Drug Profile

APR 2020

Alternative Names: APR-2020

Latest Information Update: 30 Apr 2026

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At a glance

Originator Apriligen
Class Antianaemics; Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Diamond-Blackfan syndrome
New Molecular Entity No

Highest Development Phases

Phase I Diamond-Blackfan syndrome

Most Recent Events

16 Apr 2026 Phase-I clinical trials in Diamond-Blackfan syndrome (In children, In adolescents, In adults) in USA (IV) (NCT07476183)
19 Mar 2026 Apriligen plans a phase-I/II trial for Diamond-Blackfan syndrome (In children, In adolescents) in USA and Europe (Parenteral), (NCT07476183) (Apriligen website, March 2026)
01 Jan 2026 APR 2020 receives Fast Track designation for Diamond-Blackfan syndrome [Parenteral] in USA (Apriligen website, March 2026)

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