If both Orphan Drug Designation (ODD) and Rare Paediatric Disease Designation (RPDD) are approved, "18-24 month when VP-001 is commercialized" would still be possible. Bringing US$1-2b of income forward by 2 - 2.5 years would have a huge boost on SP.
Yes it would, but sadly, it’s not about to happen.
The (very short) just under 3 year clinical timeline that the company has indicated for VP-001 factors in the time reduction that might be achieved by combining Phase 2 and 3 and faster FDA review. Neither ODD nor RPDD will offer any further timeline shortcuts.
And even just under 3 years is optimistic when compared with the rapid development timeline of another antisense rare ocular disease drug.
Pro QR is developing its drug, QR-110 (sepofarsen), in Leber's Congenital Amaurosis (LCA) due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene. The drug has received both orphan drug and rare paediatric disease designation.
A Phase 1/2 safety study in 11 patients was commenced in October 2017 and completed in October 2019.
In April 2019, an Efficacy, Safety, Tolerability and Exposure After a Repeat-dose Phase 2/3 study was commenced in 30 participants. The estimated study completion date is December 2021. The company has simply said that it aims to receive approval by 2023.
Even if sepofarsen receives approval by October 2022, the process from IND will have taken 5 years.
I also beg to differ on your statement that “the latest estimated timeline” for generation of revenue from PYC-001 in Diabetic Retinopathy is 2025/6. Not only has this not been suggested by the company, it is frankly fanciful, imo.
Diabetic Retinopathy is a much more prevalent condition and therefore will not qualify as an orphan drug or be permitted the small trials seen in orphan drug development. The company anticipates a human safety (Phase 1) trial in 2024. Trials in other DR drugs suggests the following Phase 2 and 3 trials that will be required have hundreds of participants and take at least 5 years. Then allow at least a further 12 months for NDA preparation and FDA review.
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