‘Approved for use in uveitis’: drug approval for an orphan disease

Metzinger, Jamie Lynne; Foster, C. Stephen

doi:10.1517/21678707.2015.1052792

Cited by 2 publications

(1 citation statement)

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“…Our team has reviewed IMT drugs approved by the US Food and Drug Administration (FDA) for use in uveitis. 5 In this study, we review and summarize conventional IMT drugs or biologics that have not received approval as an “orphan drug” from the FDA for use in noninfectious uveitic patients, despite having been approved for use in treating other systemic diseases or organ transplantation.…”

Section: Introductionmentioning

confidence: 99%

A review and update on orphan drugs for the treatment of noninfectious uveitis

You¹,

Sahawneh²,

Ma³

et al. 2017

OPTH

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IntroductionUveitis, a leading cause of preventable blindness around the world, is a critically underserved disease in regard to the medications approved for use. Multiple immunomodulatory therapy (IMT) drugs are appropriate for uveitis therapy but are still off-label. These IMT agents, including antimetabolites, calcineurin inhibitors, alkylating agents, and biologic agents, have been designated as “orphan drugs” and are widely used for systemic autoimmune diseases or organ transplantation.Area coveredThe purpose of this paper is to comprehensively review and summarize the approved orphan drugs and biologics that are being used to treat systemic diseases and to discuss drugs that have not yet received approval as an “orphan drug for treating uveitis” by the US Food and Drug Administration (FDA).Our perspectiveIMT, as a steroid-sparing agent for uveitis patients, has shown promising clinical results. Refractory and recurrent uveitis requires combination IMT agents. IMT is continued for a period of 2 years while the patient is in remission before considering tapering medication. Our current goals include developing further assessments regarding the efficacy, optimal dose, and safety in efforts to achieve FDA approval for “on-label” use of current IMT agents and biologics more quickly and to facilitate insurance coverage and expand access to the products for this orphan disease.

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Section: Introductionmentioning

confidence: 99%