Sanofi responds to NICE negative FAD of olipudase alfa
07.08.2025 - 18:04:38[iii] WORLDSymposium (2023) WORLDSymposium2023 New Treatment Award. Available at: https://worldsymposia.org/worldsymposium-2023-new-treatment-award/ [Accessed February 2024]
[iv] PR Newswire (2023) The Galien Foundation Announces 2023 Prix Galien USA Nominees for 'Best Biotechnology Product', 'Best Pharmaceutical Product' and 'Best Product for Rare/Orphan Diseases'. Available at; https://www.prnewswire.com/news-releases/the-galien-foundation-announces-2023-prix-galien-usa-nominees-for-best-biotechnology-product-best-pharmaceutical-product-and-best-product-for-rareorphan-diseases-301898938.html [Accessed February 2024]
[v] NORD (2023) NORD Announces 2023 Rare Impact Award Honourees and 40th Anniversary Celebration. Available at: https://rarediseases.org/announces-2023-ria-honorees/ [Accessed February 2024]
[vi] Scottish Medicines Consortium (2024) Olipudase alfa (Xenpozyme) Available at: https://www.scottishmedicines.org.uk/medicines-advice/olipudase-alfa-xenpozyme-ultra-orphan-smc2560/ [Accessed February 2024]
[vii] Department of Health and Social Care (2023) England Rare Diseases Action Plan 2023: Main Report. Available at: https://www.gov.uk/government/publications/england-rare-diseases-action-plan-2023/england-rare-diseases-action-plan-2023-main-report [Accessed February 2024]
[viii] NHS Wales (2024) Wales Rare Disease Action Plan 2022-2026. Available at: https://executive.nhs.wales/functions/networks-and-planning/rare-diseases/rdig-documents/action-plan-refresh-jan-2024/ [Accessed February 2024]
[ix] BioIndustry Association (2023) Evaluating patient access to rare disease treatments. Insights from the UK and Beyond. Available at: https://www.bioindustry.org/static/4509759a-9e70-4089-aaa7963f6b6bb1ec/d5fb0438-cb6e-49b1-96af17ff71a1f04b/BIA-PwC-report-Evaluating-patient-access-to-rare-disease-treatments.pdf [Accessed February 2024)
[x] National Organization for Rare Disorders (2022) Acid Sphingomyelinase Deficiency. Available at: https://rarediseases.org/rare-diseases/acid-sphingomyelinase-deficiency/ [Accessed February 2024]
[xi] McGovern, M.M., Avetisyan, R., Sanson, BJ. et al. Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD). Orphanet J Rare Dis 12, 41 (2017). https://doi.org/10.1186/s13023-017-0572-x
[xii] British National Formulary (BNF) Olipudase alfa. Available at: https://bnf.nice.org.uk/drugs/olipudase-alfa/#:~:text=Olipudase%20alfa%20is%20an%20enzyme,such%20as%20the%20liver%2C%20spleen%2C [Accessed February 2024]
[xiii] Pokrzywinski R, Hareendran A, Nalysnyk L, Cowie S, Crowe J, Hopkin J, Joshi D, Pulikottil-Jacob R. Impact and burden of acid sphingomyelinase deficiency from a patient and caregiver perspective. Sci Rep. 2021 Oct 25;11(1):20972. doi: 10.1038/s41598-021-99921-6. PMID: 34697402; PMCID: PMC8546120.
[xiv] National Institute for Health and Care Excellence (2022) Highly Specialised Technologies Evaluation. Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann Pick disease type B and AB). Final Scope. Available at: https://www.nice.org.uk/guidance/gid-ta10788/documents/final-scope [Accessed February 2024]
[xv] British National Formulary (BNF) Olipudase alfa. Available at: https://bnf.nice.org.uk/drugs/olipudase-alfa/#:~:text=Olipudase%20alfa%20is%20an%20enzyme,such%20as%20the%20liver%2C%20spleen%2C [Accessed February 2024]
[xvi] European Medicines Agency. Xenpozyme. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/xenpozyme [Accessed February 2024]
View original content:https://www.prnewswire.co.uk/news-releases/sanofi-responds-to-nice-negative-fad-of-olipudase-alfa-302079823.html
