Feb 20, 2025 - The use of zebrafish-based preclinical trial data in drug development has become a significant milestone in securing Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). Zefit’s partner, iQure Pharma Inc. (iQure), announced that its drug candidate for Dravet Syndrome (DS) has been granted ODD status by the FDA.

Zefit’s zebrafish preclinical data played a pivotal role in the FDA’s approval, highlighting the increasing importance of zebrafish models in drug development and regulatory approval processes.

Zebrafish Preclinical Data Recognized as a Valid FDA-Approved Case Study

Zefit is a Contract Research Organization (CRO) specializing in high-throughput preclinical screening and disease modeling using zebrafish models. The company provides fast and precise efficacy and safety assessments of drug candidates, offering a reliable and efficient alternative to traditional models.

During the FDA's ODD approval process, Zefit’s zebrafish preclinical data was officially recognized, reinforcing the scientific credibility of zebrafish models in regulatory pathways.

Pawel Zolnierczyk, CEO of iQure Pharma, emphasized, “The FDA’s Orphan Drug Designation is a critical milestone that brings our drug candidate closer to clinical trials. Zefit’s preclinical research data played a significant role in regulatory evaluation.”

Zebrafish Models: A Key Platform in Drug Development

Compared to traditional mammalian models, zebrafish provide a more cost-effective, scalable, and rapid drug evaluation method. As a result, zebrafish models are increasingly recognized as an essential tool in preclinical drug discovery.

This case serves as strong evidence that zebrafish-based preclinical studies can be accepted and trusted by regulatory agencies—particularly in neurological disorder research and orphan drug development.

Ki Baek Lee, CEO of Zefit, stated, “This achievement is highly significant as it marks an official recognition by the FDA of zebrafish-based preclinical testing as a reliable evaluation method. Moving forward, we aim to expand our collaborations with innovative biotech and pharmaceutical companies to accelerate drug development and facilitate the regulatory approval process for zebrafish models.”

FDA Orphan Drug Designation: Accelerating Clinical Trials for iQure’s Drug Candidate

The FDA’s Orphan Drug Designation (ODD) program provides various benefits to pharmaceutical companies, including market exclusivity, tax credits, and expedited regulatory reviews—key incentives designed to encourage the development of treatments for rare diseases such as Dravet Syndrome.

With the FDA designation secured, iQure's drug candidate is now preparing to enter clinical trials. This regulatory milestone is expected to accelerate its research, development, and commercialization processes.

Ultimately, this FDA approval reinforces the role of zebrafish-based preclinical research as a valuable and credible evaluation model in drug development and regulatory approvals.

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Company Name: Zefit Inc.
Contact Person: James Kang
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Phone: +82-53-716-0816
Country: South Korea
Website: www.zefit.co.kr