Replies to post #603462 on NorthWest Biotherapeutics Inc (NWBO)

[CrashOverride]

I agree VI.

[underpar71]

That would be a good paragraph to send to DI and simply ask if he can confirm or deny that theory.

[supersteve13]

I was in Chicago first week of June and was assured it’s a rolling application. :)

[alphapuppy]

Thanks Viking this does make the most sense to me.

[dennisdave]

He said that there is not a clear time differential between submission and acceptance in these situations,

yes there is 45 days

[martyDg]

I agree with you.

[hoffmann6383]

Thanks for sharing! This makes sense.

[ilovetech]

Viking - Exactly my thinking in post 603152:

Nickybeans - With the introduction of new expedited review programs, it's feasible that the definition of submission and acceptance may have changed. In other words, suppose a program allows for rolling modular reviews, as part of the entire submission. Once each section has been greenlit for final drafting, and the rest follows suit, then a formal compilation could be "submitted" but pre-approved, which covers both terms submitted and approved upon the regulator's receipt of the final copy. If this scenario is true or feasible, then imo, DI could arguably provide answers in his riddled manner of answering, and still be compliant. IMHO.

ILT

[Nickybeans]

Thanks for this post. I was ripped by certain people yesterday for saying I was in the rolling review camp.

[martyDg]

So there’s no timeframe or number of time to revise the documents? Is it just really up to mhra to tell them that all docs looks here and we acceptable?

[StonkMaster]

Was it your impression that they could be utilizing a rolling review with multiple regulators concurrently? Does/would this include ORBIS?

[TTsr]

Thank you VI for this excellent intel

[skitahoe]

Thanks VI,

The only thing that I am uncertain about is whether they have submitted the information on how they are doing the commercial production of the vaccine.

Gary

[antihama]

Other biotechs do PR rolling review beginning/completion/acceptance. Vertex and Adaptimmune examples below.

Vertex and CRISPR Therapeutics Announce Global exa-cel Regulatory Submissions for Sickle Cell Disease and Beta Thalassemia in 2022
- Exa-cel will be submitted to the U.S. FDA for rolling review beginning in November, with completion of the U.S. submission package in Q1 2023 -
- EMA and MHRA submissions are on track for Q4 2022 -
- Exa-cel granted Fast Track, Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations in the U.S., and Priority Medicines (PRIME) and Orphan Drug designations in Europe -
BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Sep. 27, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that Vertex has concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission by the end of Q1 2023.

Vertex and CRISPR Therapeutics Complete Submission of Rolling Biologics License Applications (BLAs) to the US FDA for exa-cel for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
-EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023-
BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Apr. 3, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced the completion of the rolling Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for the investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The BLAs include requests for Priority Review, which if granted, would shorten the FDA’s review of the application to eight months from the time of submission versus a standard review timeline of 12 months.

FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia
- First CRISPR gene-editing filings to be accepted for review by FDA -
- FDA grants Priority Review for severe sickle cell disease (SCD) and Standard Review for transfusion-dependent beta thalassemia (TDT) –
- PDUFA target action date of December 8, 2023, for SCD and March 30, 2024, for TDT –

And on que 2 months later

BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Jun. 8, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively. Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023.

Adaptimmune Announces Initiation of Biologics License Application (BLA) Submission for Afami-cel, Its First-Gen Engineered TCR T-cell Therapy targeting MAGE-A4, For the Treatment of Synovial Sarcoma
December 23, 2022 1:20pm EST
- Afami-cel has the potential to be the first approved engineered TCR T-cell therapy for a solid tumor -
-Synovial sarcoma has a high unmet medical need and the pivotal trial met its primary endpoint with an overall response rate of ~39% after a single dose of afami-cel -
- Adaptimmune plans to complete its rolling BLA submission for afami-cel in mid-2023; and with RMAT status for synovial sarcoma, the application will be eligible for priority review by the FDA -

Why on earth they wouldn't PR positive news I wouldn't know. They could very well be doing what you're thinking and I wouldn't be too surprised but I'll believe it when I see it.

[FeMike]

Had a long talk last night with a 30+ year veteran in biotech/pharma. He told me that what NWBO is likely doing is a rolling submission that will have questions and answers back and forth between the company and the regulators. When the regulators are satisfied, they will let NWBO know that their application has been accepted. He said that there is not a clear time differential between submission and acceptance in these situations, so he didn’t think that NWBO could PR the submission as they wouldn’t know when that was completed until they got the acceptance of the application.....I briefly explained what kind of drug they were developing and he quickly stated that then it was, in his mind, definitely a rolling submission.

Oh look, Viking with some more great hot takes. He said this one in June of 2023. Totally not bullshit pumping. Definitely listen to this guy.