GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Given that the safety of ATryn is well-established in several clinical trials (including U.S. trials in the CABG indication from the late 1990s), the FDA agreed to a single phase-3 trial with a control arm as the requirement for a BLA submission in the HD indication.

There is no Fast Track designation for this program and it’s unlikely that there will be such a designation in the future because Thrombate from Talecris nominally meets the medical need for the small HD market. (In practice, Thrombate has not met this medical need due to chronic supply shortages, but that’s a separate issue.)

More important than Fast Track is Priority Review, which shortens the FDA review time by four months relative to a standard review. It’s possible that ATryn could get this, but the company has conservatively guided investors to expect a standard review.