Agree that MSB intends that it have that effect. CEO Itescu and the exec management have held the line brilliantly (IMO) thus far - I don't for a minute think they'll be giving away any critical opportunities to secure dominant competitive advantage through potency and other assays which MSB has been developing (with or without its CDMOs/ CMOs). As you say, a 'MOAt', but one which could be at least as wide as 'the Ditch', in the right hands. The magic word for me in this space isn't actually "
appropriate" - it's "
proprietary", especially in relation to healthcare methodologies and technologies. More about that in other threads.
But giving the FDA's position a quick once-over, I don't see anything at all to be concerned about with CEO Itescu's announcement, as quoted by
@nosluggos above. In fact, quite the opposite.
In
Guidance for Industry: Potency Tests for Cellular etc Products, Jan. 2011 (see
link), the FDA explains the concept of 'appropriate' potency testing in quite some detail (e.g. p.2):
"
Potency is defined as “the specific ability or capacity of the product, as indicated by appropriate laboratory tests or by adequately controlled clinical data obtained through the administration of the product in the manner intended, to effect a given result.” (21 CFR 600.3(s)). Strength is defined as “[t]he potency, that is, the therapeutic activity of the drug product as indicated by appropriate laboratory tests or by adequately developed and controlled clinical data. . . .” (21 CFR 210.3(b)(16)). Regulations require that “[t]ests for potency shall consist of either in vitro or in vivo tests, or both, which have been specifically designed for each product so as to indicate its potency in a manner adequate to satisfy the interpretation of potency given by the definition in § 600.3(s) of this chapter.” (21 CFR 610.10).". (underlining & bolding added for emphasis)
Note that the FDA equates strength with potency. But, as I see it, for MSB the most relevant & important references to 'appropriate' assays is on p.3 of the Guidance, viz:
"
FDA regulations allow for considerable flexibility in determining the appropriate measurement(s) of potency for each product. Potency is determined based on individual product attributes; therefore, the adequacy of potency tests is evaluated on a case-by-case basis. However, all potency assays used for release testing of licensed biological drug productsmust comply with applicable biologics and CGMP regulations including:...." (underlining & bolding added for emphasis)
IMO MSB is 'chafing at the bit' to sit down with OTAT and show that MSB's new potency assay(s) is/are both:
1. appropriate, based on remestemcel-l's individual product attributes as verified by recent biomarker results/ analysis; &
2. compliant with cGMP regulations for use as a release assay.
In this manner, MSB will be able to knockout
at least 2 birds with just 1 stone i.e. add a new assay(s) onto the existing product matrix assay framework (endorsed by FDA during the October 2020 ODAC meeting) which both:
- satisfies the requirements of 'appropriate' potency testing for the cellular product, AND
- meets OTAT's/ CBER's requirements for cGMP compliance on release of all of the existing remestemcel-l product being held in the US as finished product. So, the entire existing remestemcel-l inventory can be tested pre-release & shift instantaneously from the expense side of the financial accounts to the current assets side of the MSB balance sheet when CBER finally approve the licensure.
I don't know how much flexibility MSB will actually need to request of OTAT when they do meet. I suspect, without having any direct knowledge, tha both OTAT and CBER officers are now being kept in regular, confidential contact by MSB leading up to the meeting. But this has to be a great time to be a holder, IMO.
Exciting, eh?
(20min delay)
