On 25 April, Santen Pharmaceutical announced that a New Drug Application (NDA) for one of its lead products, Opsiria (sirolimus), was accepted by the FDA for review. If approved, the drug will be used to treat patients with non-infectious uveitis of the posterior segment (NIU-PS), a disease with a high level of unmet need. An action date of 24 December has been set by the FDA for the orphan drug-designated treatment. Following positive data from the Sakura global development program, GlobalData believes that, if approved, Ospiria has significant market opportunity to fulfil a large clinical unmet need.
NIU-PS is a progressive and chronic inflammatory disease of the eye that includes intermediate, posterior, and panuveitis. The pathogenesis of uveitis is believed to be driven by different lines of T lymphocytes. First-line treatment for acute NIU-PS commonly involves systemic corticosteroids, which are well known for their adverse effects.
According to key opinion leaders interviewed by GlobalData, corticosteroids are inadequate in permanently controlling inflammation in approximately 40% of patients, as their side effects become less tolerable over time. At this stage in treatment, immunosuppressive drugs and biologics become part of the treatment strategy. They are used in a ‘step-ladder’ approach as second- and third-line treatment. However, concerns have been raised about the long-term side effects of these systemically applied agents that often become treatment-limiting. Consequently, GlobalData believes developing a therapy with an improved side effect profile is essential in meeting the many high unmet needs in this disease area.
Since Opsiria is administered as an intravitreal injection, such local, eye-specific administration could potentially allow the prevention of systemic side effects seen with corticosteroids, immunosuppressant drugs and biologics. Furthermore, intravitreal injection is a well-established route of administration for uveitis patients. Given its positive results in Phase III studies and its potential to address the high unmet need in this area, GlobalData anticipates Opsiria to cannibalize the sales of corticosteroid treatments in uveitis patients, and possibly even lead to their discontinuation. This would be a welcome result for patients, given that long-term corticosteroid treatment is associated with severe adverse effects.
GlobalData (2013). Opportunity Analyzer: Uveitis – Opportunity Analysis and Forecasts to 2017, December 2013, GDHC008POA