FDA fast track status granted to iECURE neonatal OTC deficiency treatment

2024-05-08

快速通道孤儿药基因疗法临床研究突破性疗法

FDA fast track status granted to iECURE neonatal OTC deficiency treatment

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来源: Pharmaceutical Technology

The study of ECUR-506 will enrol newborn males aged up to seven months diagnosed with severe neonatal onset OTC deficiency. Credit: Gorodenkoff / Shutterstock.com.

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来源: Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted fast track designation for iECURE’s gene editing therapy, ECUR-506, for the treatment of neonatal onset ornithine transcarbamylase (OTC) deficiency ornithine transcarbamylase (OTC) deficiency.

ECUR-506 is an in vivo gene insertion programme.

The status will accelerate the development and review of treatments for serious conditions with unmet medical needs.

The company can now hold frequent interactions with the FDA to discuss the development plan and appropriate data collection for potential approval.

The therapeutic benefits of ECUR-506 have been recognised previously, with the FDA awarding it rare paediatric disease and orphan drug designations.

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The European Commission also granted orphan designation for the treatment of OTC deficiency.

ECUR-506 is being evaluated in the Phase I/II OTC-HOPE study, a first-in-human trial targeting newborn males with genetically confirmed neonatal onset OTC deficiency.

The study is enrolling participants at the Great Ormond Street Hospital for Children in London, UK with additional sites in the US and Australia set to commence enrolment later in 2024.

The OTC-HOPE study is focused on enrolling newborn males aged up to seven months diagnosed with severe neonatal onset OTC deficiency.

Its primary goal is to evaluate the safety and tolerability of a single intravenous dose of ECUR-506.

Secondary objectives include evaluating the pharmacokinetics and efficacy of the therapy.

Exploratory endpoints will investigate disease-specific biological markers, developmental milestones and the impact on the patient’s quality of life.

iECURE CEO Joe Truitt stated: “Receipt of fast track designation from the FDA is a validation of the severe unmet need for patients with neonatal onset OTC deficiency and a testament to the preclinical data generated to date for ECUR-506.

“The benefits of fast track designation may accelerate our ability to get ECUR-506 into physicians’ hands, which is incredibly important when every second counts for these babies.”

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