Neuren Pharmaceuticals (ASX: NEU) today reported that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to Neuren’s NNZ-2591 for the treatment of Prader-Willi syndrome.
Prader-Willi syndrome (PWS) is a highly debilitating neurodevelopmental disorder, caused by defects in the 15q11-q13 region of chromosome 15. The estimated incidence is 1 in 10,000 – 30,000 males and females across all races and ethnicities. Infants with PWS have very low muscle tone and suffer from feeding difficulties. An unregulated appetite and easy weight gain characterize the later stages of PWS, which can lead to morbid obesity. A range of other problems can include intellectual and learning disabilities, growth hormone deficiency, sleep disturbances, speech difficulties, obsessive-compulsive symptoms, gastrointestinal complications, and difficulty controlling emotions.
Neuren previously announced positive results in the Magel2-null mouse model of Prader-Willi syndrome, in which treatment with NNZ-2591 for 6 weeks normalized fat mass, insulin levels, IGF-1 levels and all behavioural deficits.
Neuren CEO Jon Pilcher commented: “We were excited by the strong pre-clinical efficacy of NNZ-2591, which clearly demonstrated the potential for the mechanism of action to have a positive impact on Prader-Willi syndrome. We are now delighted to receive Orphan Drug designation from the FDA following review of our rationale and data. This underpins the commercial opportunity and follows Orphan Drug designation already granted for Phelan-McDermid, Angelman and Pitt Hopkins syndromes.”
Orphan Drug designation is a special status that the FDA may grant to a drug to treat a rare disease or condition. Amongst other incentives, Orphan Drug designation qualifies the sponsor of the drug for 7 years of marketing exclusivity, plus 6 months if approved for pediatric use, as well as waiver of the prescription drug user fee for a marketing application.