One of the requirements in support of Orphan Disease Designation is provision of a scientific rationale relevant to the disease/condition.
The FDA guidance states
Note: The scientific rationale is best supported by human data that show the clinical benefit of using the drug in patients with the rare disease or condition. If clinical data for the drug/active moiety in treating, diagnosing, or preventing the rare disease are available, present the clinical data in this section; preclinical in vitro and in vivo studies should only be briefly mentioned. Clinical data may include preliminary results as well as case reports.
Note: In absence of human data, the request for orphan drug designation may be satisfactorily supported with preclinical data using a relevant animal model for the human disease. Animal toxicology data describing the safety of the drug in animals do not provide efficacy data and are not generally relevant in supporting the scientific rationale.
I suspect that the main reason that PYC applied for Orphan Drug Designation at such an early stage of development was to support its attempt to receive Rare Paediatric Disease Designation by 30 September.
As discussed here before, the Rare Paediatric Disease Voucher Scheme expired on 30 September this year. Under sunset clause provisions, no Rare Paediatric Disease Designation requests were to be accepted after 31 July this year. A 60 day review timeline was guaranteed by the FDA for a Rare Paediatric Disease Designation if the application was accompanied by an Orphan Drug Designation request. Notably, PYC submitted both requests on 31 July.
Clearly, PYC didn’t receive an RPDD by September 30, which may well not matter, because at this point, the RPDD scheme hasn’t been extended by the US Congress.
Had there not been the pressure to receive an RPDD by 30 September (which would have been nice timing prior to an equity raising in October), PYC, imo, would have waited for more data before submitting their Orphan Disease Designation application.
Using ProQR as a guide, QR-110 received its ODD around the time that clinical trials were due to start and QR-1123 received ODD after an IND and fast track had already been received from the FDA.
If PYC now follows ProQR's example, ODD for VP-001 in RP11 might be at least two years off.
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