European approval of the IGF-1-derived small molecule analogue offers a new option for patients aged five and over with the rare neurodevelopmental disorder.
The European Commission (EC) has approved Acadia Pharmaceuticals’ Daybu (trofinetide) as the first treatment for neurobehavioral symptoms of the Rett Syndrome in the EU.
The oral small molecule analogue of glycine-proline-glutamate is indicated for treating neurobehavioral symptoms in individuals five years and old and over.
Patients with the condition have low levels of the hormone IGF-1, which is important for the normal development and functioning of the nervous system. The active substance in Daybu contains a molecule derived from IGF-1.
The EC’s decision follows earlier marketing authorisations of Daybu for the rare disorder in the US and Canada.
Dr Nadia Bahi, PhD, Pediatric Neurology at Necker-Enfants Malades University Hospital, Paris Cité University, France, remarked: “Rett syndrome places a profound burden on individuals, families, and caregivers, yet treatment options have remained extremely limited.”
As such, authorisation of Daybu provides a new option to treat the genetic neurodevelopment disorder. This is primarily based on the Phase III LAVENDER study. Data illustrated statistically significant and clinically meaningful improvements in core features of Rett syndrome with Daybu.
Catherine Owen Adams, Acadia’s Chief Executive Officer, said: “The approval of Daybu marks a significant milestone for the Rett syndrome community in the EU and advances our mission to bring this innovative treatment to patients and families who have long faced a profound unmet medical need.”
The approval of Daybu marks a significant milestone for the Rett syndrome community in the EU and advances our mission to bring this innovative treatment to patients and families who have long faced a profound unmet medical need”
Catherine Owen Adams, Acadia’s Chief Executive Officer
Also in the rare disease space, earlier this month Jazz Pharmaceuticals acquired Actio Biosciences for up to $1.32 billion, to advance a first-in-class therapeutic for a rare epilepsy. Like Rett syndrome, KCNT1+ epilepsy is associated with significant developmental and neurological impairment. If the candidate advances through development, its approval could also help address unmet need in this patient population.
No comments yet