- Neuren Pharmaceuticals (NEU) is granted orphan drug designation to treat neurodevelopmental disorder, Prader-Willi syndrome
- Neuren previously returned positive results from a trial using a mouse model of Prader-Willi syndrome, which saw a normalisation in fat mass, insulin levels and behavioural deficits after six weeks of treatment
- The designation follows the company’s previous grants for PhelanMcDermid, Angelman and Pitt Hopkins syndromes
- Neuren Pharmaceuticals was up 12 per cent, trading at $2.52 at 2:00 pm AEST
Neuren Pharmaceuticals (NEU) has been granted orphan drug designation to treat Prader-Willi syndrome.
Orphan Drug Designation is a status that grants a drug to treat a rare disease or condition. It also qualifies the drug to be sponsored for seven years of marketing exclusivity.
The US Food and Drug Administration (FDA) granted the designation to Neuren’s NNZ-2591 for treating Prader-Willi, a neurodevelopmental disorder.
Infants with the disease have low muscle tone and suffer from feeding difficulties, and later develop a range of intellectual and learning disabilities, growth hormone deficiency, sleep disturbances and speech difficulties, along with constant hunger and difficulty controlling emotions.
Neuren previously returned positive results from a trial using a mouse model of Prader-Willi syndrome, which saw treatment for six weeks normalise fat mass, insulin levels and all behavioural deficits.
Neuren CEO Jon Pilcher says the company is excited by strong pre-clincial efficacy of the treatment.
“We are now delighted to receive Orphan Drug designation from the FDA following review of our rationale and data,” said Jon Pilcher.
“This underpins the commercial opportunity and follows Orphan Drug designation already granted for PhelanMcDermid, Angelman and Pitt Hopkins syndromes.”
Neuren Pharmaceuticals was up 12 per cent, trading at $2.52 at 2:00 pm AEST.