Keryx Biopharmaceuticals Inc. (KERX) said the U.S. Food and Drug Administration has given orphan-drug designation to perifosine, a treatment for cancer including neuroblastoma, or cancer of the nervous system in infants.
Shares of the biopharmaceutical company jumped 13% to $4.04 in recent trading, while U.S.-traded shares of Keryx's Canadian partner Aeterna Zentaris Inc. (AEZ.T, AEZS) were recently up 10% to $1.20.
The designation was announced three months after the drug received fast-track status, which authorizes an expedited review for drugs that treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
"The Orphan Drug designation is an important component of our development plan for perifosine in neuroblastoma, an indication where no FDA-approved therapies currently exist," said Chief Executive Ron Bentsur.
The Orphan Drug Act provides incentives to create therapies for so-called orphan diseases--those that affect fewer than 200,000 Americans. There are about 7,000 such maladies, most of them serious, that have few or no drugs to treat them. Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including seven years' marketing exclusivity and tax breaks.
Bentsur said the company is exploring the next steps for the development, which "we hope, ultimately, could provide a new treatment option for children and infants" suffering with the illnesses.
Perifosine also is in Phase 3 clinical trial for treating refractory advanced colon cancer and multiple myeloma, as well as in Phase 1 and Phase 2 trials for several other tumor types.
-By Jodi Xu, Dow Jones Newswires; 212-416-3037; firstname.lastname@example.org