Ann: Shareholder Update - Forward View 2020 Milestones, page-11

KZA had a nice move today based on winning rare paediatric disease voucher. I guess we will be applying for one of those eventually, worth 100m or so on their own.

KZA didn’t win a rare disease paediatric voucher today. KZA was awarded rare paediatric disease designation. They are two different things.

Sunset provisions apply to the current rare paediatric disease voucher scheme and for a company to even be in with a chance of getting a rare paediatric disease voucher, it must have received rare paediatric disease designation by 30 September 2020. KZA has scraped in with just weeks to spare.

The actual rare paediatric disease voucher can only be awarded by the FDA once a drug has been approved, and approval is at the discretion of the FDA. But, under the current rules, that approval has to happen prior to September 30, 2022. After that, the FDA may not award any more vouchers.

It is always possible that the scheme may be extended but that would require the agreement of Congress prior to the end of next month.

Even if PYC managed to get designation by the end of next month, it has no hope of having its first drug approved prior to October 2022. And I suspect that KZA is in the same position. The only hope for both companies to receive a rare paediatric disease voucher is if there is an extension to the scheme.

https://www.fda.gov/media/90014/download

https://www.fda.gov/industry/develo...-disease-rpd-designation-and-voucher-programs