NorthWest Biotherapeutics Inc (NWBO)

[hoffmann6383]

Regulatory Path Comparison: Vertex Therapeutics’ (Market Cap ~$82B) Exagamglogene Autotemcel v. Northwest Biotherapeutics’ DCVax-L

Why? Vertex is a big pharmaceutical company with a novel treatment that is blazing the same path NWBO appears to be taking. It's not just NWBO that decided to strategically progress with the MHRA before the FDA.

Vertex Therapeutics (VRTX)

Exagamglogene Autotemcel (exa-cel): novel CRISPR based gene editing therapy
Regulatory Submission Order: UK/EU followed by FDA

exa-cel in the USA

FDA Submission process: Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, Rare Pediatric Disease Designation
BLA Submission date: April 3, 2023
BLA Acceptance date: ?
BLA Approval date: ?

exa-cel in the UK

MHRA submission process: Innovative Passport and Innovative Licensing and Access Pathway (ILAP)
MAA Submission date: December 2022
MAA Acceptance date: January 2023
MAA Approval date: ?

PIP submitted: 10-18-21
PIP Procedure started: 5-4-22
Final Decision Letter: 6-17-22
PIP Deferral Granted: Yes
TOTAL PIP TIMELINE: 242 Days
https://cms.mhra.gov.uk/system/files/2022-07/mhra-100266-pip01-21.pdf

NICE Progress: 1-19-23 to 2-16-23 -> “Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators”
NICE Project Documents: Draft Matrix Post Referral & Draft Scope Post Referral
https://www.nice.org.uk/guidance/indevelopment/gid-ta11250/documents

exa-cel in the EU (not going into detail here)

EMA submission process: Orphan Drug Designation, Priority Medicines (PRIME)

Additional Sources: https://www.fiercepharma.com/marketing/vertex-teases-launch-plan-first-crispr-gene-editing-therapy-ahead-fda-decision & https://news.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-complete-submission-rolling


Northwest Biotherapeutics (NWBO)

DCVax-L (Murcidencel): novel autologous dendritic cell vaccine
Regulatory Submission Order: Unknown (signs point towards UK 1st)

DCVax-L in the USA

FDA Submission process: Orphan Designation / Unknown (Real Time Oncology Review, imo)
BLA Submission date: ?
BLA Acceptance date: ?
BLA Approval date: ?

DCVax-L in the UK

MHRA Submission process: Orphan Designation / Unknown (ILAP, imo)
MAA Submission date: ?
MAA Acceptance date: ?
MAA Approval date: ?

PIP submitted: 2-21-22
PIP Procedure started: 3-28-22
PIP Final Decision Letter: 8-16-22
PIP Deferral Granted: Yes
TOTAL PIP TIMELINE: 176 Days
https://cms.mhra.gov.uk/system/files/2022-09/mhra-100409-pip01-21.pdf

NICE Progress: 12-23-22 -> “NICE continues to liaise with the company regarding next steps for this technology appraisal and will provide updates in due course”
NICE Project Documents: Final Scope, Equality Impact Assessment, Final Matrix (note: these all probably need to be updated and are from ~2018)
https://www.nice.org.uk/guidance/indevelopment/gid-ta10143/documents

Thoughts: Going UK 1st for RA approval seems to be gaining traction, even with large pharmaceutical companies. A good comparison of timelines and processes can be found in VRTX’s exa-cel and NWBO’s DCVax-L. Both are novel treatments. Both are, imo, going for ILAP in the UK. Both seem to be months ahead with the MHRA's regulatory path as compared to the FDA.

^^ Something to follow as VRTX & NWBO continue through the RA process.