Shire Says FDA Permits Vela Use; Reports Phase III Success
August 03 2009 - 3:05AM
Dow Jones News
U.K. pharmaceutical company Shire PLC (SHP.LN) Monday said the
U.S. Food and Drug Administration has agreed to permit doctors to
use an as yet unapproved drug to treat patients with a rare genetic
condition, following a shortage of an approved drug from Genzyme
Corp. (GENZ).
The FDA asked Shire in early July to submit a treatment protocol
for its experimental drug velaglucerase alfa, or vela, because of a
shortage of Genzyme's Cerezyme caused by manufacturing problems. A
treatment protocol permits doctors to use an unapproved drug under
certain circumstances.
Around the same time, Israel's Protalix BioTherapeutics Inc.
(PLX) said the FDA approached it about submitting a treatment
protocol for its experimental drug to treat Gaucher disease, prGCD.
It isn't yet approved for sale either, and a Phase III trial is
underway.
The drugs from Shire and Protalix and the approved treatment
from Genzyme are designed to treat Gaucher disease, a rare genetic
condition.
Shire said Monday the FDA has accepted its submission and
physicians will be able to start using vela to treat Gaucher
patients.
Also Monday, Shire said vela met its goals in a Phase III trial,
one of three such late-stage studies planned. It has begun
submitting vela for regulatory approval on a rolling basis.
Company Web site: www.shire.com
-By Jason Douglas, Dow Jones Newswires; 44-20-7842-9272;
jason.douglas@dowjones.com