The US Food and Drug Administration (FDA) has delayed its decision on Soleno Therapeutics investigational therapy for children with Prader-Willi syndrome.
The new decision date for Soleno’s DCCR (diazoxide choline) extended-release tablets is set for 27 March 2025 – three months longer than the previous FDA extended review period.
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The delay comes after the FDA classified recent responses from Soleno as a “major amendment” to its new drug application (NDA), requiring additional time to review. However, the agency has not raised concerns about the drug’s safety, efficacy, or manufacturing.
Prader-Willi syndrome is a rare genetic disorder caused by abnormalities in chromosome 15. Symptoms include low muscle tone, developmental delays, and behavioural challenges. Hyperphagia, or extreme hunger, is one of the most serious and defining characteristics of the disorder and can lead to severe obesity and associated health complications if unmanaged. There are currently no FDA-approved treatments specifically addressing hyperphagia in Prader-Willi syndrome.
The US-based biotech’s NDA is supported by data from the Phase III DESTINY-PWS trial (NCT03440814), which enrolled 127 individuals with genetically confirmed Prader-Willi syndrome. The trial did not demonstrate a statistically significant difference in hyperphagia reduction overall, but Soleno said that the DCCR group showed “nominally significant” reductions in fat mass, and overall improvements in condition as assessed by investigators.
DCCR has been granted breakthrough therapy, fast track, and orphan drug designations in the US, as well as orphan drug designation in the EU. Originally slated for discussion at an FDA advisory committee (AdComm) meeting, the agency decided in October 2024 that such a meeting was unnecessary.
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By GlobalDataDCCR is an extended-release tablet containing a crystalline salt formulation of diazoxide, an activator of the adenosine triphosphate (ATP)-sensitive potassium (KATP) channel. These channels are present in a variety of cells, including pancreatic β cells, and adipocytes. The regulation of these cells can help manage hyperphagia.
The delay represents a setback for Soleno on its first potential approved treatment. Soleno’s stock price decreased by 6.9% from $57.22 at close on 25 November to a low of $53.25 on 26 November. According to GlobalData’s Pharma Intelligence Center, DCCR could generate up to $759m in sales by 2030.
GlobalData is the parent company of Pharmaceutical Technology.
The company is also evaluating DCCR as a treatment for paediatric patients with certain genetic obesities, including SH2B1 deficiency, obesity associated with PCSK1 mutation (rs6232 variant), and SIM1 deficiency. A Phase II trial (NCT05532020) is set to enrol approximately 30 patients aged five years or younger suffering from either of the three genetic obesity disorders.
