I’ll start with the not-so-good news – authorization of the Rare...

I’ll start with the not-so-good news – authorization of the Rare Paediatric Disease Priority Review Voucher scheme is set to expire at the end of next month.

The good news is that rare disease organisations have been lobbying politicians to have the scheme reauthorized and legislation has already been introduced into both the US House and Senate to authorize the scheme for another 5 years.

This has happened before and reauthorization was given at the last minute and I am anticipating it will go that way again. I’ll be watching what happens next month.

As stated already, the voucher, which the FDA has the discretion to grant at the time of drug approval, can be sold to another party which is very keen to shorten drug review time for one of its drugs - typically a high-value market drug, especially if it is in close competition with another drug.

Over the past year, PRV sales have typically fetched around US$100m.

Today’s announcement relates to PYC’s second drug, PYC-001 for ODOA. PYC applied for orphan designation of VP-001 for RP11 in 2020 but the FDA at the time wanted animal data which was difficult to supply for that particular condition. At the time, PYC said it might wait until early human clinical data had come in before reapplying.

I am expecting that orphan disease designation and rare paediatric disease priority review for VP-001 will also be applied for, and received, by PYC in the not-too-distant future.

Phelan McDermid syndrome should also be eligible for both designations.

Polycystic kidney disease is likely to also be eligible for orphan disease designation (there is precedent) though I am not so sure that it would be eligible for the PRV scheme.