Provectus Biopharmaceuticals Inc (PVCT)

[Leave_the_gun]

Rare Pediatric Disease Designation Priority Review Voucher


"The FDA grants RPDD for diseases that primarily affect children from birth to 18 years old, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases"


Here are the stats for each of the 4 cancer types that POETIC displayed this year for PV-10 at ASCO:

1. 800 new cases of neuroblastoma each year in the United States
2. Ewing sarcoma = 1 case per 1 million per year in the United States.
3. rhabdomyosarcoma = Approximately 250 new cases are diagnosed in the U.S. each year
4. Each year, about 800 to 900 new cases of osteosarcoma are diagnosed in the United States

Notice that every one of them fits within the requirements to get the FDA designation?


On September 30, 2016, the Advancing Hope Act of 2016 (Public Law No: 114-229) amended Section 529 of the FD&C Act. Among the changes, the term "rare pediatric disease" now means a disease that meets each of the following criteria:
A. The disease is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.
B. The disease is rare disease or conditions, within the meaning of Section 526.
The Act changed the language of Subsection (A) from, "The disease primarily affects individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents." The full text of the Advancing Hope Act is available at: https://www.gpo.gov/fdsys/pkg/BILLS-114s1878enr/pdf/BILLS-114s1878enr.pdf

Effective 90 days after the enactment of the Advancing Hope Act of 2016, the sponsor of a rare pediatric disease product application that intends to request a priority review voucher must submit such request in a cover letter to their NDA/BLA submission.
https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/rarepediatricdiseasepriorityvoucherprogram/default.htm


SRPT sold their voucher to Gilead for $125M for a pediatric treatment and this analyst thinks they should have received more money.

https://www.bizjournals.com/boston/news/2017/02/21/sarepta-sells-fda-review-voucher-for-125m-but.html

"analyst Joseph Schwartz said that the price tag was lower than his expectation of $200 million. In recent years, such vouchers have fetched anywhere from $67.5 million to $350 million."

PVCT could be starting 4 (possibly more) P1 trials that each could qualify for a PPDD and thus, a voucher for possibly each one.

Within a one month time frame, the FDA granted two PPDD to Cellectar Biosciences for the same drug treating both neuroblastoma and rhabdomyosarcoma; two of the cancers POETIC just presented on behalf of PV-10 and PVCT at ASCO.

If this is the precedent being set by the FDA, things could start moving very quickly in the near future for PVCT.