Neuren Pharmaceuticals (ASX:NEU) - CEO, Jon Pilcher
CEO, Jon Pilcher
Source: Neuren Pharmaceuticals
Market Herald logo

Subscribe

Be the first with the news that moves the market
  • 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.

NEU by the numbers
More From The Market Herald

" Race Oncology (ASX:RAC) reports $1.8m in cash outflows

Race Oncology (ASX:RAC) reports $1.8 million in cash outflows
Imugene (ASX:IMU) - CEO, Leslie Chong

" Imugene (ASX:IMU) signs clinical trial agreement with Roche for lung cancer

Imugene (ASX:IMU) signs a clinical trial supply agreement with Roche to evaluate the combination of treatments…
Fiji Kava (ASZ:FIJ) - CEO and Managing Director, Dr Anthony Noble

" Fiji Kava (ASX:FIJ) hits quarterly revenue record

Fiji Kava (ASX:FIJ) has tabled a record quarterly revenue of $476,531 for the December quarter, up…