12:00 AM
Jul 23, 2007
 |  BioCentury  |  Product Development

Neurochem sticking it out

When Neurochem Inc. began talks with FDA about the design of a pivotal trial for Kiacta eprodisate in amyloid A amyloidosis, the discussions centered on executing a single Phase II/III trial with a primary composite endpoint. At the time, a single trial gauging renal response seemed to be the best move forward, as AA amyloidosis has a small patient population, well-defined renal response criteria and no treatments that address the underlying disease.

Two FDA approvable letters later, the second of which was issued last week, Neurochem is counting on follow-up data from the Phase II/III trial and data from an open-label extension study to fulfill FDA's latest requests.

What isn't clear is whether Neurochem set its sights too high for the first pivotal trial ever done in the indication, a manifestation of chronic inflammatory disease characterized by deposition of fibrils in organs such as the kidney and liver, or whether FDA's requirements have been a moving target.

Kiacta is an oral small molecule that works by competing with glycosaminoglycans for their binding sites on amyloid fibrils. Glycosaminoglycan binding facilitates the deposit of such fibrils, which leads to the manifestation of the disease.

Kiacta (formerly Fibrillex) missed the primary endpoint in the Phase II/III trial just four months after...

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