Sirolimus for the treatment of noninfectious uveitis

Expert Opin Pharmacother. 2016;17(1):127-35. doi: 10.1517/14656566.2016.1124855.


Introduction: Noninfectious posterior uveitis (NIPU) remains a significant burden of legal blindness. Because of its immune mediated and chronic recurrent nature, common therapy includes (systemic) corticosteroids and immune modulatory agents. Most treatments bear the risk of significant adverse effects. Therefore efforts are made to administer therapeutic agents directly into the vitreous cavity. The purpose of this article is to identify the role of intravitreally applied sirolimus as a recently approved therapeutic option in NIPU.

Areas covered: A MEDLINE database search was conducted through August 2015 using the terms: intravitreal injection, pharmacology, sirolimus, treatment and uveitis. To provide ongoing and future perspectives in treatment options, also clinical trials as registered at were included. Sirolimus (Opsiria) was in licensed from SANTEN in 2015 and approved in Phase III registration trials in the US, Europe and other countries for NIPU. Current information results mainly from registration and Phase III trials.

Expert opinion: Intravitreal sirolimus appears to be an interesting option in the treatment algorithms of NIPU because of its highly targeted molecular effects, nonsteroidal nature and good safety profile. It has the advantage to avoid systemic side effects, but this has to be balanced against the fact that treatment covers one eye only and bears the risks of any intraocular procedure. Nevertheless a careful evaluation of this agent has to be made, as current experience is almost exclusively based on registration trials and long-term effects still have to be explored.

Keywords: intravitreal injection; mTOR inhibitor; sirolimus; uveitis.

MeSH terms

  • Clinical Trials, Phase III as Topic
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Intravitreal Injections
  • Sirolimus / therapeutic use*
  • Uveitis / drug therapy*


  • Immunosuppressive Agents
  • Sirolimus