LONDON -(Dow Jones)- 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