FDA staffers endorse transgenic anti-clotting drug

The field of transgenics--developing therapies in genetically engineered animals--just got a big boost from staffers at the FDA. Regulators said that Framingham, MA-based GTC BioTherapeutics's drug ATryn [1], an anti-clotting treatment produced in goats, appeared to be safe and effective. The recommendation was released in documents made available ahead of an expert advisory committee meeting on Friday.

"The safety profile appears to be acceptable," FDA staff said, according to a report in Xconomy, and "ATryn at the recommended dose is effective" in patients with a hereditary deficiency of the anti-clotting protein.

GTC is one of a handful of companies out to demonstrate that it can produce a less-expensive stream of therapeutics from transgenic animals, harvesting drugs through their milk. A regulatory win here would be a first for the field and help open the door to more such therapeutics. It would also be a huge win for GTC. As Xconomy notes, its stock is trading at 57 cents and it badly needs an FDA approval. ATryn is already being marketed in Europe.

- check out the report [2] from Xconomy

ALSO: FDA staff also backed CSL's Riastap, a clot disorder therapy, saying it too appeared safe and effective. The drug is designed to treat patients with congenital fibrinogen deficiency. Report [3]

Related Articles:
GTC shares jump on $257M ATryn pact [1]
GTC announces positive ATryn data [4]
GTC inks new transgenics collaboration deal [5]
Regulators focusing on transgenic animals [6]