As the nearly 1,000-page 21st Century Cures Act is implemented in phases, the full impact of the law is just beginning to be realized, and for one provision, the law has created a new program that rewards biopharmas for developing products that the government purchases and often pays to develop.
Under section 3086 of the Cures Act, the FDA must establish a new priority review voucher program for material threat medical countermeasures — or MCMs — which are products that may be used in the event of a potential public health emergency, like an anthrax or ricin attack, or a naturally emerging disease like SARS or other natural disaster.
If a company wins approval for such a material threat MCM, FDA will award the company a priority review voucher that can be used or sold to another sponsor to expedite by four months FDA’s review of another product that would otherwise not receive priority review from the regulator.
The vouchers and expedited reviews are a hot commodity for competitive pharmaceutical companies trying to bring their products to market first — some have already been sold for as much as $350 million.
This is the third PRV program created by Congress — the other two were designed to incentivize the development of new rare pediatric and tropical disease treatments. And although all three programs are distinct, all the vouchers awarded by FDA compete with one another, meaning that generally the value of the vouchers can go down if there more become available for sale.
But what makes this program unique is that the US government — usually via the Biomedical Advanced Research and Development Authority (BARDA), National Institutes of Health and FDA grants — frequently pays for such products to be developed and often is the only purchaser of such products alongside other governments. Obviously, no consumer market exists for material threat medical countermeasures.
FDA says it intends to issue guidance to address MCM-specific issues and the agency has already issued guidance to address questions related to the tropical disease PRV program and the rare pediatric disease PRV program.
No MCM priority vouchers have been awarded by FDA yet, but Soligenix, a company with no FDA-approved products marketed, is developing an MCM vaccine, known as RiVax, to prevent the effects of exposure to the dangerous poison known as ricin.
On Monday, the company announced that NIH’s National Institute of Allergy and Infectious Diseases has exercised an option to fund GMP-compliant RiVax bulk drug substance and finished drug product manufacturing, which is required for future preclinical and clinical safety and efficacy studies.
To date, Soligenix has received $21.2 million from NIAID, and if all contract options are exercised, the company and the University of Texas Southwestern, where the vaccine protein originated, will have received almost $25 million to develop RiVax thanks to a series of grants from both NIAID and FDA. A spokeswoman from Soligenix confirmed to Focus: “Development of heat stable RiVax, which includes manufacture, has been fully funded to date by NIAID.”
If approved by FDA, RiVax, which has already received an orphan drug designation, would likely be purchased by the US Centers for Disease Control for its $7 billion Strategic National Stockpile and the company would win a coveted PRV.
Another example of a product that would have won such an MCM PRV if the program had been created earlier and was also developed with the help of BARDA and NIAID funds is Emergent BioSolutions’ anthrax vaccine, known as BioThrax. Last December, Emergent signed a $911 million contract with CDC to supply the Strategic National Stockpile with almost 30 million doses of the vaccine through 2021.
An Emergent spokesman told Focus that the company does invest in research and development and has four product candidates in its pipeline that could have PRV potential, including a dengue therapeutic in Phase 1, a Zika therapeutic, a broad-spectrum antibiotic being developed for burkholderia pseudomallei and a therapeutic for pan-filoviruses.
First published here. Regulatory Focus is the flagship online publication of the Regulatory Affairs Professionals Society (RAPS), the largest global organization of and for those involved with the regulation of healthcare and related products, including medical devices, pharmaceuticals, biologics and nutritional products. Email email@example.com for more information.
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