Drugs / OTL-203
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial · 1 met primary | ||||||
| Phase 1/2 | NCT03488394 | May 2018 → Jan 2035 expected | Hurler syndrome | Orchard Therapeutics | Active not recruiting | Met primary |
| Phase 31 trial | ||||||
| Phase 3 | NCT06149403 | Dec 2023 → Mar 2028 expected | Hurler syndrome | Orchard Therapeutics | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 15
| Date | Issuer | Release |
|---|---|---|
| 2023-11-30 | Orchard Therapeutics | Regulatory Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). |
| 2023-10-26 | Orchard Therapeutics | Results Orchard Therapeutics Announces Presentation of Additional Positive Data from Proof-of-concept Study of OTL-203 in MPS-IH at ESGCT 2023 orchard-tx.com ↗
New clinical data beyond previously reported neurological and skeletal results demonstrate favorable outcomes for multiple disease manifestations not effectively addressed by the current standard of care |
| 2023-10-24 | Orchard Therapeutics | Results Orchard Therapeutics Announces Multiple Presentations at ESGCT 2023 orchard-tx.com ↗
Data from proof-of-concept study of OTL-203 in MPS-IH demonstrate favorable outcomes for clinical manifestations of the disease not well addressed by the current standard-of-care |
| 2023-08-03 | Orchard Therapeutics | Results Orchard Therapeutics Completes Submission of Biologics License Application for OTL-200 in MLD to U.S. FDA orchard-tx.com ↗
Additional OTL-203 proof-of-concept data demonstrated extensive metabolic correction in the skeletal system of patients with mucopolysaccharidosis type I Hurler syndrome (MPS-IH) resulting in normal growth rates, skeletal remodeling, improvement in joint function and progressive acquisition of motor skills. |
| 2023-05-19 | Orchard Therapeutics | Results Orchard Therapeutics Announces Positive Clinical and Preclinical Data in Programs Targeting Neurometabolic and CNS Disorders at ASGCT orchard-tx.com ↗
New OTL-203 proof-of-concept data demonstrate extensive metabolic correction in the skeletal system of patients with MPS-IH including normal growth rates, improvement in joint function and progressive acquisition of motor skills |
| 2023-01-09 | Orchard Therapeutics | Regulatory Orchard Therapeutics Highlights Recent Progress Across HSC Gene Therapy Portfolio and Outlines Key 2023 Milestones orchard-tx.com ↗
OTL-203 (MPS-IH) IND application cleared by U.S. FDA with global registrational trial to initiate in the second half of 2023 |
| 2023-01-05 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces U.S. FDA Clearance of IND Application for OTL-203 in MPS-IH orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for OTL-203, a hematopoietic stem cell (HSC) gene therapy being developed for the treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). |
| 2021-11-18 | Orchard Therapeutics | Results Orchard Therapeutics Announces New England Journal of Medicine Publication of Interim Proof-of-concept Study Results of OTL-203 for Hurler Syndrome orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine (NEJM) evaluating the safety and efficacy of OTL-203 for the treatment of the Hurler subtype of Mucopolysaccharidosis type I (MPS-IH). |
| 2021-06-29 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces Regulatory and Clinical Updates for Lead Gene Therapy Programs orchard-tx.com ↗
Orchard recently held a productive meeting with the U.S. Food and Drug Administration (FDA) and has received written feedback concerning the clinical package expected to support a Biologics License Application (BLA) for OTL-200 in MLD. |
| 2021-02-09 | Orchard Therapeutics | Results Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH) orchard-tx.com ↗
All eight patients treated with OTL-203 show stable cognitive function, motor function and growth within the normal range at multiple data points post-treatment |
| 2020-09-28 | Orchard Therapeutics | Regulatory Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. |
| 2020-09-01 | Orchard Therapeutics | Results Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I orchard-tx.com ↗
Data on all eight patients demonstrate sustained engraftment and supranormal IDUA enzyme expression |
| 2020-07-20 | Orchard Therapeutics | Regulatory Orchard Therapeutics Announces Orphan Drug and Rare Pediatric Disease Designations for OTL-203 for the Treatment of MPS-I orchard-tx.com ↗ |
| 2020-05-15 | Orchard Therapeutics | Results Orchard Therapeutics Presents New Interim Data from OTL-203 Proof-of-concept Study for MPS-I orchard-tx.com ↗
We also observed reconstitution of enzyme activity in the cerebral spinal fluid (CSF) in the first two treated patients with 12 months of follow-up, complemented by a decrease in glycosaminoglycan levels in the CSF. |
| 2019-09-04 | Orchard Therapeutics | Results Orchard Therapeutics Announces Encouraging Update from Proof-of-Concept Study of OTL-203 for the Treatment of Mucopolysaccharidosis Type I (MPS-I) orchard-tx.com ↗
Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced data from an ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203, a gene therapy for the treatment of mucopolysaccharidosis type I (MPS-I) developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan. |
All press releases naming this drug 17 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | OTL-203 | “overall OTL-203 Clinical Development Program” NCT03488394 ↗ |
| Action | Restore | “encoding for the human α-L-iduronidase gene” NCT03488394 ↗ |
| Modality | Cell therapy | “genetically modified autologous hematopoietic stem cells” NCT03488394 ↗ |
| Route | Intravenous | “The product will be injected intravenously.” NCT03488394 ↗ |
| Target | IDUA | “IDUA lentiviral vector encoding for the human α-L-iduronidase gene” NCT03488394 ↗ |