OTL-201 is under clinical development by Orchard Therapeutics and currently in the Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect OTL-201’s likelihood of approval (LoA) and phase transition for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) took place on 12 Feb 2021, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their OTL-201 Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

OTL-201 overview

OTL-201 is under development for the treatment of mucopolysaccharidosis-IIIA (Sanfilippo Syndrome Type A). The therapeutic candidate is developed based on ex-vivo lentiviral gene therapy. It is an autologous MPSIIIA HSCs transduced with either LV-PGK-coSGSH or LV-CD11b-coSGSH vectors expressing codon-optimized SGSH. The therapeutic candidate acts by targeting N-sulfoglucosamine sulfohydrolase (SGSH) enzyme.

Orchard Therapeutics overview

Orchard Therapeutics (Orchard) is a biotechnology company that develops gene therapies for the treatment of serious and life-threatening orphan diseases. Its pipeline products include Strimvelis, an autologous ex vivo gammaretroviral gene therapy for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID); OTL-103 for Wiskott-Aldrich syndrome (WAS); OTL-102 for X-linked chronic granulomatous disease (X-CGD); OTL-300 for transfusion-dependent beta-thalassemia; and OTL-200 for metachromatic leukodystrophy (MLD), among others. It collaborates with institutions that specialize in gene and cell therapy such as The University of Manchester, Boston Children’s Hospital, and The San Raffaele Telethon Institute for Gene Therapy. It operates in France, Germany, Italy, Switzerland, the Netherlands, the US and the UK. Orchard is headquartered in London, Greater London, the UK.

Quick View OTL-201 LOA Data

Report Segments
  • Innovator
Drug Name
  • OTL-201
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Genetic Disorders
Key Developers
Highest Development Stage
  • Phase II

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.