The FDA has issued another approvable letter – this time to Neurochem for eprodisate (KIACTA(TM)) for the treatment of Amyloid A (AA) amyloidosis. This was not good news on two fronts – first, it was the product’s second approvable letter and, second, it asks for more trial information, though the need for that information may change, pending circumstances. The first approvable letter was sent to the company in August, 2006 and the company submitted a response in October, 2006.
As you will no doubt remember from previous postings on approvable letters, such a letter represents a willingness by the FDA to approve a product, but sets out some conditions for that approval, which can include any combination of labeling changes, more clinical trials, risk management programs or other types of information or data.
According to the company press release on the approvable letter, "the FDA indicates that the Phase II/III clinical trial provided some evidence of the effectiveness of eprodisate (KIACTA™) in the treatment of the renal manifestations of amyloidosis; however, the FDA also indicated that an additional efficacy trial with a target p-value of 0.05 will be necessary before the FDA could approve the investigational product candidate. Further, the approvable letter states that additional submissions, filed by Neurochem as part of its complete response to this approvable letter, may address issues raised in this letter. The FDA has indicated that such additional submissions could persuade the agency to eliminate the requirement for an additional trial."
Another factor involved is that the FDA has asked that a QT clinical study should be submitted as part of a post-approval commitment. While the FDA does have the authority to force such a study it is only a matter of time, and the role of QT has been growing for all new approvals, signalling that companies that suspect a QT issue should try and address it proactively.
As noted in yesterday’s posting, sometimes companies receiving multiple approvable letters are tempted to file an appeal to the FDA with a Formal Dispute Resolution (FDR), but that FDRs have not appeared to be a particularly dynamic way of achieving success. The news sent the stock down this morning.