Sobi® today announced that the
Aspaveli is already approved in
"Today's approval underscores the robust clinical data supporting Aspaveli 's efficacy and safety profile, offering healthcare professionals and patients an expanded toolkit for effectively managing PNH," said
PNH is a rare, chronic and life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells. Characterised by persistently low haemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue caused by anaemia. Despite improvements with C5 inhibitor treatment, up to 86 per cent of people with PNH treated with C5 inhibitors remain anaemic, according to cross-sectional surveys conducted in US and EU.1,2, 3,
The indication extension is based on data from APL2-308 (
Aspaveli is the European trade name for pegcetacoplan, which is known as Empaveli® in
About Aspaveli®/ Empaveli®
Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as Aspaveli/Empaveli in the
About the Sobi and Apellis collaboration
Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive US commercialisation rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).
About Sobi®
Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across
Contacts
To contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.
References
- Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol 2022;101(2):251-63.
- de Fontbrune F, Burmester P, Piggin M, et al. The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors: clinical outcomes and medical encounters from the patient perspective. Hematology 2022;27(1):1140-51.
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Panse J, de Fontbrune FS, Burmester P, et al. The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors in
France ,Germany and theUnited Kingdom : Patient-reported insights on symptoms and quality of life. Eur J Haematol 2022;109(4):351-63. - Wong RSM, Navarro-Cabrera JR, Comia NS, et al. Pegcetacoplan controls hemolysis in complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria. Blood Adv 2023;7(11):2468-78;
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ClinicalTrials.gov. A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH. Retrieved
January 18, 2024 from https://clinicaltrials.gov/study/NCT04085601
Postal address SE-112 76
Phone: 46 8 697 20 00 https://www.sobi.com
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