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The PharmaTell team is catching up with current events in Pharma after the Quarterly Reporting Season. I’m touching on a couple here and will be back to regular updates soon!
Hemgenix for Hemophilia B is Approved!
I am so excited to see that the first Gene Therapy for Hem B is approved. After working in this area for many years, I know just how much this condition affects the boys/men, their families, and their communities. This can be a game changer for many patients. Seeing a long-term treatment approved is something many thought would never happen. Check out some of the trial data and market thoughts about the CSL/UniQure therapy [and the pricing] here and here .
This approval was also good news for BioMarin as they announced that the FDA no longer plans to hold an advisory committee meeting to review its BLA for Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A. They are expecting feedback on their BLA on 31 March 2023 and are preparing for the launch. Some details can be found here.
ImmunoGen receives approval for Elahere
ImmunoGen received accelerated approval from the FDA for Elahere (mirvetuximab soravtansine) for the treatment of adult patients with folate receptor alpha (FRα)-high, platinum-resistant ovarian cancer, who have received one to three prior systemic therapies. Elahere is the first FDA-approved antibody-drug conjugate for platinum-resistant ovarian cancer. More details here.
UCB Re-Submits Bimzelx in the US
Remember in May 2022 UCB received a CRL for manufacturing issues that delayed approval. On November 22, 2022 UCB announced the re-submission of the BLA to the FDA. The FDA is expected to determine whether the resubmission constitutes a complete response, and if so, the classification of the re-submission will be communicated. More here and here
GSK Takes Another Hit
GSK announced that it will stop selling their rrMM Blenrep in the US. This follows the recent announcement that the drug had failed the primary endpoint of showing better efficacy that current treatment. They will continue the DREAMM trial program ongoing in the US and work with the FDA on a path forward for the product. More details here and here.
Tip of the Week
We are now including English transcripts of many of the earnings calls, in the database (you can use your own translation service to convert to your language). Search under company name and add the keyword transcript in the search box. A sample transcript can be found here. Please note that there are other transcripts available in the database – so feel free to do a general search for Transcripts and see what else might be useful to you. We hope these are helpful – let us know if there is a specific company you are interested in and we will see if we can locate a transcript.
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