A Proactive FDA (and $PVCT)

I first wrote about a proactive FDA in April blog post "$PVCT: A Proactive FDA" and June post "$PVCT is Going for BTD. BTD is Tantamount to Approval." I thought and think this when reading Sarepta Therapeutics' mid-April PR and Adam Feuerstein's "A Proactive FDA Is Already Reviewing Sarepta's Muscular Dystrophy Drug (Updated)" article, and placing Peter's comments about the Provectus' interactions with the FDA over the last 6-12 months (in particular) in this context.

Sarepta wrote what I thought was a cogent PR, such as noting among other things "The FDA has requested that Sarepta provide additional information from the existing eteplirsen dataset to inform a decision on the acceptability of this dataset for a New Drug Application (NDA) filing under the Subpart H Accelerated Approval regulatory pathway."

Feuerstein's piece quotes Baird analyst Brian Skorney: "On the surface it appears that the FDA is requesting information that would normally be part of the NDA review process," he writes in a research note. "This appears to be a bit of a departure from precedent and shows the FDA's willingness to remain flexible (at least publicly) on this review. We believe that the agency is essentially doing the bulk of the review on this application prior to submission and would view a recommendation by the agency to file based on the available data as a very strong indication that it will result in an approval."

He then concludes: "Exactly. Once you get your head around the FDA reviewing a drug that's not officially filed for approval, the Sarepta situation makes more sense."

I think the rumor the FDA suggested/asked/etc. Provectus to submit an application for breakthrough therapy designation ("BTD") is true. Another rumor, which I cannot as yet validate, is that Provectus used Pfizer’s palbociclib BTD application as a guide.

Whether the FDA has indeed been proactive with Provectus is unlikely to be validated until we gain a measure of regulatory clarity, as I do not think management will alter is communications approach to regulatory matters. By proactive I mean the FDA granting BTD (only securing the SPA is insufficient to frame the outcome as proactive).

I came across this "Regulation of immunotherapeutic products for cancer and FDA’s role in product development and clinical evaluation" article by FDA Center for Biologics Evaluation and Research ("CBER") and Center for Drug Evaluation and Research ("CDER") staff.

The abstract is: "Immunotherapeutics include drugs and biologics that render therapeutic benefit by harnessing the power of the immune system. The promise of immune-mediated therapies is target specificity with a consequent reduction in off-target side effects. Recent scientific advances have led to clinical trials of both active and passive immunotherapeutic products that have the potential to convert life-ending diseases into chronic but manageable conditions. Clinical trials investigating immunotherapeutics are ongoing with some trials at advanced stages of development. However, as with many products involving novel mechanisms of action, major regulatory and scientific issues arising with clinical use of immunotherapeutic products remain to be addressed. In this review, we address issues related to different immunotherapeutics and provide recommendations for the characterization and evaluation of these products during various stages of product and clinical development."

I think the FDA's BTD decision translates into either a safety/truncated trial or accelerated approval. Better and best. Time will tell. But, it strikes me the effort and path Provectus has engaged in to prove and elucidate the systemic properties and benefits, as well as the immune system properties and benefits, of local-regional agent PV-10 have been high and far from straight/straight forward. The path does seem like it is coming to some end soon.

It does appear the discussion of systemic proof and benefit has expanded to a discussion of the proof and benefit of the stimulation of the immune system. Moffitt has much more information to convey about PV-10, which we await in the form of a peer-reviewed publication. Provectus will present proof of a local immune effect at a European cancer conference this fall.

Much or a good amount of the Pfizer-Provectus-related activity recently in New York appears to be related to China, and what appears to be an effort by Pfizer to encourage or establish a transaction/relationship between Hisun-Pfizer Pharmaceuticals and China. Again, time will tell, as I think more time is needed here.