Friday, September 30, 2022

561.316.3330

Biotechnology News Magazine

FDA grants Orphan Drug Designation for NNZ-2591 to treat Prader-Willi syndrome

Latest Posts

City of Hope to Accelerate Immunotherapy Research & Treatment Innovation with $15 Mil Gift from Ted Schwartz Family

Ted Schwartz, who is now cancer free, achieved complete remission at City of Hope in 2020 with the center's leading CAR T cell therapy after a 16-year battle with lymphoma, provided the gift to City of Hope to advance treatment options that offer better outcomes and quality of life for people living with cancer.

Neurocrine Biosciences Appoints Dr Ingrid Delaet as Chief Regulatory Officer

Prior to joining Neurocrine Biosciences, Dr Ingrid Delaet served as Senior Vice President, Regulatory Affairs at Intercept Pharmaceuticals, which she joined in 2016.

Astrea Bioseparations Introduces Nereus LentiHERO, a Fit-for-purpose Solution for Lentiviral Vector Purification

“We believe that AstreAdept will be a game-changer,” explained Astrea Bioseparations’ CEO Terry Pizzie. “Our approach was to rapidly develop and incorporate this material into the Nereus LentiHERO, a simple, fit-for-purpose device that radically transforms how lentivirus can be purified [in terms of speed, recovery, and efficiency].

At Pack Expo, Schreiner MediPharm to Debut Functional Labels Designed from More Sustainable Materials

Schreiner MediPharm advises he new label concepts are based on existing items in Schreiner MediPharm’s roster of functional labeling solutions.

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.

Latest Posts

Learn More

spot_img

Subscribe

spot_img

Our Sister Publication

Medical Device News Magazine