

Drug discovery
2
drugs
With orphan designations
Overview
Central retinal artery occlusion (CRAO) is an ophthalmic emergency characterized by sudden, painless monocular vision loss due to retinal ischemia, typically caused by thromboembolism from carotid/cardiac sources or vasculitis (e.g., giant cell arteritis). Diagnosis relies on funduscopy findings (pale retina, cherry-red spot, arterial attenuation) and systemic evaluation for embolic sources. Prognosis is poor, with <20% achieving functional visual recovery without timely intervention. Urgent referral to stroke centers is critical due to elevated cerebrovascular risk [1][4][9].
Therapies
Acute phase: Ocular massage, intraocular pressure reduction (topical timolol, IV acetazolamide), and hyperbaric oxygen (if <12 hours) [3][9][17].
Thrombolytics: IV or intra-arterial tPA within 4.5–6 hours may improve outcomes but lack robust evidence [7][9][12].
Secondary prevention: Antiplatelet therapy, statins, and management of vascular risk factors [4][6][9].
Categories: rare ophthalmic disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Sodium 4-amino-3- [6-( 4-fluoro-2-methylphenyl)pyridine-3-ylazo] naphthalene-1-sulfonate dihydrate | small molecules | FDA | 2022-05-03 | — | Kyoto Drug Discovery & Development Co., Ltd. |
mesencephalic, astrocyte-derived neurotrophic factor | proteins | FDA | 2015-09-10 | — | Amarantus BioScience Holdings, Inc. |