FDA Approves Neuren Pharmaceutical's Phase II Trial of NNZ-2591 for Prader-Willi Syndrome

The FDA has approved Neuren Pharmaceuticals’ Phase 2 clinical trial for its NNZ-2591 drug for children with Prader-Willi syndrome, a neurological disorder with no approved treatment options. The trial is part of Neuren’s effort to develop NNZ-2591 for multiple serious neurological disorders that affect early childhood. The open-label, phase two trial will enroll up to 20 children to examine the safety and efficacy of the drug over 13 weeks of treatment, marking the first trials of NNZ2591 in youths. The FDA has granted all four programs orphan drug designation.

Publish Date: 23-01-2023 Source: Neuren Pharmaceutical

Prader-Willi Syndrome (PWS) is a genetic disorder that affects multiple systems in the body. Infants with PWS experience reduced muscle tone, weak suck and feeding difficulties, resulting in poor weight gain and growth. The disease can be caused by paternal deletion of the 15q11-q13 region, maternal disomy, or rare imprinting defects. This results in life-long oral and social skill difficulties and distinctive facial features. Childhood symptoms include short stature, small genitals, excessive appetite, and lack of satiety. Without intervention, this can lead to severe obesity. Other associated endocrine abnormalities include growth hormone deficiency, hypogonadism, hypothyroidism, premature pubarche, and rarely corticotropin deficiency. Cognitive dysfunction is common, ranging from mild to moderate, and is associated with learning disabilities, impaired speech and language development, and emotional control difficulties. The syndrome may also result in comorbidities such as diabetes, sleep-related breathing disorders, gastrointestinal problems, and infections. Early genetic testing and multidisciplinary care, including growth hormone (GH) treatment, can improve the quality of life for individuals with PWS. GH treatment has been shown to stabilize body mass index, improve linear growth and cognitive development, and reduce comorbidities. While full autonomy cannot be reached, GH treatment can help individuals with PWS lead better lives.

In the USA, the incidence of Prader-Willi Syndrome at birth is estimated to be 1/10,00 to 1/30,000 and the estimated prevalence of 10,000 to 20,000 living individuals.

However, the current Prader-Willi Syndrome (PWS) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (RGH-706, ARD-101, Eutropin, etc.) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for Prader-Willi Syndrome (PWS) treatment. The key companies in the advanced development stage are Gedeon Richter Plc., Rhythm Pharmaceuticals, Inc., LG Life Sciences, etc.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Prader-Willi Syndrome (PWS) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.

About Thelansis:

Thelansis specializes in pharmaceutical market outlook and market forecast reports. We published reports across the therapeutic area, including rare / ultra-rare and mainstream indications. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.