Ann: Investor Call - 8am AWST - Wednesday 12 August 2020, page-16

CMO, May Orfali, mentioned that applications for both orphan drug designation (ODD) and rare paediatric disease designation (RPDD) were submitted on 31 July and that the company expected to hear back from the FDA before the end of September.

Orphan Disease Designation

Historically, approximately two thirds of ODD requests have been granted by the FDA.

The FDA aims to answer ODD requests within 120 days.

The two main criteria that need to be met for ODD are:

• that the drug must be for a disease/condition that affects <200,000 persons in the U.S. and
• that the drug must show promise for the prevention, diagnosis, or treatment of the rare disease or condition

The three main financial incentives attached to ODD are:

• Tax credits up to 25% of qualified clinical trials costs
• Waiver of FDA User Fees (~US$3m)
• Eligibility to receive 7-years of marketing exclusivity

Rare Paediatric Disease Designation

Historically, approximately half of RPDD requests have been granted by the FDA, although in the most recent years for which data has been published, 70-80% of requests have been granted.

The FDA aims to answer RPDD requests within 60 days.

The two main criteria that need to be met for rare paediatric disease designation are:

• that the drug must be for a disease/condition that affects <200,000 persons in the U.S. and
• that the disease is serious or life-threatening disease and primarily affects individuals aged from birth to 18 years

The main incentive attached to RPDD is that the drug sponsor becomes eligible to receive a Priority Review Voucher (PRV). The PRV is a transferrable voucher that allows the bearer to receive priority review for any future product. Products with priority review can expect an FDA approval decision within 6 months of NDA submission, instead of the usual 10 months.

RPDD isn’t required, nor is it sufficient, to receive a PRV, but requesting RPDD in advance will expedite a sponsor's future request for a PRV. A PRV can only be granted if clinical trial data supporting the NDA come from studies in a paediatric population.

Although the PRV can be redeemed for the next drug in the drug sponsor’s pipeline, the main perceived value of the PRV comes from the right to sell the voucher to another company which is willing to pay a significant sum to expedite FDA review of a particular drug. To date, PRVs have been sold for amounts ranging from US$67 - $350 million.

The PRV scheme expires on 30 September 2020. The FDA has said that it can't commit to responding by this deadline for any applications received after July 31, 2020 - hence, PYC has just scraped in!  However, even if a drug does receive RPDD by 30 September, 2020 it must also meet a further deadline of gaining PRV approval no later than 30 September, 2022. Of note is that PRV approval can only be granted after drug approval has been received.

There remains a possibility that the Rare Paediatric Disease PRV scheme may be extended (it has been before) but this will require legislative action by the U.S. Government.

https://www.fda.gov/media/135236/download

https://www.raps.org/news-and-artic...e-pediatric-disease-prvs-fda-updates-guidance

https://www.gao.gov/assets/710/704207.pdf