 74.3% in the Empaveli group. This is compared with 11.8% in the placebo group. The safety profile was generally consistent with that previously established for Empaveli.
With this approval, Sobi Japan and Asahi Kasei Therapeutics will work with healthcare professionals, including those in nephrology, pediatric nephrology, and pathological diagnosis. They aim to improve disease awareness, promote early and appropriate diagnosis, and advance the proper use of Empaveli, thereby benefiting patients suffering from these intractable kidney diseases.
Daisuke Okajima, President of Asahi Kasei Therapeutics, commented, “We are thrilled for Empaveli’s approval in Japan for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis. Though rare, these are progressive and difficult-to-treat conditions that impose serious burdens on patients and their families. I find it highly notable that this is the first approval of a complement inhibitor for pediatric use for these indications in Japan. Asahi Kasei Therapeutics looks forward to continuing to work with Sobi Japan to advance the proper use of Empaveli while raising awareness of these diseases and supporting their early diagnosis.”
Asahi Kasei positions Pharmaceuticals as a First Priority business in its medium-term management plan, emphasizing value creation by expanding its research and development activities globally to deliver innovative therapeutic options worldwide.
About Swedish Orphan Biovitrum
Swedish Orphan Biovitrum was established in 2009 and is a biopharmaceutical company headquartered in Stockholm, Sweden, specializing in the treatment of rare and intractable diseases. The company operates globally in the fields of hematology, immunology, and specialty care, with approximately 2,000 employees working every day across Europe, North America, the Middle East, Asia, and Australia. Sobi Japan, its Japanese subsidiary, was established in June 2020 and conducts activities to “deliver innovative new medicines to patients with rare and intractable diseases and help them live with smiles.” For more information about Sobi, please visit https://www.sobi.com/.