OTL-203 is under clinical development by Orchard Therapeutics and currently in Phase III for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ). According to GlobalData, Phase III drugs for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) does not have sufficient historical data to build an indication benchmark PTSR for Phase III. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the OTL-203 LoA Report. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

OTL-203 overview

OTL-203 is under development for the treatment of Mucopolysaccharidosis type I (MPS I). It is administered intravenously. It acts by targeting a-L-iduronidase (IDUA). The therapy is composed of autologous CD34 positive enriched HSPCs genetically modified by LVs to deliver IDUA gene. Lentiviral vectors are used to transduce hematopoietic stem cells and direct IDUA expression.

Orchard Therapeutics overview

Orchard Therapeutics (Orchard) is a biotechnology company that develops gene therapies for the treatment of serious and life-threatening orphan diseases. The company’s pipeline products include OTL-200, OTL-203, OTL-201, OTL-204, OTL-103, OTL-104 and OTL-105. Its pipeline candidates treat metachromatic leukodystrophy (MLD), mucopolysaccharidosis type I (MPS-I), mucopolysaccharidosis type IIIA and Wiskott Aldrich syndrome (WAS). It markets under the brand name of Strimvelis, Libmeldy.

For a complete picture of OTL-203’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.