The very first trofinetide in Rett syndrome trial was restricted to adolescents and adults (16-45 years). Lavender covers the 5-20 year age group, which will allow paediatric labelling (and potentially a PRV) if approved, albeit for those aged 5 and over. Anavex’s pivotal trial in Rett is restricted to 5+ years. Newron’s trial of sarizotan in Rett syndrome was held in 4+ year olds.
But what happens in the case of a disease/condition that also impacts children in early childhood, such as Rett syndrome? Do those aged below 5 years miss out on access to therapies?
If you take Covid vaccines as an example of how it works, initial trials were conducted in 12+ year olds (30 mcg). Then, at the FDA’s urging, trials were held in children aged 5 -11 (10 mcg). The next age group will be 2-5 years (3mcg), followed by those aged 6 mths-2 years.
As well as ensuring that it’s safe to administer trofinetide in those aged younger than 5 years of age, testing in that specific age group may allow an expanded label (which equates to a larger market). More importantly, as early onset of Rett syndrome typically occurs between the age of 6 and 18 mths, evidence of safety in children as young as two could enable early access to treatment, perhaps before the second, rapid destructive stage occurs.
I would think that the Rett community will be pleased to see Acadia moving early on this early childhood safety study.
Just my thoughts.
NEU Price at posting:
$1.70 Sentiment: Hold Disclosure: Held
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