Prana Biotechnology (ASX: PBT) has today announced the US Food and Drug Administration (FDA) has granted Orphan Drug designation to PBT2 for the treatment of Huntington Disease.
Orphan drug designation is granted by the FDA to promote the development of drugs for diseases affecting less than 200,000 people in the United States. Orphan drug designation entitles Prana to seven years of market exclusivity for the use of PBT2 in the treatment of Huntington disease; protocol assistance by the FDA to optimize drug development in the preparation of a dossier that will meet regulatory requirements; and reduced fees associated with applying for market approval.
The company will also be applying for Orphan Drug designation in Europe and other jurisdictions.
Huntington disease is a neurodegenerative genetic disorderthat affects muscle coordination and leads to cognitive decline and behavioral symptoms. More than 30,000 people in the US have Huntington disease and there are currently no treatments for the cognition or executive function symptoms of the disease.
Prana announced in February 2014 that its lead MPAC (Metal Protein Attenuating Compound) PBT2 had met its primary end point of safety and tolerability, and improved measures of cognitive performance – a secondary endpoint in its Reach2HD Phase 2 clinical trial involving 109 people with Huntington disease.
“The FDA’s decision to grant Orphan Drug designation is a reflection of the high unmet need for neurological drugs that can slow, halt or improve the decline of cognition and allow sufferers to have a better quality of life,” said Prana Chairman and CEO Geoffrey Kempler.
“We are very pleased to have received Orphan Drug designation from the FDA, which is a highly important input to determining the commercialisation pathway for PBT2 for the treatment of Huntington disease, and to bring to market as quickly as possible an improved treatment for people suffering from Huntington disease.”
Prana is preparing its Post Phase 2 Trial dossier for submission to the FDA to commence discussions on the next development steps for PBT2.