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Recombinant human acid sphingomyelinase · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Recombinant human acid sphingomyelinase | EXPERIMENTAL | Participants will receive rhASM of an initial dose of 0.1 mg/kg, followed by several dose escalations, as tolerated, up to 3.0 mg/kg. All doses are given 2 weeks apart. |
| Name | Type | Description |
|---|---|---|
| Recombinant human acid sphingomyelinase | DRUG | Administered intravenously every 2 weeks for 26 weeks |
Inclusion Criteria: * Patients with documented non-neuronopathic acid sphingomyelinase deficiency * The patient has a diffusing capacity of carbon monoxide (DLco) \>20% and ≤80% of the predicted normal value. * The patient has a spleen volume ≥6 multiples of normal(MN). A partial splenectomy will b...
Recombinant human acid sphingomyelinase is an investigational therapy being developed for the treatment of Human Acid Sphingomyelinase Deficiency, a rare disease. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
Recombinant human acid sphingomyelinase is being developed by Sanofi, a global biopharmaceutical company listed on the stock exchange under the ticker SNY. The drug is in Phase 1 clinical development for Human Acid Sphingomyelinase Deficiency.
Recombinant human acid sphingomyelinase is in Phase 1 clinical development. It is an investigational drug for Human Acid Sphingomyelinase Deficiency and has not been approved by the FDA or other regulatory agencies.
Recombinant human acid sphingomyelinase has one completed Phase 1 trial, NCT01722526, which studied its tolerability and safety in patients with Acid Sphingomyelinase Deficiency. The trial enrolled 5 participants in the United States and United Kingdom.
No, Recombinant human acid sphingomyelinase is not the same as olipudase alfa. Recombinant human acid sphingomyelinase is a distinct investigational drug being developed by Sanofi for Human Acid Sphingomyelinase Deficiency, and it is currently in Phase 1 clinical trials.