Short action commenced again once Class II was designated and a 6 month PDUFA date set. Their strategy is two fold- one , another CRL will be issued or two- another capital raise coming , after the next AGM.
With Anjo Roch's commentary ( thankyou) -
Some takeaways from Silviu- FDA PDUFA: We think approval will be ahead of 7th Jan 2025 (PDUFA() . Then we launch Adult trial.
- Assays :We think we have met the criteria : In dialogue with FDA
We know that, "after additional consideration" there was a reversal on the hold on the BLA resubmission by the FDA.
that the company went back to their original strategy.
that new potency assay data was to be generated from the 001 trial and provided to the FDA.
that data was requested at a Type A in 2023, delivered at the start of 2024
that a potency assay had to be agreed upon prior to commencing an adult trial
that the Australian had Silviu quoting the FDA " you've got enough to get approved for children"
that Anjo Roch's summary has Silviu stating - we think we have met the criteria : in dialogue with FDA
that there is no need for another Adcom
that efficacy is no longer questioned in the CRL
that Lonza's manufacturing facility and processes have passed an FDA inspection
that FDA clarified that the key remaining issue for pediatric approval is providing further evidence that the potency
assay will assure the consistent efficacy of commercial product.
that the first quarter 2024 meeting with the FDA was to be regarding potency assay data re the pediatric BLA
that the Australian quoted Silviu re above- " and instead what they said was " no , we've thought this through,
and you've convinced us that the clinical data are very strong".
that the refiled
BLA contains only items relating to the CMC that are that are new and responsive to the CRL.
My opinion- 001 has been ticked off as an adequate and well controlled trial.
- Ryoncil works - in both kids and adults- unmet need in both- (adults failed Rux)
- The adult results, n51, and potency assay data have been a key part of establishing consistent product efficacy of Ryoncil, without it we could not get approval for sale of the already manufactured product.
- Cash is being conserved through salary decreases, slow CLBP enrolment and Adult SR aGvHD postponement.
- I think the shorts have this one wrong timewise- the resubmission won't take 6 months to review- too much of the process has already been ticked off. Leaves them with expecting another CRL.
- Leaves the board thinking they have met the criteria
- Leaves me thinking we are in pretty good shape
Reg
(20min delay)