Neurochem will respond to a second FDA "approvable" letter for the experimental Amyloid A amyloidosis therapy Kiacta (eprodisate) in about two months, the firm told "The Pink Sheet" DAILY.

In the letter, FDA indicated that a Phase II/III pivotal trial provided some evidence of the effectiveness of Kiacta in the treatment of the renal manifestations of amyloidosis, Neurochem announced July 18.

However, the agency also requested "an additional trial ... at a p-level of 0.05," Neurochem VP-Corporate Communications Lise Hebert told "The Pink Sheet" DAILY.

"At the same time, we were encouraged by the FDA to submit ... a consolidated report on the clinical efficacy and the associated statistics with a safety update. It's going to include new information, as we still have patients ongoing into the extension study," Hebert said. FDA also requested pharmacokinetic study results.

The Phase II/III trial showed drug activity (p = <0.025) but="" failed="" to="" meet="" fda's="" pre-specified="" criterion="" for="" statistical="" significance="" of="" p=""><0.01. findings="" from="" the="" study="" were="" published="" in="" the="" june="" 7="" new="" england="" journal="" of="">

Currently, there is no approved treatment for AA amyloidosis. FDA granted Kiacta fast track status in 2004, and it received orphan drug designation in 1999. FDA accepted the candidate as a rolling NDA under the Continuous Marketing Application Pilot 1 and 2 programs (¹ Also see "Neurochem Gets FDA Priority Review For AA Amyloidosis Drug" - Pink Sheet, 19 April, 2006.).

Laval, Quebec-based Neurochem received its first "approvable" letter in August 2006 asking for further information and saying that it would likely require additional efficacy trials with a 0.05 p-value. In February, the firm submitted a "comprehensive summary of data and analysis reflecting our ongoing discussions with the agency," that included extended clinical results and retrieved data, Neurochem said (² Also see "Neurochem/J&J’s Kiacta Action Date Extended To July 16" - Pink Sheet, 11 April, 2007.).

Despite the second letter, Rodman and Renshaw analyst Elemer Piros predicted in a July 18 note that the firm has "a 75 percent chance of obtaining regulatory approval" for Kiacta.

The European Medicines Agency accepted Neurochem's marketing authorization application for eprodisate for review in September 2006. Hebert said the firm expects a ruling from EMEA by the end of 2007 or beginning of 2008 at the earliest.

Neurochem has an exclusive collaboration and distribution agreement for Kiacta with Johnson & Johnson subsidiary Centocor.

Piros reported that the firm has said it would discontinue development of the candidate if it could not get FDA approval without another Phase III clinical trial. When asked to comment, Hebert said the firm hopes to meet FDA's requirements with existing data.

-Pamela Taulbee (p.taulbee@elsevier.com)

Neurochem Gets Second FDA “Approvable” Letter For Kiacta

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