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;
jodi.xu@dowjones.com