Replies to post #583237 on NorthWest Biotherapeutics Inc (NWBO)

[aesop1]

Yeah but in that article you see in Europe it is tougher to get reimbursement for these expensive gene therapy drugs. BLUe got approved for the same indication in Europe but failed to gain reimbursement. This is why the US is the king of revenue.

[flipper44]

Excellent find Hoffman.

[dstock07734]

hoff,
Great post.
UK has to move faster probably because of urgency in cancer care.

https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(22)00754-9/fulltext

Here is the paper in case anyone has interest.

[Lykiri]

Thanks hoffmann6883.

EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023

-

In Europe, the Marketing Authorization Applications (MAAs) for exa-cel were submitted in December 2022 and validated by the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) in January 2023. In the EU, exa-cel has been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the EMA, for both SCD and TDT. In the U.K., exa-cel has also been granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA.

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.

https://news.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-complete-submission-rolling

[Springbok80]

Home
Genomics Research
Product News
Vertex and CRISPR Therapeutics Announce MHRA Marketing Authorisation Application Validation for CRISPR/Cas9 Gene-Edited Therapy
Product News
Published: February 6, 2023

Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics (NASDAQ: CRSP) have announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has validated the Marketing Authorisation Application (MAA) of exa-cel for the treatment of sickle cell disease (SCD) and transfusion dependent beta thalassaemia (TDT) in Great Britain. The submission is supported by two global Phase 3 studies investigating exa-cel as a potential one-time therapy for people with SCD or TDT.

“Today marks a significant milestone in our efforts to bring a new one-time therapy to people living with sickle cell disease or transfusion-dependent beta thalassaemia,” said Nia Tatsis Ph.D., Executive Vice President, Chief Regulatory and Quality Officer. “We look forward to working with the MHRA on our application, the first for a CRISPR-based therapy for a genetic disease.”

Vertex has requested Orphan Drug Designation as part of the MAA. (Interesting, NWBO has Orphan status for DCVAX-L if I am not mistaken)