Sanofi responds to NICE negative FAD of olipudase alfa

LONDON, March 5, 2024 /PRNewswire/ --

Rippon Ubhi, Country Lead, Sanofi UK and Ireland, said:

"We are extremely disappointed and saddened by the decision by the National Institute for Health and Care Excellence (NICE) not to recommend Xenpozyme (olipudase alfa) as a treatment for acid sphingomyelinase deficiency (ASMD; Niemann-Pick Disease) in people with type AB or type B. This is devastating news for the ASMD community.

ASMD is a progressive, life-limiting, ultra-rare disease^[i] that affects a very small number of children and predominantly young adults (approximately 40 people in the UK).^[ii] Olipudase alfa has been recognised as an innovative treatment with the potential to change the lives of patients and their families^{.[iii],[iv],[v]}  yet this decision means that patients with type AB or type B in England and Wales are unable to access the only licenced treatment for this disabling condition.

This is in stark contrast to Scotland, where the Scottish Medicines Consortium (SMC) has made olipudase alfa available via its ultra-orphan pathway.^[vi] This pathway, created to assess medicines for very rare conditions, allows eligible patients to access olipudase alfa whilst additional data is collected for three years.^vi 

As such, patients with ASMD in England and Wales will be denied access to treatment with olipudase alfa because of where they live, creating inequity in access within the UK. In recognition of the urgent unmet need in England and Wales, Sanofi proposed a number of commercial arrangements to support access to olipudase alfa. Despite these efforts, we regret that no solution could be found within the constraints of the existing framework.

We believe this decision demonstrates that the framework for assessing and funding innovative treatments for ultra-rare diseases in England and Wales is fundamentally flawed and is failing to keep pace with innovation. In our view, patients with ultra-rare diseases in England and Wales are being let down by an inflexible appraisal system which fails to recognise the benefits and full value of treatments such as olipudase alfa for patients, carers, families and wider society.

Without change we believe that England and Wales risk falling short on the commitment to improve rare disease care and treatment^[vii],[viii],and will progressively fall further behind our European neighbours when it comes to patient access.^[ix] Olipudase alfa is already available to patients in a number of other countries, including Germany, Austria, Italy, Cyprus, and Hungary. In England only 59% of non-oncology orphan medicines with a marketing authorisation are reimbursed, compared to 86% in Germany and 77% in France.^ix 

We are keen to have the opportunity to engage with Government, NICE and NHS England to urgently discuss and adapt the way that innovative treatments for ultra-rare conditions are assessed, to ensure that patients in England and Wales can access the same potentially life-changing treatments as those living elsewhere in the UK, and in other countries.

Building on our heritage in rare diseases, Sanofi remains deeply committed to patients with rare diseases, including the ASMD community, and will continue working towards improving their lives through the development of new and innovative treatments."

Notes to Editors: 

Contact details

Nadav Cohen-Keidar:
[email protected] 

Grant Fisher
[email protected].uk
+44 (0) 7947 573313

About ASMD (Niemann-Pick disease)

Acid Sphingomyelinase Deficiency (historically known as Niemann-Pick disease) type A/B or B is a lysosomal storage disorder that results from a deficiency of the enzyme acid sphingomyelinase.^[x]

ASMD is a progressively debilitating and life-limiting ultra-rare disease.^[xi] ASMD has a highly variable severity and disease course^xi and affects the normal function of tissues and organs such as the liver, spleen and lungs.^[xii] The debilitating nature of the condition can have a significant physical, financial and emotional impact on families and caregivers.^[xiii]

It is estimated that around 1 to 2 people are diagnosed with ASMD type A, B or AB per year, and most are diagnosed in childhood.^[xiv]

About olipudase alfa

Olipudase alfa is indicated as an enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in paediatric and adult patients with type A/B or type B.^{[xv], [xvi]} 

^[i] Scarpa M, Barbato A, Bisconti A, Burlina A, Concolino D, Deodato F, Di Rocco M, Dionisi-Vici C, Donati MA, Fecarotta S, Fiumara A, Galeone C, Giona F, Giuffrida G, Manna R, Mariani P, Pession A, Scopinaro A, Spada M, Spandonaro F, Trifirò G, Carubbi F, Cappellini MD. Acid sphingomyelinase deficiency (ASMD): addressing knowledge gaps in unmet needs and patient journey in Italy-a Delphi consensus. Intern Emerg Med. 2023 Apr;18(3):831-842. doi: 10.1007/s11739-023-03238-3. Epub 2023 Mar 7. PMID: 36882619.

^[ii] 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]

^[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]