A Note About the FDA Process for Acceptance and Review of a BLA.
I was speaking with another shareholder last week who is quite certain that the big action for us will be when the FDA "accepts" the BLA package submitted by IMMU. There was a bit of confusion on my part about the process so I checked into it, and essentially, there are two stages: (1) acceptance or rejection of the package; and (2) notification of "filing review" issues. I'll try to demonstrate the distinctions below, and I'll commence with the latter item.
The FDA recently confirmed (as of March 27, 2018), the procedures for identifying and communicating "review issues," which would cause the Agency to refuse to act upon the BLA application. Here's the policy summary and timeline, and then note the "Definitions" excerpt and its highlighted sentence that will serve as a springboard to my initial point about acceptance or rejection of the package:
POLICY:
• Any filing review issues identified during the filing review will be communicated to the applicant no later than 14 calendar days after the 60-day filing date.
• If the review team does not identify any filing review issues, the applicant will be informed of this fact no later than 14 calendar days after the 60-day filing date.
• This MAPP applies only to original NDA applications, original BLA applications, and original efficacy supplements. It does not apply to labeling supplements that contain clinical data.
DEFINITIONS:
• Filing review issues: Substantive deficiencies or concerns identified by the review team during the initial filing review for an NDA, BLA, or efficacy supplement that appear to have been inadequately addressed in the application and merit particular attention during the review process. These issues may have significant impact on the FDA’s ability to complete the review of the application or approve the application or parts of the application. Filing review issues are distinct from application deficiencies that serve as the basis for a refuse-to-file action. Filing review issues pertain only to applications that have been filed. [emphasis supplied]
Source: fda.gov
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With respect to a "refusal to file," some of us were discussing here what occurred with Celgene back in February, when the FDA issued to the company a Refusal to File letter regarding its New Drug Application (NDA) for multiple sclerosis treatment ozanimod (for application review procedures, a BLA and NDA are apparently treated the same). My search for the regulations turned up only "draft" guidance issued in December of 2017, but from articles I've reviewed, it seems to be the controlling authority, and here's an excerpt:
II. BACKGROUND. The FDA will file an NDA within 60 days of receipt or inform the applicant of the refusal to file. The FDA generally makes filing determinations for BLAs within the same time frame. Filing an application means that the FDA has made a threshold determination that the application is sufficiently complete to permit a substantive review. FDA regulations describe the possibility that the FDA will consider an application to be deficient, on its face, in a way that precludes a complete review (see §§ 314.101(d) and 61 601.2(a)). Specifically, § 314.101(d)(3) provides that the FDA may refuse to file an NDA if: “The NDA . . . is incomplete because it does not on its face contain information required under section 505(b) . . . of the Federal Food, Drug, and Cosmetic Act and § 314.50 . . . .” When this is the case, the FDA may not accept the application for review and may refuse to file it. [emphasis supplied]
Source: fda.gov
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Thus, I agree with the shareholder with whom I was speaking that our watershed event will be whether the BLA is accepted or not. I think that the Company is going to great lengths to ensure that it does not get a "Refusal to File" letter, and if we get no such notice within 60 days after the filing, given the strong data acknowledged thus far and what appears to be an FDA eager to approve SG, we will be in very good shape going forward. This shareholder also strongly believes that once the 60 day period passes and the BLA is thus accepted, the company will likely be an acquisition target. I don't know about that, but if we do indeed not slip the May filing target, we will know a lot by the end of July about the path forward.
That's all I got! MAF
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