Shares in Canada’s Aeterna Zentaris and Keryx Biopharmaceuticals of the USA have leapt on the news that regulators have granted orphan drug status to perifosine for the treatment of neuroblastoma.

The US Food and Drug Administration has granted orphan-drug designation to perifosine, a first-in-classoral Akt inhibitor, for neuroblastoma, a cancer of the nervous system affecting mostly children and infants for which there are no approved therapies. The agency grants orphan drug status for diseases affecting fewer than 200,000 patients and the designation provides the drug developer with a seven-year period of US marketing exclusivity.

Neuroblastoma is the most common cancer in infants aged less than one and accounts for about 7% of all paediatric cancers. There are about 650 new cases each year in the USA.

Keryx is Aeterna's partner and licensee for perifosine in the USA, Canada and Mexico. The compound has also been out-licensed by the Quebec City-based firm to Handok Pharmaceuticals Co in South Korea, while retaining rights for the rest of the world.

Perifosine is only in Phase I for neuroblastoma, but is currently in Phase III trials for advanced colorectal cancer and multiple myeloma and has fast-track designation granted by the FDA for both indications. The agency and its European counterpart has granted the compound orphan drug status for the MM indication as well.

The news went down well with investors and Aeterna shares were up 9.7% at C$1.24 each, while Keryx ended the day at $4.11, an increase of 15.5%.