$PVCT: A Proactive FDA

Ripples become waves. It takes time. It also takes a ripple.

Entitled "A Proactive FDA Is Already Reviewing Sarepta's Muscular Dystrophy Drug (Updated)," Adam Feuerstein recently wrote about the FDA actively reviewing the efficacy and safety of Sarepta Therapeutics' Duchenne muscular dystrophy drug eteplirsen even though the drug has not been formally filed for approval.

In his article, Feuerstein writes "Baird analyst Brian Skorney gets it. "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.""

Feuerstein further writes "Why is this good news, again? Because the FDA could have easily told Sarepta to come back with data from the planned phase III study. That would have told us FDA didn't believe the existing eteplirsen data."

In its equity research update note Neuralstem Receives Phase 2 ALS Trial Approval, Aegis Capital wrote about this company receiving approval from the FDA to begin Phase 2 testing of its neural stem cell line therapeutic, designated NSI-566, in patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

The analysts wrote "FDA Giving Neuralstem What They Wanted. We note that the regulatory agency provided Neuralstem with the approval that the firm requested to begin testing substantially higher doses of NSI-566, which we consider a crucial step forward."

The FDA provides regular updates on submissions and actions taken (within the prescribed time frame) for breakthrough therapy designation (BTD).

When I speak with Pete about the regulatory path, he consistently describes Eric and his team's relationship with the FDA as accommodative.

In regards to Provectus, I think a proactive FDA would:

• Suggest the company consider submitting a request for BTD,
• Grant Provectus BTD for PV-10 for use in the expanded label of recurrent and metastatic melanoma (the focused label being sought for PV-10 under the SPA is metastatic melanoma in Stage III patients),
• Accelerate PV-10's path to market, in a manner similar to granting the drug accelerated approval, and
• Permit the trial design, including progression definition and programmatic element work, the company currently is utilizing in seeking the SPA to be used as a post-marketing study.

"Accommodative" probably works well for now. "Proactive" will be appropriate in Provectus' case (with the benefit of hindsight, of course) whenever the company achieves the above.

Adam's Feuerstein's description of a "proactive FDA" may continue to ripple until the number of Agency's like actions causes a wave of awareness of its seemingly new behavior and assistance to patients, drugs and companies.