Interesting speech below by the FDA commissioner a couple of...

Interesting speech below by the FDA commissioner a couple of months ago on rare diseases and the FDA’s desire to work harder and faster to come up with better systems in order to provide approval for these, and also a comment at the end on how the agency lacks the legislative authority to recall faulty drugs quickly enough. Both comments are relevant to the company, the latter, more general one, showing perhaps the rationale for the agency to be putting more focus on risk management and manufacturing controls (see guidance the agency put out on the topic for cell products just before last Christmas, referred to in Phaedrus’ post on the agency’s Matthew Klinker making noises, in an online explanation, about the greater importance of such risk management and product assurance strategies where potency testing is not entirely satisfactory.

All the indications are, to me anyway, that the agency wants to approve. It is not mere coincidence that all these havehttps://www.raps.org/news-and-articles/news-articles/2024/2/califf-new-systems-needed-for-bringing-rare-diseas happened in the sequence that they did.

The link I pasted seems broken - if I don’t manage to correct it, you can google FDA Dr. Cailiff rare diseases systems. The Dec 23 guidance on product assurance strategies can also be brought up by googling. The latter gives me confidence in saying that the agency has moved on from its fixation on the potency assay.

Phaedrus’ comment is interesting because if the company can get the FDA to review such a strategy in the supplementary kids aGVHD information provided for the upcoming meeting, it will surely become a blueprint for the same strategy for other indications under development or whose BLA, as for instance in the case of Rex for HF LVAD, will likely be in front of the FDA for AA in maybe 12-18 months’ time. All this understanding of the subject as seen from the regulator’s viewpoint widens the moat for the products.

Is all this just idle theorising? I don’t think so.

Not financial advice.