A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency

US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment. The total overall study duration will be 5 years. The follow-up period will be a minimum of 1 year to a maximum of 3 years. The enrollment period will be up to 4 years, to allow a minimum of 1 year of follow-up for the last participant enrolled.

Start Date16 Apr 2024

Sponsor / Collaborator

Sanofi

NCT05359276 / RecruitingNot Applicable

Acid Sphingomyelinase Deficiency (ASMD): Data Analysis of Adult and Pediatric Patients on Early Access to Olipudase Alfa in France

Primary Objective:
To describe the lung, spleen and liver outcomes of olipudase alfa
Secondary Objectives:
To describe the patient's characteristics
To describe conditions of olipudase alfa use
To describe safety data related to the use of olipudase alfa
To describe complementary effectiveness outcomes parameters

Start Date10 Jun 2022

Sponsor / Collaborator

Sanofi

JPRN-jRCT2031210279 / CompletedPhase 2/3

An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712

Start Date15 Sep 2021

Sponsor / Collaborator-

100 Clinical Results associated with Olipudase alfa

100 Translational Medicine associated with Olipudase alfa

100 Patents (Medical) associated with Olipudase alfa

Literatures (Medical) associated with Olipudase alfa

Orphanet journal of rare diseases

Real-life impacts of olipudase alfa: The experience of patients and families taking an enzyme replacement therapy for acid sphingomyelinase deficiency

Article

Author: Wiseman, Samantha ; Donnelly, Conan ; Pountney, Jackson ; Crowe, Joslyn ; Raebel, Eva M ; Mathieson, Toni ; Hopkin, Justin

Abstract:

Background:

Acid Sphingomyelinase Deficiency (ASMD) is an ultra-rare autosomal recessive lysosomal storage disorder characterized by intracellular lipid accumulation resulting from reduced function of acid sphingomyelinase. Olipudase alfa, an enzyme replacement therapy, was recently approved in several countries for the treatment of the non-neurologic manifestations of ASMD. Studies demonstrate improvement in organomegaly, pulmonary function and lipid profiles with olipudase alfa, yet little is known about its impact on quality of life (QoL) for patients and caregivers. The purpose of this study is to better understand the real-life impact of ASMD on patients and caregivers and assess how olipudase alfa impacts QoL for pediatric patients and their caregivers.

Methods:

Caregivers of pediatric patients (≤ 18 years of age) with a confirmed diagnosis of ASMD that received olipudase alfa for at least 12 months were recruited in early 2022 through national patient organizations to participate in a global online questionnaire followed by semi-structured interviews. Ten caregivers of patients with ASMD who utilized olipudase alfa as an experimental therapy for pediatric patients participated in the study. Quantitative analysis of the results was undertaken, and qualitative data was analyzed using an inductive thematic approach.

Results:

Ten eligible participants completed questionnaires, and 8 of the 10 went on to participate in structured interviews. Symptom burden of ASMD and impact on symptomatology and quality of life after olipudase alfa use are reported here. Five themes emerged from analysis: (1) ASMD is a systemic disease with a wide array of manifestations that significantly impact QoL; (2) Olipudase alfa was associated with improvements in all non-neurologic manifestations of ASMD; (3) Participants perceived the risk associated with olipudase alfa to be low and the benefits to greatly outweigh any risk or burden; (4) Participants reported an unmet need to treat the neurologic manifestations of the disease despite the benefits of olipudase alfa in the management of non-neurological symptoms; (5) Participants felt all patients with ASMD need access to olipudase alfa based on the life-changing experience they perceived.

Conclusions:

These findings highlight the sustained positive impact olipudase alfa had in many domains that are deemed important to patients and families living with ASMD and outline the extensive unmet need for patients and families living with ASMD.

01 Jul 2023·Journal of molecular medicine (Berlin, Germany)

Acid sphingomyelinase expression is associated with survival in resectable pancreatic ductal adenocarcinoma.

Article

Author: Turner, Kevin M ; Patel, Sameer H ; Gulbins, Erich ; Wilson, Gregory C ; Carpinteiro, Alexander ; Xu, Kui ; Wang, Jiang ; Szabo, Ildiko ; Becker, Katrin Anne ; Ahmad, Syed A

Pancreatic adenocarcinoma (PDAC) is one of the most common cancers worldwide. Unfortunately, the prognosis of PDAC is rather poor, and for instance, in the USA, over 47,000 people die because of pancreatic cancer annually. Here, we demonstrate that high expression of acid sphingomyelinase in PDAC strongly correlates with long-term survival of patients, as revealed by the analysis of two independent data sources. The positive effects of acid sphingomyelinase expression on long-term survival of PDAC patients were independent of patient demographics as well as tumor grade, lymph node involvement, perineural invasion, tumor stage, lymphovascular invasion, and adjuvant therapy. We also demonstrate that genetic deficiency or pharmacological inhibition of the acid sphingomyelinase promotes tumor growth in an orthotopic mouse model of PDAC. This is mirrored by a poorer pathologic response, as defined by the College of American Pathologists (CAP) score for pancreatic cancer, to neoadjuvant therapy of patients co-treated with functional inhibitors of the acid sphingomyelinase, in particular tricyclic antidepressants and selective serotonin reuptake inhibitors, in a retrospective analysis. Our data indicate expression of the acid sphingomyelinase in PDAC as a prognostic marker for tumor progression. They further suggest that the use of functional inhibitors of the acid sphingomyelinase, at least of tricyclic antidepressants and selective serotonin reuptake inhibitors in patients with PDAC, is contra-indicated. Finally, our data also suggest a potential novel treatment of PDAC patients with recombinant acid sphingomyelinase. KEY MESSAGES: Pancreatic ductal adenocarcinoma (PDAC) is a common tumor with poor prognosis. Expression of acid sphingomyelinase (ASM) determines outcome of PDAC. Genetic deficiency or pharmacologic inhibition of ASM promotes tumor growth in a mouse model. Inhibition of ASM during neoadjuvant treatment for PDAC correlates with worse pathology. ASM expression is a prognostic marker and potential target in PDAC.

01 May 2023·Advances in therapy

Healthcare Service Use Patterns Among Patients with Acid Sphingomyelinase Deficiency Type B: A Retrospective US Claims Analysis

Article

Author: Fournier, Marie ; Ganz, Michael L ; Petruski-Ivleva, Natalia ; Pulikottil-Jacob, Ruth

INTRODUCTION:

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disease. Patients with ASMD type B experience multiple morbidities, potentially leading to early mortality. Before the 2022 approval of olipudase alfa for non-neuronopathic ASMD manifestations, only symptom management was offered. Data on healthcare services used by patients with ASMD type B are limited. This analysis used medical claims data to evaluate real-world healthcare service use by patients with ASMD type B in the United States of America (USA).

METHODS:

The IQVIA Open Claims patient-level database (2010-2019) was cross-examined. Two patient cohorts were identified: the primary analysis cohort, which included patients with at least two claims associated with ASMD type B (ICD-10 code E75.241) and more total claims with ASMD type B than any other ASMD types, and the sensitivity analysis cohort, which included patients with a high probability of having ASMD type B identified using a validated machine-learning algorithm. Claims for ASMD-associated healthcare services were recorded, including outpatient visits, emergency department (ED) visits, and inpatient hospitalizations.

RESULTS:

The primary analysis cohort included 47 patients; a further 59 patients made up the sensitivity analysis cohort. Patient characteristics and healthcare service use were similar in both cohorts and were consistent with established characteristics of ASMD type B. Overall, 70% of the primary analysis cohort from this study were aged < 18 years, and the liver, spleen, and lungs were the most frequently affected organs. Cognitive, developmental, and/or emotional problems and respiratory/lung disorders caused most outpatient visits; respiratory/lung disorders accounted for most ED visits and hospitalizations.

CONCLUSION:

This retrospective analysis of medical claims data identified patients with ASMD type B who had characteristics typical of this condition. A machine-learning algorithm detected further cases with a high probability of having ASMD type B. High use of ASMD-related healthcare services and medications was observed in both cohorts.

News (Medical) associated with Olipudase alfa

20 Mar 2024

·www.fiercepharma.com

#FierceMadness is back: Get voting in the 2024 drug name tournament

This year we’re taking the branded drug names that were officially approved by the FDA in 2022 and 2023 - now it's your turn to vote for your winner. LAUNCHED: Wednesday, March 20, 7:00 a.m. ET It’s March, so it’s time for some #FierceMadness. Last year, we channeled the NCAA tournament for the best pharma ad campaigns, but, this year, we’re looking for the best of the best in drug names. Here’s how it works. We’ve chosen 64 of the most distinctive drug names to come out of the FDA’s 2022 and 2023 crop of new approvals. Whittling down to 64 from the 92 drug names available was our staff challenge, but now it’s up to you, readers, to deliberate and vote down to the final winner. Remember, this isn’t about the drugs themselves—neither how well they may work (or not) nor any controversies in how they may have been approved. Our goal is to assess their marketing suitability, considering how well they perform in their respective field and against competitors. Ready to play? Check out the bracket here (PDF) and see the full Madness 64 list below. Then vote is via our poll here. You can vote from now until Friday, March 22, at 6 p.m. ET. Check back here Monday, March 25, for your results, and the chance to vote in the next round. After that, we’ll be back with a new round each Wednesday and Monday through Monday, April 8, when you can vote for your championship winner. Any remote robo-voting will be noted, and those votes will be discarded. Please play fair. Don’t forget to tell us why you picked what you picked in the voting poll comments section—and what you think each drug name really sounds like. We’ll include the funniest and most out-of-the-box comments in our recaps every round, so make 'em good! New this year: Our sister publication Fierce Biotech is also running its own Madness tournament for the best of the best in biotech company names. Check out and vote in its championship here. Good luck, and happy Madness! #FierceMadness 2024: The full bracket of 64 Jesduvroq vs. Lamzede Jesduvroq Maker: GSK Indication: Anemia due to chronic kidney disease Sounds like: When you hit your finger with a hammer, but it’s in front of your children so you’re trying not to swear Lamzede Maker: Chiesi Indication: Non-central nervous system manifestations of alpha-mannosidosis Sounds like: A vegan meal Pluvicto vs. Lunsumio Pluvicto Maker: Novartis Indication: Prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer following other therapies Sounds like: A new planet Lunsumio Maker: Roche Indication: Relapsed or refractory follicular lymphoma, a type of non-Hodgkin lymphoma Sounds like: A Japanese wrestler Briumvi vs. Camzyos Briumvi Maker: TG Therapeutics Indication: Relapsing forms of multiple sclerosis Sounds like: A sixth-generation cheese Camzyos Maker: Bristol Myers Squibb Indication: Obstructive hypertrophic cardiomyopathy Sounds like: A Spanish holiday resort Voquezna vs. Mounjaro Voquezna Maker: Phathom Pharmaceuticals Indication: Helicobacter pylori infection Sounds like: A sports drink with a kick Mounjaro Maker: Eli Lilly Indication: Lowering blood sugar levels in Type 2 diabetes Sounds like: A legendary medieval sword fighter Vtama vs. Amvuttra Vtama Maker: Dermavant Indication: Plaque psoriasis Sounds like: A mouthful Amvuttra Maker: Alnylam Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A pope’s gift Xenpozyme vs. Daxxify Xenpozyme Maker: Sanofi Indication: Acid sphingomyelinase deficiency Sounds like: An alien protein Daxxify Maker: Revance Indication: The treatment of cervical dystonia and for the temporary improvement of frown lines Sounds like: A ray gun to make you sassy Fruzaqla vs. Sotyktu Fruzaqla Maker: Takeda Pharmaceuticals Indication: Refractory, metastatic colorectal cancer Sounds like: A European swear word Sotyktu Maker: Bristol Myers Squibb Indication: Moderate to severe plaque psoriasis Sounds like: Gesundheit! Terlivaz vs. Imjudo Terlivaz Maker: Mallinckrodt Pharmaceuticals Indication: Improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function Sounds like: A Hungarian dance Imjudo Maker: AstraZeneca Indication: Unresectable hepatocellular carcinoma Sounds like: A self-confident martial artist Elahere vs. Vabysmo Elahere Maker: ImmunoGen Indication: Recurrent ovarian cancer that is resistant to platinum therapy Sounds like: An inebriated attempt at Hawaiian Vabysmo Maker: Roche Indication: Neovascular (wet) aged-related macular degeneration and diabetic macular edema Sounds like: A new way of saying fantastic Pyrukynd vs. Rystiggo Pyrukynd Maker: Agios Indication: Hemolytic anemia in pyruvate kinase deficiency Sounds like: A drunk Scrabble game Rystiggo Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive Sounds like: An Italian village Beyfortus vs. Xdemvy Beyfortus Maker: AstraZeneca Indication: To prevent respiratory syncytial virus lower respiratory tract disease Sounds like: A medieval stronghold Xdemvy Maker: Tarsus Pharmaceuticals Indication: Demodex blepharitis Sounds like: Elon Musk’s drunk attempt at renaming his business Tzield vs. Rezlidhia Tzield Maker: Provention Bio Indication: Delay the onset of stage 3 Type 1 diabetes Sounds like: An electronic barrier Rezlidhia Maker: Rigel Pharmaceuticals Indication: Relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation Sounds like: A new Disney princess Krazati vs. Leqembi Krazati Maker: Mirati Therapeutics Indication: KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer in adults who have received at least one prior systemic therapy Sounds like: A crazy party Leqembi Maker: Eisai Indication: Alzheimer’s disease Sounds like: An inspirational French aphorism Cibinqo vs. Brenzavvy Cibinqo Maker: Pfizer Indication: Refractory, moderate to severe atopic dermatitis Sounds like: A game of cheeky bingo Brenzavvy Maker: TheracosBio Indication: Glycemic control in adults with Type 2 diabetes mellitus as an adjunct to diet and exercise Sounds like: Heard in a copy of "Pirates of the Caribbean" Jaypirca vs. Skyclarys Jaypirca Maker: Eli Lilly Indication: Relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitor Sounds like: A strong coffee Skyclarys Maker: Biogen Indication: Friedreich’s ataxia Sounds like: A high-rise building Zavzpret vs. Paxlovid Zavzpret Maker: Pfizer Indication: Migraine treatment Sounds like: A Turkish fast-food restaurant Paxlovid Maker: Pfizer Indication: Mild to moderate COVID-19 in adults at high risk for progression to severe COVID-19 Sounds like: A bizarre Pac-Man sequel Inpefa vs. Columvi Inpefa Maker: Lexicon Pharmaceuticals Indication: Heart failure Sounds like: An anti-fascist offshoot group Columvi Maker: Roche Indication: Diffuse large B-cell lymphoma, not otherwise specified, or large B-cell lymphoma arising from follicular lymphoma after two or more lines of systemic therapy Sounds like: An ancient Roman detective Daybue vs. Zynyz Daybue Maker: Acadia Indication: Rett syndrome Sounds like: How my five-year-old insists debut is spelled Zynyz Maker: Incyte Indication: Metastatic or recurrent locally advanced Merkel cell carcinoma Sounds like: Drug naming companies may be running out of ideas Rezzayo vs. Qalsody Rezzayo Maker: Melinta Therapeutics Indication: Candidemia and invasive candidiasis Sounds like: A phrase used when you’ve had a great nap in the day Qalsody Maker: Biogen Indication: Amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation Sounds like: From the makers of “Better Call Saul” comes “better Qalsody” Elfabrio vs. Veozah Elfabrio Maker: Chiesi Indication: Fabry disease Sounds like: The lost Spanish Mario brother Veozah Maker: Astellas Indication: Moderate to severe hot flashes caused by menopause Sounds like: A Broadway play Epkinly vs. Zurzuvae Epkinly Makers: AbbVie/Genmab Indication: Relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma after two or more lines of systemic therapy Sounds like: Someone who watches too many streaming episodes Zurzuvae Makers: Sage/Biogen Indication: Postpartum depression Sounds like: A mouthful Izervay vs. Talvey Izervay Maker: Iveric Bio Indication: Geographic atrophy secondary to age-related macular degeneration Sounds like: A new app for scanning the land Talvey Maker: Johnson & Johnson Indication: Relapsed or refractory multiple myeloma who have received at least four prior therapies Sounds like: A little too close to talc… Elrexfio vs. Veopoz Elrexfio Maker: Pfizer Indication: Relapsed or refractory multiple myeloma who have received at least four prior lines of therapy Sounds like: A new dinosaur found in Mexico Veopoz Maker: Regeneron Pharmaceuticals Indication: Patients 1 year old and older with CD55-deficient protein-losing enteropathy Sounds like: Double-plus good! Zilbrysq vs. Ojjaara Zilbrysq Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive Sounds like: Absolute zero Ojjaara Maker: GSK Indication: Intermediate or high-risk myelofibrosis in adults with anemia Sounds like: “What’s my name?” Exxua vs. Pombiliti Exxua Maker: Fabre-Kramer Pharmaceuticals Indication: Major depressive disorder Sounds like: Someone who’s always leaving Pombiliti Maker: Amicus Indication: Late-onset Pompe disease Sounds like: The ability to treat Pompe disease (this feels a little on the nose) Rivfloza vs. Velsipity Rivfloza Maker: Novo Nordisk Indication: Urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function Sounds like: An exuberant DJ Velsipity Maker: Pfizer Indication: Moderately to severely active ulcerative colitis in adults Sounds like: A deodorant brand you wouldn’t want to buy Bimzelx vs. Omvoh Bimzelx Maker: UCB Indication: Moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy Sounds like: Throwing away unwanted antacids Omvoh Maker: Eli Lilly Indication: Ulcerative colitis Sounds like: A new age prayer Loqtorzi vs. Defencath Loqtorzi Maker: Coherus Biosciences Indication: Recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies Sounds like: How you close the door in a magical kingdom Defencath Maker: CorMedix Indication: Reduce the incidence of catheter-related bloodstream infections in adults with kidney failure receiving chronic hemodialysis Sounds like: A Dungeons and Dragons character Augtyro vs. Ryzneuta Augtyro Maker: Bristol Myers Squibb Indication: ROS1-positive non-small cell lung cancer Sounds like: A virtual reality game where you’re the bad guy Ryzneuta Maker: Acrotech Biopharma Indication: Neutropenia Sounds like: Getting rid of someone’s charisma Truqap vs. Ogsiveo Truqap Maker: AstraZeneca Indication: Breast cancer that meets certain disease criteria Sounds like: A limit on truth Ogsiveo Maker: SpringWorks Indication: Progressing desmoid tumors who require systemic treatment Sounds like: Oh, give us a go! Quviviq vs. Ztalmy Quviviq Maker: Idorsia Indication: Insomnia Sounds like: A producer asking a biotech founder turned politician to get ready for his TV appearance Ztalmy Maker: Marinus Indication: Seizures in cyclin-dependent kinase-like 5 deficiency disorder Sounds like: A nefarious Yevgeny Zamyatin character Opdualag vs. Wainua Opdualag Maker: Bristol Myers Squibb Indication: Unresectable or metastatic melanoma Sounds like: A Russian prison Wainua Maker: AstraZeneca Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A Premier League footballer

27 Oct 2023

·www.globenewswire.com

Press Release: Sanofi Enters Next Chapter of Play to Win Strategy

Sanofi Enters Next Chapter of Play to Win Strategy Increases investments in its pipeline to fully realize long-term growth potential, bolstered by successful launches and R&D progress.Launches strategic cost initiatives, with most of the savings to be reallocated to fund innovation and growth drivers.Announces intention to separate the Consumer Healthcare Business at the earliest in Q4 2024 via the creation of a publicly listed entity headquartered in France.Reiterates capital allocation policy and 2023 Business EPS growth target, and provides preliminary 2024 and 2025 outlook. Paris, October 27, 2023. Sanofi today provided a comprehensive update regarding its Play to Win strategy. This strategy continues to focus on the critical goals of executing transformative medicine and vaccines launches, driving agile and efficient resource deployment and enhancing R&D productivity. To that end, Sanofi announced plans to increase its R&D investments to fully realize its pipeline potential, drive long-term growth and enhance shareholder value. The Company also announced its intention to separate its Consumer Healthcare Business enabling greater management focus and resource allocation to the needs of the Biopharma business, where value-creating opportunities and longer-term operational levers have been identified to support the accelerated R&D investments. Sanofi reiterates its financial guidance for 2023 and provides a preliminary outlook for 2024 and 2025 that reflects this new chapter of its strategy. Paul HudsonChief Executive Officer, Sanofi “We have made tremendous progress on our Play to Win strategy by bringing new and transformative products to market and building an industry-leading immunology pipeline, evidenced by our recent, strong flow of positive R&D data readouts. In this new chapter of our strategy, we are deepening our investment in R&D, taking steps toward becoming a pure play biopharma company, and further optimizing our cost structure. This will help us accelerate innovation and strengthen our growth drivers, while ensuring long-term profitability and enhancing shareholder value. We are excited to build on the success of our strategy and confident in the long-term value our investments will generate for all Sanofi stakeholders.” Play to Win Achievements Since 2019Sanofi’s multi-year Play to Win strategy, which focused on growth, innovation, and efficiency, has positioned Sanofi for long-term success and achieved significant progress since 2019, including: Achieving 13 consecutive quarters of growthSuccessfully scaling Dupixent® to be among the world’s leading medicines with Q3 2023 annualized sales nearing €11 billion Building a high-growth Vaccines business, which delivered mid- to high-single digit sales growth since 2018, with at least five new programs expected to enter Phase 3 by 2025 Successfully launching six new molecular entities in the last two years (Xenpozyme®, Nexviazyme®, Enjaymo®, ALTUVIIIO®, TZIELD® and Beyfortus®)Developing an industry-leading immunology and strong neuro-inflammation pipeline, with currently 7 assets in mid- to late-stage development, each with a peak sales potential of at least €2 to €5 billion (itepekimab, tolebrutinib, amlitelimab, frexalimab, rilzabrutinib, SAR443765 (IL13/TSLP), SAR441566 (oral TNFi))Executing a €2.7 billion cost savings plan to reinvest in the Company’s growth driversInvesting in over 25 value-creating business development and M&A deals to further strengthen Sanofi’s pipelineStreamlining Consumer Healthcare product portfolio to focus on priority brandsSignificantly improving BOI4 and free cash flow Strengthening the Pipeline through Increased R&D InvestmentSanofi’s strategic mission is to transform the practice of medicine through breakthrough science that improves people’s lives. To that end, the Company is accelerating its R&D investments to deliver high-value innovation. These further investments will strengthen and sustain the Company’s long-term trajectory of profitable growth by fueling existing or new clinical development. As previously announced, Sanofi will host an R&D Day for investors in New York City on December 7, 2023, where the Company will provide further insights into its pipeline and new growth avenues. The meeting will include presentations from Sanofi’s new Head of R&D, Houman Ashrafian, as well as key members from the research and development teams. Houman AshrafianHead of Research and Development, Sanofi “This is an exciting and important moment for Sanofi’s R&D journey, as we double down on our investments from a position of strength in order to unlock our pipeline’s full potential. Our investments will follow Sanofi’s strategy of transforming medicine through breakthrough science, enabling greater access to medicines and vaccines that benefit the patients and communities we serve. We are committed to investing in innovation with discipline, and we will reallocate resources, further prioritizing first and or best in class medicines to help fund our future growth. We look forward to discussing our plan further with investors at our upcoming R&D Day, where we will dig deeper into the compelling science underpinning this opportunity.” Strategic Cost InitiativesSanofi is announcing new steps to further its ongoing effort to improve its cost structure, launching efficiency initiatives across the Biopharma business that will free operational resources to support the accelerated R&D investments and unlock value-creation opportunities. This will include prioritizing its investments in R&D and modernizing its approach to commercial delivery. Sanofi is targeting savings of a total of up to €2 billion from 2024 to the end of 2025, of which most will be reallocated to fund innovation and growth drivers. Intention to Separate the Consumer Healthcare BusinessFollowing the announcement in December 2019 of the creation of a standalone Global Business Unit, Sanofi’s Consumer Healthcare (“CHC”) has grown into a leading consumer healthcare platform with a presence in 150 countries and over 11,000 employees, dedicated resources in R&D, manufacturing, information technology as well as its distinct sustainability roadmap. Sanofi announces its intention to separate the CHC Business as it increases its focus on innovative medicines and vaccines. The intended separation will seek to create two entities, each better equipped to pursue its own business strategy, resourcing and capital allocation and enabling each to focus on long-term growth in its respective markets. Sanofi believes that the separation will unlock further opportunities for CHC to leverage its portfolio of leading brands and continue to drive growth and shareholder value. Julie van OngevalleExecutive Vice President, Consumer Healthcare Sanofi “We have achieved significant milestones at Sanofi Consumer Healthcare since we embarked on our journey to become a standalone Global Business Unit within Sanofi. We have adapted our business to the evolving needs of the Consumer Healthcare sector, offered a streamlined yet diverse brand portfolio of science-based products and solutions and achieved the B Corp accreditation as the first large consumer healthcare business in North America. The CHC teams and I look forward to becoming a standalone global leader in the attractive and growing consumer health market, with increased agility and flexibility to grow our portfolio of brands and further address our customers’ needs across categories.” Sanofi is reviewing potential separation scenarios, but believes that the most likely path would be through a capital markets transaction, by creating a listed entity headquartered in France. The timing is driven by the desire to maximize value creation and reward Sanofi shareholders. Subject to market conditions, the separation could be achieved at the earliest in Q4 2024, following consultation with social partners. Further details will be provided at a later date. 2023 Guidance and Preliminary 2024 and 2025 Outlook1Sanofi reiterates its 2023 guidance of mid-single digit2 Business EPS3 growth at constant exchange rates. As a result of changes to global tax regulations, Sanofi’s effective tax rate is expected to increase from 19% in 2023 to 21% in 2024. Due to the increased R&D investment, Sanofi expects 2024 Business EPS to remain roughly stable to 2023 levels excluding the impact of the expected tax rate change, and therefore decline low-single digits including the higher expected tax rate. In 2025, Sanofi expects a strong rebound in business EPS growth, driven by continued sales growth supported by its leading franchises, the full benefit from planned efficiency initiatives, and its expectation of relatively stable R&D expenses year on year. Given Sanofi’s decision to support the full realization of its pipeline’s long-term potential, its continued investment around the new launches, as well as pricing headwinds in General Medicines, the Company will no longer target a 32% BOI4 margin for 2025 while maintaining a focus on long-term profitability. The company reiterates its goal to generate over €22 billion in sales in immunology, and over €10 billion in sales in vaccines by 20301. Capital Allocation ReaffirmedThe Company’s capital allocation policy remains unchanged. Sanofi expects to maintain a gradually growing dividend, consistent with its historical dividend policy. Q3 2023 Earnings Call & Upcoming R&D Day InformationThird quarter 2023 results and the announcements above will be discussed by management during a conference call with the financial community today. The presentation will be followed by a Q&A session. The link to the audio webcast and the corresponding presentation can be found on the Investors Relations section of Sanofi corporate website: www.sanofi.com/investors. A replay will be available shortly after the live webcast.Sanofi will host an R&D Day for investors and analysts in New York City on December 7, 2023. A live webcast will be available for those unable to attend in person. Event registration details will be forthcoming.About SanofiWe are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Sanofi Media Relations Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.comSally Bain | + 1 617 834 6026 | sally.bain@sanofi.comEvan Berland | +1 215 432 0234 | evan.berland@sanofi.comNicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.comVictor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com Sanofi Investor RelationsEva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.comArnaud Delépine | + 33 06 73 69 36 93 | arnaud.delepine@sanofi.comCorentine Driancourt | + 33 06 40 56 92 | corentine.driancourt@sanofi.comFelix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.comTarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.comNathalie Pham | + 33 07 85 93 30 17 | nathalie.pham@sanofi.com Sanofi Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, business transformations, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans”, “potential”, “outlook”, “guidance” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete capital markets or other transactions and/or obtain regulatory clearances, risks associated with developing standalone businesses, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation,  trends in exchange rates and prevailing interest rates, volatile economic and capital market conditions, cost containment initiatives and subsequent changes thereto, and the impact that pandemics, political disruption or armed conflicts or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2022. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. 1 2024 and 2025 outlook excludes unforeseen major adverse events and assumes constant exchange rates.2 2022 business EPS was €8.26.3 In order to facilitate an understanding of operational performance, Sanofi comments on the business net income statement. Business net income is a non-IFRS financial measure (definition in Appendix 7 of the Q3 press release dated October 27, 2023). The consolidated income statement for Q3 2023 is provided in Appendix 3 of the Q3 press release dated October 27, 2023 and a reconciliation of reported IFRS net income to business net income is set forth in Appendix 4 of the Q3 press release dated October 27, 20234 See Q3 press releases Appendix 7 for definitions of financial indicators Attachment Press_Release

Vaccine

02 May 2023

·www.businesswire.com

Recombinant Therapeutic Antibodies, Proteins, Biosimilars & Other Biologics Sales Analysis Report 2023: 71 Biologic Therapeutics Reached Blockbuster Status with 2022 Sales Exceeding $ 1 Billion - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "2022 Sales of Recombinant Therapeutic Antibodies, Proteins, Biosimilars & Other Biologics" report has been added to ResearchAndMarkets.com's offering. This report provides a compilation of the sales data of originator recombinant therapeutic proteins and antibodies in the calendar year 2022. Sales data were obtained from company publications and refer to branded products. All sales data were converted from their original, reported currency into US$ using official currency exchange rates as of February 01, 2021. Sales figures represent the sum of revenues in all territories where the products are marketed. Growth rates mostly are presented as reported (in some cases as constant exchange rates). Publicly available 2022 sales data of biosimilar antibodies and proteins are included in the report provided from the companies. This report also includes sales data of new biologic modalities such as gene and cell therapy and RNA (antisense, siRNA). Due to biosimilar erosion of sales of established therapeutic proteins such as erythropoietins, interferon, G-CSF, human growth hormone and FSH, these products are summarized in a new group of established therapeutic proteins. Sales and growth rates of cancer antibodies were analyzed by subgroup, especially for the strongly growing class of PD-1 & PD-L1 antibodies. Development of sales of the growing drug modality of antibody-drug conjugates are also presented. A total of 71 biologic therapeutics reached blockbuster status with 2022 sales exceeding US$ 1 bln. For the first time, sales of 22 new antibody and protein products for the year 2022 were included. Antibody sales represented more than 80% of total biologics sales in 2022. In addition, commercial information about sales of selected biosimilars of recombinant antibodies and proteins is provided. Sales data are presented for each product within the respective class of biologics. The data were analyzed to establish a ranking list of blockbuster biologics with 2022 sales higher than US$ 1 bln. Another ranking list was prepared for the TOP 30 companies according to biologics sales in 2022. Newly included innovator biologics for which sales are reported for the first time are Aidexi Anniko Cejemly Daxxify Elahere Enjaymo Enweida Hansizhuang Idefirix Inmazeb Kimmtrak Lunsumio Opdualag Rolvedon Saphnelo Skytrofa Susvimo Telitacicept Tezspire Vabysmo Vyvgart Xenpozyme Key Topics Covered: Biologics Sales per Class of Branded Originator Products Sales of Established Therapeutic Proteins 2012 - 2022 Sales and Growth Rates of Cancer Antibodies by Subgroup 2012 - 2022 Sales and Growth Rates of Other Anti-Inflammatory Antibodies by Therapeutic Area 2012 - 2022 Sales of Antibody-Drug Conjugates by Product 2012 - 2022 Published Biosimilar Sales 2022 by Company Published 2022 Sales of Other Biologics Based on DNA, RNA & Cells TOP 30 Company Ranking List for Sales of Innovator Biologics 2022 Blockbuster Biologics in 2022 (by generic name) Biologic Product Sales 2022 1. Cancer Antibodies 1.1 PD-1 & PD-L1 Immuno-Oncology Antibodies 1.2 Other Immuno-Oncology Antibodies 1.3. Cancer Antibodies 1.3.1 Her2 1.3.2 VEGF & VEGF-R 1.3.3 EGF-R 1.3.4 CD20 1.4 Other Cancer Antibodies 2. Other Anti-Inflammatory Antibodies 2.1 Dermatology 2.2 Rheumatoid & Autoimmune 2.3 Neuro-Inflammation (multiple sclerosis, myasthenia gravis) 2.4 Pulmonary & Respiratory 2.5 Gastrointestinal 3. Anti-TNF Antibodies 4. Cardiometabolic Antibodies 5. Ophthalmic Antibodies 6. Insulin and Insulin Analogs 7. Recombinant Coagulation Factors 8. Established Therapeutic Proteins 8.1 Interferon beta (IFN-b) 8.2 Erythropoiesis Stimulating Agent (ESA) 8.3 human Growth Hormone (hGH) 8.4 Granulocyte Colony Stimulating Factor (G-CSF) 8.5 Follicle Stimulating Hormone (FSH) 9. Anti-Infective Antibodies 10. Enzyme Replacement Therapy (ERT) 11. Migraine & Neurodegenerative Disease Antibodies 12. Other Proteins 12.1 Cardiometabolic Proteins 12.2 Cancer Proteins 12.3 Various Proteins 13. Selected Biosimilar Antibodies & Proteins 14. Selected Other Biologics 14.1 DNA 14.2 Cell Therapy 14.3 RNA For more information about this report visit https://www.researchandmarkets.com/r/h0vhl1 About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

100 Deals associated with Olipudase alfa

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KEGG	Wiki	ATC	Drug Bank
D10820	-	-	DB12835

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Acid sphingomyelinase deficiency	JP	Sanofi KK	28 Mar 2022

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Niemann-Pick Disease, Type A	Phase 3	US	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	JP	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	AR	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	AU	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	BE	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	BR	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	BG	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	CL	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	FR	Genzyme Corp.	18 Dec 2015
Niemann-Pick Disease, Type A	Phase 3	DE	Genzyme Corp.	18 Dec 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EHA2023	Not Applicable	Acid sphingomyelinase deficiency	-	Olipudase alfa	myeaiorbqb(cdjhuojxms) = gtbuzfgdfj jzkvkgammr (bohnvkvakd, 7.3) View more	-	08 Jun 2023
SSIEM2022	Not Applicable	Acid sphingomyelinase deficiency	60	Olipudase alfa	hnoibkifsm(izmmvnjhje) = rrpyswrhmk zdxfvybciu (cccbyjukkz, ±15.6)	-	30 Aug 2022
NCT02004691 (ASCEND, EHA2022)	Phase 2/3	Acid sphingomyelinase deficiency	36	Olipudase alfa	klaignsvxq(mjkwwqiiab) = ndifusfxzk kutaemkitg (xgshzwxgsp, 6.2) View more	-	10 Jun 2022
NCT02004691 (ASCEND, Pubmed \| Genet Med) Manual	Phase 2/3	Acid sphingomyelinase deficiency	36	Olipudase alfa	hxhynmgnmd(gvmbjuzahq) = pfznbwnbon ijykavalrj (rrjmcvygph ) View more	Positive	26 Apr 2022
				Placebo	hxhynmgnmd(gvmbjuzahq) = bmwtlonwet ijykavalrj (rrjmcvygph ) View more
NCT02004704 (GlobeNewswire) Manual	Phase 3	Acid sphingomyelinase deficiency	25	Olipudase alfa (Adult)	mtsjucedll(vxpmnbuyhf) = 7 events were observed in four patients: one patient had an anaphylactic reaction but restarted treatment after tailored desensitization and reached the target dose; one patient had two events of transient alanine aminotransferase elevation; one patient had urticaria and rash; and one patient had two hypersensitivity reactions. iznwbvswof (ymebwuwclr ) View more	Positive	09 Feb 2022
				Olipudase alfa (Pediatric)
EHA2021	Not Applicable	Acid sphingomyelinase deficiency	-	Olipudase alfa	rvyckgyloo(zdqvfocpmn) = snyohrdcsj fplsaswkwu (jpawpyzfup ) View more	-	09 Jun 2021
				Placebo	rvyckgyloo(zdqvfocpmn) = zxoslodybf fplsaswkwu (jpawpyzfup )
NCT02292654 (CTgov)	Phase 1/2	Acid sphingomyelinase deficiency \| Niemann-Pick Disease, Type A	20	Olipudase Alfa (Olipudase Alfa: Adolescent Cohort)	elwqfrywhc(jvkglfywrf) = lukmdqiani fhfewtjigl (pfzwgfjlmq, imljbjlarb - slglipdsxu) View more	-	01 Feb 2021
				Olipudase Alfa (Olipudase Alfa: Child Cohort)	elwqfrywhc(jvkglfywrf) = pqodsmzmth fhfewtjigl (pfzwgfjlmq, awfonkedbn - wdxqkhrkhq) View more
ASCEND (Biospace) Manual	Not Applicable	Acid sphingomyelinase deficiency	36	Olipudase alfa	ozzqxjluax(ogbcjbdsqy) = azyuoxvkom sqbxktfsmk (ftluydphgw ) View more	Positive	30 Jan 2020
				Placebo	ozzqxjluax(ogbcjbdsqy) = jnspgcecsw sqbxktfsmk (ftluydphgw ) View more
NCT02004704 (Pubmed) Manual	Phase 2	Acid sphingomyelinase deficiency	5	Olipudase alfa	vpzbkmkmnh(bffyvszpzt) = The majority of adverse events were mild and included headache, nausea, and abdominal pain xwzuhombvo (pwrzblwybe )	Positive	01 Sep 2018
NCT01722526 (Pubmed \| Mol Genet Metab)	Phase 1	Acid sphingomyelinase deficiency	5	Olipudase alfa	qsezuozlat(arnkqzzahh) = rrqtfqzsvv cqhooacely (xfaihgimbi ) View more	-	30 Sep 2015

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