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29 Diseases |  |
3 Trials |
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3 Trials |  |
151 News |  |
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- | Xenpozyme (olipudase alfa) / Sanofi
Journal: Unexplained splenomegaly as a diagnostic marker for a rare but severe disease with an innovative and highly effective new treatment option: A case report. (Pubmed Central) - Oct 11, 2024
Acid Sphingomyelinase Deficiency (ASMD) is a lysosomal storage disorder that can lead to severe complications if not promptly treated. This case aims to highlight the critical importance of early awareness of ASMD and to introduce, for the first time in the literature, a new and highly effective treatment option for children.
- | Xenpozyme (olipudase alfa) / Sanofi
Journal: On the role of excipients in biopharmaceuticals manufacture: Modelling-guided formulation identifies the protective effect of arginine hydrochloride excipient on spray-dried Olipudase alfa recombinant protein. (Pubmed Central) - Aug 24, 2024
The Arg-HCl effect was explained by the calculated thermal stability and structural order of the protein wherein Arg-HCl acted as a crowding agent to suppress protein aggregation and promote stabilization of Olipudase post-spray-drying. Therefore, by rational selection of appropriate excipients, our experimental and modelling dataset confirms spray-drying is a promising technology for the manufacture of Olipudase and demonstrates the potential to accelerate development of continuous manufacturing of parenteral biopharmaceuticals.
- Xenpozyme (olipudase alfa) / Sanofi
LONG-TERM IMPACT OF OLIPUDASE ALFA TREATMENT IN CHILDREN WITH CHRONIC ACID SPHINGOMYELINASE DEFICIENCY: FINAL RESULTS OF THE ASCEND-PEDS TRIAL (Poster Room | Level 0) - Aug 21, 2024 - Abstract #SSIEM2024SSIEM_1105;
P1/2, P2
Therefore, by rational selection of appropriate excipients, our experimental and modelling dataset confirms spray-drying is a promising technology for the manufacture of Olipudase and demonstrates the potential to accelerate development of continuous manufacturing of parenteral biopharmaceuticals. Children with ASMD experienced significant improvements in hepatosplenomegaly and lung, lipid, and growth outcomes with ?4Y of olipudase alfa treatment.
- | Xenpozyme (olipudase alfa) / Sanofi
Journal: Desensitization of olipudase alfa-induced anaphylaxis in a child with chronic neurovisceral acid sphingomyelinase deficiency. (Pubmed Central) - Jul 31, 2024
We report a case of anaphylaxis to olipudase alfa in a child affected by chronic neurovisceral Acid sphingomyelinase deficiency (ASMD) and describe a 7-step desensitization procedure. This procedure, with the total administered dose of 0.03
- Xenpozyme (olipudase alfa) / Sanofi
Efficacy and Safety data for olipudase alfa: the first and only disease specific treatment for ASMD type A/B or type B (non-CNS manifestations) (Camellia Auditorium | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_1013;
This procedure, with the total administered dose of 0.03 Sponsored by Sanofi
- Xenpozyme (olipudase alfa) / Sanofi
ENZYME REPLACEMENT THERAPY FOR ACID SPHINGOMYELINASE DEFICIENCY - A REAL-WORLD EXPERIENCE OF A SINGLE CENTER IN BRAZIL (Poster Room | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_932;
The current disease-specific treatment is enzyme replacement therapy (ERT) with a recombinant human acid sphingomyelinase, olipudase alfa... ERT was safe, improved liver transaminases and hematological parameters, attenuated facial features, and markedly reduced hepatosplenomegaly.
- Xenpozyme (olipudase alfa) / Sanofi
EARLY RESULTS OF OLIPUDSE ALPHA TREATMENT IN A CASE DIAGNOSED WITH ACID SPHINGOMYELINASE DEFICIENCY (Poster Room | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_931;
Early initiation of ERT, a specific treatment for ASMD deficiency, in NP type B and A/B cases will cause changes in the natural course of the disease. Palavras-chave : ACID SPHINGOMYELINASE DEFICIENCY, OLIPUDASE ALPHA
- Xenpozyme (olipudase alfa) / Sanofi
A REAL-WORLD EFFECT OF OLIPUDASE ALFA ON ASMD CHILDREN OF VARIABLE PRESENTATIONS (Poster Room | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_573;
The effect on weight was variable between the three patients (keeping in mind that all patients had decrease in organ volumes which is part of their weight). Long term follow up is needed to verify the impact of olipudase alfa on other manifestations in type A/B patients.
- Xenpozyme (olipudase alfa) / Sanofi
ENZYME REPLACEMENT THERAPY FOR CHILDREN WITH ACID SPHINGOMYELINASE DEFICIENCY: A 3-YEAR REAL-WORLD EXPERIENCE AT A SINGLE CENTER IN TAIWAN (Poster Room | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_561;
P=N/A
The 3Y results in our study provide real-world experience indicating that olipudase alfa is safe and effective in improving clinical outcomes for pediatric chronic ASMD patients. Monitoring liver and spleen volumes, as well as liver stiffness, using abdominal ultrasonography and shear wave elastography, represents a noninvasive procedure that can monitor treatment efficacy during enzyme replacement therapy.
- Xenpozyme (olipudase alfa) / Sanofi
LONG-TERM IMPACT OF OLIPUDASE ALFA TREATMENT IN CHILDREN WITH CHRONIC ACID SPHINGOMYELINASE DEFICIENCY: FINAL RESULTS OF THE ASCEND-PEDS TRIAL (Poster Room | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_438;
P1/2, P2
Monitoring liver and spleen volumes, as well as liver stiffness, using abdominal ultrasonography and shear wave elastography, represents a noninvasive procedure that can monitor treatment efficacy during enzyme replacement therapy. Children with ASMD experienced significant improvements in hepatosplenomegaly and lung, lipid, and growth outcomes with ?4Y of olipudase alfa treatment.
- Xenpozyme (olipudase alfa) / Sanofi
CASE REPORT: TREATMENT WITH OLIPUDASE ALFA AMONG US ASMD PATIENTS WITH SIGNIFICANT NEUROLOGICAL SYMPTOMS (Poster Room | Level 0) - Jul 30, 2024 - Abstract #SSIEM2024SSIEM_311;
Children with ASMD experienced significant improvements in hepatosplenomegaly and lung, lipid, and growth outcomes with ?4Y of olipudase alfa treatment. By leveraging recruitment through WylderNation Foundation and PicnicHealth medical record data, this case study provides insight into the clinical outcomes of ERT in ASMD patients with more severe phenotypes.
- Xenpozyme (olipudase alfa) / Sanofi
ACID SPHINGOMYELINASE DEFICIENCY - INITIAL OUTCOMES OF TREATMENT WITH ENZYME REPLACEMENT THERAPY IN INDIAN CHILDREN (Camellia Auditorium | Level 0) - Jun 13, 2024 - Abstract #SSIEM2024SSIEM_177;
Olipudase alfa is the first diseasemodifying treatment approved for adults and children with ASMD. Replicating literature findings, the results from our data demonstrate the clinical efficacy and safety of ERT in patients with ASMD, specifically focusing on its impact on various clinical parameters and patient outcomes over a short term.
- Xenpozyme (olipudase alfa) / Sanofi
Transforming ASMD Management: Clinical Perspectives on Timely Diagnosis and Treatment of ASMD (Camellia Auditorium | Level 0) - Jun 13, 2024 - Abstract #SSIEM2024SSIEM_45;
Cassiman and Ganesh will discuss the disease burden and unmet needs among patients with ASMD by leveraging real- world patient cases. This program will also feature olipudase alfa efficacy and safety data that supports use in appropriate real-world patients and highlight the significant impact of accurate diagnosis and timely intervention with olipudase alfa.
- | Xenpozyme (olipudase alfa) / Sanofi
FDA event, Journal: Regulatory news: Olipudase alfa-rpcp (Xenpozyme (Pubmed Central) - May 22, 2024
This program will also feature olipudase alfa efficacy and safety data that supports use in appropriate real-world patients and highlight the significant impact of accurate diagnosis and timely intervention with olipudase alfa. No abstract available
- | Xenpozyme (olipudase alfa) / Sanofi
Journal: Systematic investigation of genetically determined plasma and urinary metabolites to discover potential interventional targets for colorectal cancer. (Pubmed Central) - Apr 23, 2024
This study identified key metabolite biomarkers associated with CRC and elucidated their mediator roles in the associations between modifiable risk factors and CRC. These findings provide new insights into the etiology and potential therapeutic targets for CRC and the etiological pathways of modifiable environmental factors with CRC.
- || Xenpozyme (olipudase alfa) / Sanofi
Review, Journal: The Genetic Basis, Lung Involvement, and Therapeutic Options in Niemann-Pick Disease: A Comprehensive Review. (Pubmed Central) - Feb 28, 2024
The only approved disease-modifying therapy in NPD type C is miglustat, a substrate-reduction treatment. The aim of this review was to delineate a state of the art on the genetic basis and lung involvement in NPD, focusing on clinical manifestations, radiologic and histopathologic characteristics of the disease, and available therapeutic options, with a gaze on future therapeutic strategies.
- |||||| Xenpozyme (olipudase alfa) / Sanofi
Enrollment open, Real-world evidence, Real-world: OBS18020: A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD) (clinicaltrials.gov) - Feb 15, 2024
P=N/A, N=10, Recruiting, Sponsor: Sanofi
The aim of this review was to delineate a state of the art on the genetic basis and lung involvement in NPD, focusing on clinical manifestations, radiologic and histopathologic characteristics of the disease, and available therapeutic options, with a gaze on future therapeutic strategies. Not yet recruiting --> Recruiting
- | Xenpozyme (olipudase alfa) / Sanofi
Journal: Real-life impacts of olipudase alfa: The experience of patients and families taking an enzyme replacement therapy for acid sphingomyelinase deficiency. (Pubmed Central) - Feb 6, 2024
Not yet recruiting --> Recruiting 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.
- |||||| Xenpozyme (olipudase alfa) / Sanofi
New trial, Real-world evidence, Real-world: OBS18020: A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD) (clinicaltrials.gov) - Jan 5, 2024
P=N/A, N=10, Not yet recruiting, Sponsor: Sanofi
- || Xenpozyme (olipudase alfa) / Sanofi
Trial completion: ASCEND: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (clinicaltrials.gov) - Dec 4, 2023
P2/3, N=36, Completed, Sponsor: Genzyme, a Sanofi Company
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. Active, not recruiting --> Completed
- Xenpozyme (olipudase alfa) / Sanofi
LONG-TERM IMPROVEMENTS IN LIVER AND LIPID OUTCOMES IN ADULTS AND CHILDREN WITH ACID SPHINGOMYELINASE DEFICIENCY TREATED WITH OLIPUDASE ALFA ENZYME REPLACEMENT THERAPY (Room 302) - Oct 11, 2023 - Abstract #AASLD2023AASLD_1013;
P1, P1/2,
Active, not recruiting --> Completed Olipudase alfa treatment resulted in sustained improvements or normalization in liver and lipid parameters in adults and children with ASMD.
- || Xenpozyme (olipudase alfa) / Sanofi
Trial completion, Trial completion date, Trial primary completion date: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (clinicaltrials.gov) - Sep 25, 2023
P2, N=25, Completed, Sponsor: Genzyme, a Sanofi Company
Olipudase alfa treatment resulted in sustained improvements or normalization in liver and lipid parameters in adults and children with ASMD. Active, not recruiting --> Completed | Trial completion date: Feb 2024 --> Sep 2023 | Trial primary completion date: Feb 2024 --> Sep 2023
- Xenpozyme (olipudase alfa) / Sanofi
Plasma Lyso-Sphingomyelin Levels Correlate with Baseline Disease and Decline with Olipudase Alfa Treatment in Clinical Trials of Adults and Children with Acid Sphingomyelinase Deficiency (Exhibitions Hall (ICC)) - Jul 11, 2023 - Abstract #SSIEM2023SSIEM_395;
P1, P1/2,
In summary, plasma lyso-sphingomyelin was highly elevated in untreated patients with ASMD and these elevations correlated with most indices of disease severity. Treatment with olipudase alfa led to rapid plasma lyso-sphingomyelin reduction.
- Xenpozyme (olipudase alfa) / Sanofi
Continued improvement in liver and lipid outcomes in clinical trials of olipudase alfa in children and adults with chronic acid sphingomyelinase deficiency treated for 2 to 6.5 years (Dulzin 1 (ICC)) - Jul 6, 2023 - Abstract #SSIEM2023SSIEM_264;
P1, P1/2,
Olipudase alfa was generally safe and well tolerated; most adverse events (AEs) were mild or moderate with no trial withdrawals due to drug-related AEs. In summary, olipudase alfa treatment resulted in sustained improvements or normalization in liver and lipid parameters in adults and children with ASMD.
- Xenpozyme (olipudase alfa) / Sanofi
Reversal of interstitial lung disease after olipudase alfa enzyme replacement therapy in adults with acid sphingomyelinase deficiency (Blue 1+2) - Jun 17, 2023 - Abstract #ERS2023ERS_3181;
In summary, olipudase alfa treatment resulted in sustained improvements or normalization in liver and lipid parameters in adults and children with ASMD. Cell and molecular biology; Imaging; Pulmonary function testing; General respiratory patient care; Physiology
- || Xenpozyme (olipudase alfa) / Sanofi
Review, Journal: Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use. (Pubmed Central) - May 22, 2023
All these risks are generally manageable. A gradual dose escalation of olipudase alfa, followed by a maintenance phase, is required to reduce the risks of toxic sphingomyelin catabolites build up, infusion-associated reactions and transient transaminase elevations.
- Xenpozyme (olipudase alfa) / Sanofi
LONG-TERM IMPACT OF OLIPUDASE ALFA ENZYME REPLACEMENT THERAPY ON SPLEEN VOLUME AND HEMATOLOGIC MANIFESTATIONS IN CHILDREN AND ADULTS WITH CHRONIC ACID SPHINGOMYELINASE DEFICIENCY (Poster area) - May 12, 2023 - Abstract #EHA2023EHA_1983;
P1/2, P2,
Mean hemoglobin levels, which were mostly normal at baseline, increased slightly in adults and children. No adult or child had an increase in spleenvolume or new thrombocytopenia or anemia after 2 years of treatment.
- || Xenpozyme (olipudase alfa) / Sanofi
Clinical data, Journal: Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results. (Pubmed Central) - Dec 16, 2022
P1/2, P2
Improvements in clinically relevant disease endpoints observed during the first year of treatment were maintained or augmented in the second year. Trial registration NCT02004704 registered 26 Nov 2013, https://clinicaltrials.gov/ct2/show/record/NCT02004704 .
- Xenpozyme (olipudase alfa) / Sanofi
Improved Growth in Children with Acid Sphingomyelinase Deficiency Treated with Olipudase Alfa Enzyme Replacement Therapy (Anaheim Convention Center, Hall A, Board # 013) - Nov 10, 2022 - Abstract #AAPNCE2022AAP_NCE_2364;
P1/2, P2
In addition to improvements in organomegaly, pulmonary function, and dyslipidemia, all 20 children with ASMD from the ASCEND-Peds trial of olipudase alfa showed significant catch-up growth, with improvements in height Z-scores and CDC percentiles. Funded by Sanofi
- | Xenpozyme (olipudase alfa) / Sanofi
Journal: Olipudase Alfa-rpcp. (Pubmed Central) - Oct 19, 2022
Funded by Sanofi No abstract available
- Xenpozyme (olipudase alfa) / Sanofi
Value Contribution of Olipudase Alfa Therapy for the Treatment of Non-Central Nervous System Manifestations of Acid Sphingomyelinase Deficiency (ASMD) By Multi-Criteria Decision Analysis (MCDA) (In-person; Virtual) - Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1538;
ASMD is a rare disease with significant unmet needs and a high impact on morbidity, mortality, and quality of life. Olipudase alfa provides very high added value to the treatment of non-CNS manifestations of ASMD using MCDA, showing a significant improvement in all severity parameters studied and potentially modifying its clinical course.
- |||| Xenpozyme (olipudase alfa) / Sanofi
“#Xenpozyme, a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid #sphingomyelin” @MontefiorePeds @EinsteinMed @SuzettePediMD (Twitter) - Sep 1, 2022
- |||| Xenpozyme (olipudase alfa) / Sanofi
New treatment for a rare condition: @US_FDA approves #XenpozymeTM (olipudase alfa-rpcp) for the Rx non-CNS manifestations of acid sphingomyelinase deficiency (#ASMD). Congrats to Dr Melissa Wasserstein at #CHAM & the @SanofiUS team. https://t.co/0NDeHYqZcn (Twitter) - Sep 1, 2022
- | Baize'an (tislelizumab) / BeiGene, Novartis, Xenpozyme (olipudase alfa) / Sanofi, Tuoyi (toripalimab) / Shanghai Junshi Biosci, Coherus Biosci
Keeping Track: Longer US FDA Reviews For Tislelizumab, Toripalimab, Olipudase; Perrigo Wants OC To Go OTC https://t.co/NibXzqLWuG #PinkSheet (Twitter) - Jul 19, 2022
- || Xenpozyme (olipudase alfa) / Sanofi
Clinical, Journal: A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results. (Pubmed Central) - Jul 15, 2022
P2/3
Olipudase alfa provides very high added value to the treatment of non-CNS manifestations of ASMD using MCDA, showing a significant improvement in all severity parameters studied and potentially modifying its clinical course. Olipudase alfa was well tolerated and associated with significant and comprehensive improvements in disease pathology and clinically relevant endpoints compared with placebo in adults with ASMD.
- || Xenpozyme (olipudase alfa) / Sanofi
Review, Journal: Olipudase Alfa: First Approval. (Pubmed Central) - Jun 23, 2022
Regulatory review in the USA is underway. This article summarizes the milestones in the development of olipudase alfa leading to this first approval for the treatment of patients with ASMD.
- ||| Xenpozyme (olipudase alfa) / Sanofi
Enrollment open: OPERA: Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France (clinicaltrials.gov) - Jun 14, 2022
P=N/A, N=55, Recruiting, Sponsor: Sanofi
This article summarizes the milestones in the development of olipudase alfa leading to this first approval for the treatment of patients with ASMD. Not yet recruiting --> Recruiting
- |||| Xenpozyme (olipudase alfa) / Sanofi
European regulatory, HEOR: "CHMP backs Sanofi’s Xenpozyme as first drug for ASMD" https://t.co/TAn0WLcvOC via @pharmaphorum.com #HEOR #MarketAccess (Twitter) - May 30, 2022
- Xenpozyme (olipudase alfa) / Sanofi
OLIPUDASE ALFA FOR ADULTS WITH ACID SPHINGOMYELINASE DEFICIENCY: IMPROVEMENTS IN CROSSOVER PLACEBO PATIENTS AND FURTHER IMPROVEMENTS IN ORIGINAL OLIPUDASE ALFA PATIENTS AFTER 2 YEARS IN ASCEND TRIAL () - May 13, 2022 - Abstract #EHA2022EHA_2232;
P2/3
Olipudase alfa reduces manifestations of chronic ASMD in adults and has sustained efficacy. Increased awareness of ASMD among hematologists is important for timely diagnosis and disease management.
- |||||| Xenpozyme (olipudase alfa) / Sanofi
New trial: OPERA: Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France (clinicaltrials.gov) - May 3, 2022
P=N/A, N=55, Not yet recruiting, Sponsor: Sanofi
- olipudase alfa (GZ402665) / Sanofi
TWO-YEAR OUTCOMES OF OLIPUDASE ALFA THERAPY IN CHILDREN WITH CHRONIC ACID SPHINGMYELINASE DEFICIENCY () - Apr 20, 2022 - Abstract #ASPHO2022ASPHO_467;
P1/2, P2
Increased awareness of ASMD among hematologists is important for timely diagnosis and disease management. Clinical improvements noted at 1 year in the ASCEND-Peds trial of 20 children with chronic ASMD were sustained or amplified with no new safety issues after 2 years of olipudase alfa enzyme replacement therapy.Funded by Sanofi Genzyme.
- || olipudase alfa (GZ402665) / Sanofi
Review, Journal: Atherogenic lipid profile in patients with Niemann-Pick disease type B: What treatment strategies? (Pubmed Central) - Apr 20, 2022
Since olipudase alfa is not yet approved and, due to its costs, it will probably not be available for all patients worldwide, further research is needed to broaden our knowledge on this clinical need and to evaluate the efficacy and the long-term effects of lipid-lowering agents in ASMD patients. A deep understanding of the pathophysiology of dyslipidemia in ASMD may promote the identification of new targets and support the identification of new therapeutic strategies.
- || olipudase alfa (GZ402665) / Sanofi
Review, Journal: Acid Sphingomyelinase Deficiency: Sharing Experience of Disease Monitoring and Severity in France. (Pubmed Central) - Feb 26, 2022
The accessibility of the new enzyme replacement therapy olipudase alfa, a recombinant human ASM, has been expedited for clinical use based on positive clinical data in children and adult patients, such as improved respiratory status and reduced spleen volume. The aim of this article is to share the authors experience on monitoring ASMD patients and stratifying the severity of the disease to aid in care decisions.
- Xenpozyme (olipudase alfa) / Sanofi
Sustained Improvement of Interstitial Lung Disease Following Enzyme Replacement Therapy with Olipudase Alfa in Children and Adults with Chronic Acid Sphingomyelinase Deficiency (Room 2018/2020 (West Building, Level 2), Moscone Center) - Feb 19, 2022 - Abstract #ATS2022ATS_5987;
P1, P1/2,
The aim of this article is to share the authors experience on monitoring ASMD patients and stratifying the severity of the disease to aid in care decisions. Olipudase-alfa treatment results in progressive, clinically meaningful improvements in functional and imaging metrics of ILD in children and adults with ASMD.
- | olipudase alfa (GZ402665) / Sanofi
If approved, olipudase alfa will become the 1st & only therapy available for #ASMD @sanofi (Twitter) - Feb 5, 2022
- | olipudase alfa (GZ402665) / Sanofi
Journal: Interstitial lung disease in lysosomal storage disorders. (Pubmed Central) - Nov 29, 2021
Results of ERT are promising regarding preliminary results of olipudase alfa in paediatric and adult ASMD populations...Early diagnosis of these three LSDs is crucial to prevent irreversible organ damage. Early initiation of ERT can, at least in part, prevent organ failure.
- | Xenpozyme (olipudase alfa) / Sanofi
Trial completion date, Trial primary completion date: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (clinicaltrials.gov) - Sep 27, 2021
P2, N=25, Active, not recruiting, Sponsor: Genzyme, a Sanofi Company
Early initiation of ERT can, at least in part, prevent organ failure. Trial completion date: Sep 2023 --> Feb 2024 | Trial primary completion date: Sep 2023 --> Feb 2024
- || Xenpozyme (olipudase alfa) / Sanofi
Clinical, Journal: One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency. (Pubmed Central) - Sep 15, 2021
P1/2
Trial completion date: Sep 2023 --> Feb 2024 | Trial primary completion date: Sep 2023 --> Feb 2024 In this study in children with chronic ASMD, olipudase alfa was generally well-tolerated with significant, comprehensive improvements in disease pathology across a range of clinically relevant endpoints.
- || olipudase alfa (GZ402665) / Sanofi
Clinical, Journal: Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment. (Pubmed Central) - Jul 2, 2021
P1, P2
The clinical relevance of these findings needs to be determined in the future, but we speculate that these improvements may reduce the risk for liver cirrhosis and cardiovascular disease. Trial registration: Clintrials.gov trial registration # NCT01722526.