Sotatercept Medicine 07 December 2022 Extended indication Treatment of pulmonary arterial hypertension. Therapeutic value Possible added value Total cost € 119,700,000.00 Registration phase Registered Product Active substance Sotatercept Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Lung diseases other Extended indication Treatment of pulmonary arterial hypertension. Current proprietary name Already available biosimilars / generics Proprietary name Winrevair Manufacturer MSD Portfolio holder MSD Mechanism of action Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Particularity New medicine with Priority Medicines (PRIME) ATMP No Submission date October 2023 Expected Registration August 2024 Orphan drug Yes Registration phase Registered Reimbursement Reimbursement request pending Medicine sluice Additional remarks Positieve CHMP-opinie juni 2024. Zorginstituut Nederland beoordeelt of sotatercept (Winrevair®) vergoed kan worden. Aanlevering dossier compleet 17 november 2025. Publicatie advies wordt verwacht in juni of juli 2026. Therapeutic value Current treatment options Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Sotatercept zal volgens experts een mogelijke meerwaarde kunnen gaan hebben in de behandeling van PAH. Bij studie patiënten of patiënten in het Early Access programma worden goede resultaten gezien. Daarnaast zijn de resultaten uit de fase 3 studie indrukwekkend te noemen, zeker in vergelijking met de resultaten van veel andere medicamenten binnen deze indicatie. Duration of treatment Frequency of administration 1 times every 3 weeks Dosage per administration 0,7mg/kg References NCT04576988 (STELLAR) Additional remarks Sotatercept start met een dosering van 0,3mg/kg en een streefdosis van 0,7mg/kg subcutaan (SC). Toegediend elke 21 dagen, plus PAH-therapie als achtergrondbehandeling. Expected patient volume per year Patient volume 255 - 884 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References ntvg.nl Additional remarks De prevalentie van PAH in Nederland is ongeveer 15 tot 52 per miljoen inwoners. Expected cost per patient per year Cost € < 210,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References ICER Additional remarks ICER schat de lijstprijs van Winrevair op ongeveer $240.000 per jaar. Dit is ongeveer €210.000. Potential total cost per year Total cost € 119,700,000.00 Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Extension of indication that aims to include the use of WINREVAIR treatment for adult patients with PAH FC IV and to acknowledge that WINREVAIR treatment improves survival and reduces the risk of clinical worsening events, including lung transplantation and hospitalization for PAH. The proposed new indication is: Winrevair, in combination with other pulmonary arterial hypertension (PAH) therapies, is indicated for the long-term treatment of PAH in adult patients with WHO Functional Class (FC) II, III, and IV References https://pubmed.ncbi.nlm.nih.gov/40167274/ Humbert M, McLaughlin VV, Badesch DB, Ghofrani HA, Gibbs JSR, Gomberg-Maitland M, Preston IR, Souza R, Waxman AB, Moles VM, Savale L, Vizza CD, Rosenkranz S, Shi Y, Miller B, Mackenzie HS, Kim SS, Loureiro MJ, Patel MJ, Koglin J, Cornell AG, Hoeper MM; ZENITH Trial Investigators. Sotatercept in Patients with Pulmonary Arterial Hypertension at High Risk for Death. N Engl J Med. 2025 May 29;392(20):1987-2000. doi: 10.1056/NEJMoa2415160. Epub 2025 Mar 31. PMID: 40167274. Additional remarks Expected EMA approval mid 2026 based on input from scientific leaders approximately 15 additional patients per year. Other information Additional remarks