Neuren Pharmaceuticals receives FDA approval for world-first drug to treat Rett syndrome
Neuren’s lead candidate Daybue is expected to be available to the US market as an oral solution by the end of April.
Melbourne-based Neuren Pharmaceuticals (ASX: NEU) has announced its North American partner Acadia Pharmaceuticals (NASDAQ: ACAD) has received US Food and Drug Administration (FDA) approval for Neuren’s lead candidate Daybue (trofinetide) to treat Rett syndrome in adult and paediatric patients.
The drug is the world’s first and only approved treatment for Rett syndrome and is expected to be available to the US market as an oral solution by the end of April.
Trofinetide works by reducing inflammation in the brain, stopping certain types of cells from becoming overactive and increasing the amount of the naturally-occurring IGF-1 protein.
In studies, participants taking trofinetide saw an improvement across several key domains including social communication, fine motor skills and breathing.
Important milestone
Acadia chief executive officer Steve Davis said the approval is an important milestone for the Rett community.
“As the first FDA-approved drug for the treatment of Rett syndrome, Daybue offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat a diverse and debilitating array of symptoms,” he said.
“We are grateful to all of the Rett syndrome patients, caregivers and clinical investigators who have contributed to making today a reality and we look forward to getting Daybue to patients as quickly as possible.”
Step in the right direction
Neuren chief executive officer Jon Pilcher said the FDA approval is a step in the right direction.
“Many people have shown great determination over the long journey to reach this historic outcome… the greatest has been shown by the Rett syndrome community and I am delighted for them,” he said.
“This is a transformational milestone which places us in a position to make the most of the opportunities ahead, as we work to make a difference in four other neurodevelopmental disorders.”
Neuren is conducting phase two trials of second drug candidate NNZ-2591 for the treatment of Phelan-McDermid, Angelman, Pitt Hopkins and Prader-Willi syndromes.
All programs have been granted orphan drug designation in the US, which recognises urgent unmet clinical needs for rare and serious diseases and provides incentives to encourage development of new therapies.
Licence agreement
In 2018, Neuren and Acadia entered into an exclusive licence agreement for Acadia to develop and commercialise trofinetide for the treatment of Rett syndrome and other indications in the US, Canada and Mexico.
Neuren retains all rights to the drug for countries outside North America and has a fully paid, irrevocable licence for use of all data generated by Acadia in those regions.
Neuren intends to pursue registration and commercialisation of trofinetide through partners and is currently advancing discussions with a number of third parties.
The company does not have the necessary approvals or available drug supply to enable any compassionate use or named patient programs.
Neuren has been granted a rare paediatric disease priority review voucher in connection with the FDA approval, which entitles the company to regulatory review of Daybue in six months rather than the standard 10 months.
Rare disorder
Rett syndrome is a complex and rare neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene which is essential for the normal functioning of nerve cells.
The condition is characterised by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use.
Symptoms may also include development of hand stereotypes (such as hand wringing and clapping) and gait abnormalities.
Rett syndrome is believed to affect up to 9,000 patients in the US, with a diagnosed population of approximately 4,500 patients.