WRAPUP 2-U.S. FDA staff backs CSL, GTC blood clot drugs
(Adds company comments, share price, additional product details)
WASHINGTON Jan 7 (Reuters) - Two proposed therapies to treat rare blood disorders appear safe and effective for U.S. approval, including one made from genetically engineered goats, Food and Drug Administration staff reviewers said in documents released on Wednesday.
GTC Biotherapeutics Inc's GTCB.O anti-clotting drug Atryn, made from human proteins inserted into goat milk, and CSL Ltd's (CSL.AX) clot disorder treatment Riastap appeared to work in company studies, the FDA staff said in reports prepared for an advisory panel that meets on Friday.
Both products would need further study after they begin sales, the agency reviewers said.
On Friday, a panel of outside advisers will make a recommendation to the FDA on whether the two therapies are safe and effective. The agency usually takes their advice.
Shares of GTC soared 28 percent to 73 cents on the Nasdaq at midday on Wednesday.
The drug is GTC's first product to be submitted for approval in the United States and is part of a series of anti-clotting drugs that the Framingham, Massachusetts-based company is developing, officials said.
"This is an important tipping point for us," the company's chief executive, Geoff Cox, told Reuters on Tuesday. He added that Atryn, if approved, could bring in between $40 million and $50 million in the next four to five years.
Atryn treats hereditary antithrombin deficiency, in which irregular protein levels can cause excessive clotting. The treatment is made using a human protein produced in milk from genetically engineered goats.
While patients usually take traditional blood thinners such as warfarin, such drugs are not suitable for high-risk surgeries or giving birth, company officials have said.
In its review, the FDA said company data showed Atryn helped prevent clotting in patients and had an "acceptable" level of risks.
GTC is also developing further studies to test the drug in patients at risk for clots in non-hereditary conditions such as coronary bypass surgery, which if approved later by the FDA could also boost sales, Cox said.
To make Atryn, company scientists injected human DNA into goat cells to breed about 200 genetically modified goats at its Massachusetts facility. Females then express the human anti-clotting protein in their milk when they begin lactating after giving birth.
The drug is licensed to Ovation Pharmaceuticals Inc [OVAPH.UL] in the United States.
The other product under FDA review, Riastap, made by the company's CSL Behring unit, is intended for patients with congenital fibrinogen deficiency, which limits the blood's ability to clot.
Company research showed Riastap increased clot firmness, which was "sufficient" to support accelerated approval for drugs for serious diseases, FDA reviewers said. Accelerated approval requires further study after marketing to confirm the product provides meaningful benefits to patients.
The safety of Riastap, derived from human blood plasma, "appears to be acceptable," the FDA reviewers said. The drug is already sold in parts of Europe as Haemocomplettan P.
Australia-based CSL is the world's top maker of plasma products. CSL shares closed down nearly 3.3 percent in Australian trading before the FDA report was released.
Summaries for both drugs were released on the FDA's website at www.fda.gov/ohrms/dockets/ac/09/briefing/2009-4410B1-00-Index.html (Editing by Gerald E. McCormick, Lisa Von Ahn and Matthew Lewis)
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