Olipudase alfa
- Generic Name
- Olipudase alfa
- Brand Names
- Xenpozyme
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 927883-84-9
- Unique Ingredient Identifier
- 6D5766Q4OP
- Background
Olipudase alfa is recombinant human acid sphingomyelinase. It is the first and only enzyme replacement therapy in the world for the treatment of Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann–Pick disease. ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the a...
- Indication
Olipudase alfa is indicated as an enzyme replacement therapy for the treatment of non-Central Nervous System manifestations of Acid Sphingomyelinase Deficiency (ASMD) in pediatric and adult patients with type A/B or type B.
- Associated Conditions
- Acid Sphingomyelinase Deficiency (ASMD)
- Associated Therapies
- -
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)
- Conditions
- Interventions
- First Posted Date
- 2024-01-05
- Last Posted Date
- 2024-12-02
- Lead Sponsor
- Sanofi
- Target Recruit Count
- 10
- Registration Number
- NCT06192576
- Locations
- 🇺🇸
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸Children's Hospital Medical Center, Cincinnati, Ohio, United States
🇺🇸Nationwide Children's Hospital, Columbus, Ohio, United States
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
- First Posted Date
- 2022-05-03
- Last Posted Date
- 2024-10-26
- Lead Sponsor
- Sanofi
- Target Recruit Count
- 55
- Registration Number
- NCT05359276
- Locations
- 🇫🇷
Investigational site in France, France, France
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
- First Posted Date
- 2014-11-17
- Last Posted Date
- 2022-03-23
- Lead Sponsor
- Genzyme, a Sanofi Company
- Target Recruit Count
- 20
- Registration Number
- NCT02292654
- Locations
- 🇮🇹
Investigational Site Number 380001, Udine, Italy
🇧🇷Investigational Site Number 076001, Porto Alegre, Brazil
🇺🇸Investigational Site Number 840001, New York, New York, United States
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
- Conditions
- Interventions
- First Posted Date
- 2013-12-09
- Last Posted Date
- 2024-11-07
- Lead Sponsor
- Genzyme, a Sanofi Company
- Target Recruit Count
- 36
- Registration Number
- NCT02004691
- Locations
- 🇺🇸
Montefiore Medical Center Site Number : 840006, Bronx, New York, United States
🇮🇹Investigational Site Number : 380002, Napoli, Italy
🇮🇹Investigational Site Number : 380001, Udine, Italy
Drug Development Updates
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