Lanacogene vosiparvovec is under clinical development by UniQure and currently in Phase II for Hemophilia B (Factor IX Deficiency). According to GlobalData, Phase II drugs for Hemophilia B (Factor IX Deficiency) have a 66% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Lanacogene vosiparvovec’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. 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.

Lanacogene vosiparvovec overview

AMT-060 (lanacogene vosiparvovec) is under development for the treatment of Hemophilia B. The drug candidate is formulated as a solution and administered as an intravenous infusion. It is developed by using proprietary AAV vector technology. This proprietary platform uses adeno-associated viral (AAV5) derived vectors as the delivery vehicle that carries a therapeutic human Factor IX, or hFIX, gene cassette. AMT-060 consists of a codon-optimized wild-type FIX gene and the LP1 liver promoter together with the adeno-associated virus vector serotype 5 (AAV5) viral vector.

UniQure overview

UniQure develops and markets gene therapy products. The company develops adeno-associated virus (AAV) based gene therapies using its gene technology platform and offers disease-modifying treatments to patients with severe genetic diseases and other devastating diseases. UniQure’s lead product, AMT-060, is a gene therapy for the treatment of moderately-severe hemophilia B. Its preclinical products include AMT-130, an AAV5 vector for the treatment of Huntington’s disease and AMT-150 for Spinocerebellar Ataxia type 3. The company develops a gene platform to bring new disease modifying therapies to patients with genetic diseases. The company conducts drug discovery and preclinical research in collaboration with academic research institutions. UniQure is headquartered in Amsterdam, the Netherlands.

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

This content was updated on 2 September 2023

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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.