Alright, I have gone through
@Wilba32's table of orphan drug reviews.
Because we all love data, have a look at the table below regarding review times.
Data characteristics: - 56 Orphan drug submissions have been reviewed so far this year
- 20 NDAs (3 Label Extensions, 17 First Cycles), 36 BLAs (17 Label Extensions, 18 First Cycles, 1 Second Cycle)
- 34 First cycle applications; 1 2nd cycle; 20 label extensions
Summary:- 6 CRLs (10.7%) (5 BLAs, 1 NDA), 49 approvals (~87.5%)
- ~7 submissions were given a result 1 week early (5 BLAs, 2 NDAs,) (~50/50 split for First Cycle and Label Extensions) (1 CRL, 6 approvals)
- 1 submission was given a result 3 weeks early (NDA) (First Cycle) (CRL)
- 5 submissions were given a result pretty much bang on 4 weeks early (4 BLAs, 1 NDA) (2 First Cycle, 3 Label Extensions) (1 CRL, 4 approvals)
- 1 submission was given a result 74 days early = ~2.5 months (BLA) (Label Extension) (Approval)
Analysis:There seems to be
pretty consistent patterns to expedited review,
>10% of applications received expedited reviews, 50/50 between first cycle and label extension submissions.
We can align these findings with what we know about PDUFA review processes.
Most early responses fall bang on 28 days early, which lines up with the removal of the late cycle meeting (mid-cycle for us ~8th October, late cycle by ~8th of November per
FDA guidance). The same guidance says that the late cycle meeting may be deemed redundant, which will be determined at a pre-submission meeting, but subject to change if necessary. This scenario would have us receive a response on the
8th of December (Aus time).
The 74-day response I find really interesting because that is approximately 2 weeks after where a typical mid-cycle review would take place, which is when the FDA guidances says that the minutes for the mid-cycle review must be in by.
So, I suspect that the
best-case scenario for approval is the 22nd of October, confirming my earlier guess for best-case scenario. However, based on what we see in the table, I suspect the
next most likely option is the 8th of December.
For and Against:The FDA has seen literally everything in this submission before, with the exception of whatever minor supplementary data MSB has supplied. The Adcom is done, the manufacturing site visit is passed, they are happy with the potency assays, they are happy with the trial, and they are happy with the efficacy and safety. Logically I feel like this should expedite the process a lot.
The against, well we'll be the first off-the-shelf allogeneic cell therapy with this mechanism of action, so I suspect that takes some serious review - but hopefully all that has been done over the previous reviews and discussions.
As I say, I really doubt (and hope) they would not have submitted the 3rd time without really really clear communication from the FDA.
(20min delay)