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

Caiyun You,1–3 Haitham F Sahawneh,1,2 Lina Ma,1,2 Buraa Kubaisi,1,2 Alexander Schmidt,1,2 C Stephen Foster1,2,4 1Massachusetts Eye Research and Surgery Institution (MERSI), Waltham, 2Ocular Immunology and Uveitis Foundation, Weston, MA, USA; 3Department of Ophthalmology, Tianjin Medical U...

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Autores principales:	You C, Sahawneh HF, Ma L, Kubaisi B, Schmidt A, Foster CS
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2017
Materias:	Immunomodulatory orphan drug steroid sparing uveitis Ophthalmology RE1-994
Acceso en línea:	https://doaj.org/article/1fddded69420494c8f2fdc398687db15
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spelling	oai:doaj.org-article:1fddded69420494c8f2fdc398687db152021-12-02T06:26:47ZA review and update on orphan drugs for the treatment of noninfectious uveitis1177-5483https://doaj.org/article/1fddded69420494c8f2fdc398687db152017-01-01T00:00:00Zhttps://www.dovepress.com/a-review-and-update-on-orphan-drugs-for-the-treatment-of-noninfectious-peer-reviewed-article-OPTHhttps://doaj.org/toc/1177-5483Caiyun You,1–3 Haitham F Sahawneh,1,2 Lina Ma,1,2 Buraa Kubaisi,1,2 Alexander Schmidt,1,2 C Stephen Foster1,2,4 1Massachusetts Eye Research and Surgery Institution (MERSI), Waltham, 2Ocular Immunology and Uveitis Foundation, Weston, MA, USA; 3Department of Ophthalmology, Tianjin Medical University General Hospital, Tianjin, People’s Republic of China; 4Harvard Medical School, Boston, MA, USA Introduction: Uveitis, 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 covered: The 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 perspective: IMT, 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. Keywords: immunomodulatory, orphan drug, steroid sparing, uveitisYou CSahawneh HFMa LKubaisi BSchmidt AFoster CSDove Medical PressarticleImmunomodulatoryorphan drugsteroid sparinguveitisOphthalmologyRE1-994ENClinical Ophthalmology, Vol Volume 11, Pp 257-265 (2017)
institution	DOAJ
collection	DOAJ
language	EN
topic	Immunomodulatory orphan drug steroid sparing uveitis Ophthalmology RE1-994
spellingShingle	Immunomodulatory orphan drug steroid sparing uveitis Ophthalmology RE1-994 You C Sahawneh HF Ma L Kubaisi B Schmidt A Foster CS A review and update on orphan drugs for the treatment of noninfectious uveitis
description	Caiyun You,1–3 Haitham F Sahawneh,1,2 Lina Ma,1,2 Buraa Kubaisi,1,2 Alexander Schmidt,1,2 C Stephen Foster1,2,4 1Massachusetts Eye Research and Surgery Institution (MERSI), Waltham, 2Ocular Immunology and Uveitis Foundation, Weston, MA, USA; 3Department of Ophthalmology, Tianjin Medical University General Hospital, Tianjin, People’s Republic of China; 4Harvard Medical School, Boston, MA, USA Introduction: Uveitis, 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 covered: The 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 perspective: IMT, 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. Keywords: immunomodulatory, orphan drug, steroid sparing, uveitis
format	article
author	You C Sahawneh HF Ma L Kubaisi B Schmidt A Foster CS
author_facet	You C Sahawneh HF Ma L Kubaisi B Schmidt A Foster CS
author_sort	You C
title	A review and update on orphan drugs for the treatment of noninfectious uveitis
title_short	A review and update on orphan drugs for the treatment of noninfectious uveitis
title_full	A review and update on orphan drugs for the treatment of noninfectious uveitis
title_fullStr	A review and update on orphan drugs for the treatment of noninfectious uveitis
title_full_unstemmed	A review and update on orphan drugs for the treatment of noninfectious uveitis
title_sort	review and update on orphan drugs for the treatment of noninfectious uveitis
publisher	Dove Medical Press
publishDate	2017
url	https://doaj.org/article/1fddded69420494c8f2fdc398687db15
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A review and update on orphan drugs for the treatment of noninfectious uveitis

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