A drug designed to help blind people maintain sleeping patterns has been recommended for approval in the US.
A Food Drug and Administration (FDA) advisory committee voted unanimously to recommend approval of Vanda Pharmaceuticals’ Hetlioz (tasimelteon) for non-24-hour disorder (N24) in the totally blind. The condition is thought to result from the lack of light stimulation for the areas of the brain that govern circadian rhythms.
If approved, Helioz would be the first drug registered for N24, which causes insomnia at night and excessive sleepiness in the daytime and is termed an “invisible disability”, with a dramatic impact on the well-being and social functioning of people with the condition.
It is estimated that around 65,000 to 95,000 people in the US are affected by N24, with most totally blind, although it does occur rarely in sighted people. Tasimelteon acts on melatonin MT1/MT2 receptors and is thought to stimulate the brain via the same pathway as sunlight.
The panel concluded not only that the N24 indication was appropriate for an FDA-approved therapy, but also that the clinical trial measures employed by Vanda to demonstrate Hetlioz’ safety and efficacy were robust.
The FDA is due to deliver its verdict on Hetlioz by the end of January and – if positive – some analysts have predicted that Helioz could achieve sales of $200m a year or more at peak.
“For the thousands of blind patients with N24, Hetlioz may offer – for the first time in their lives – the ability to live in sync with a 24-hour world,” commented Vanda chief executive Mihael Polymeropoulos during the company’s third-quarter results call last week.
As a result, they can “fully enjoy the opportunities of education, employment and social interactions in our 24-hour society,” he added.
Hetlioz would be Vanda’s first self-marketed product and the company has been steadily building up a commercial unit to market the drug if approved by the FDA, establishing contacts with physicians dealing with N24 patients and discussing the new drug with healthcare payers.
“All key leadership positions are filled, across marketing, sales, managed markets and trade, and our current focus is the build-out of the direct customer-facing teams across all groups,” noted Polymeropoulos.
Vanda previously developed a schizophrenia treatment called Fanapt (iloperidone) which is licensed in North America to Novartis but has failed to make much headway in the market since its launch in 2010.Vanda withdrew a marketing application for this product in the EU earlier this year.