Dr. Mellgård, MD, PhD, Vice President, Global Program Lead, Rare Genetics and Hematology, Takeda, explains the introduction of Adzynma and how it affects congenital thrombotic Thrombocytopenic purpura (cTTP).
Transcription:
Just like the several other products that we have in Takeda, Adzynma is a pure replacement. We are giving the patient what they are missing. These C-T-T-P patients have a congenital deficiency of ADAMTS13 and Adzynma is then a recombinant form of ADAMTS13, and we're just giving that back. Now, I don't want to jump ahead, but I still want to comment that before Adzynma was available to patients, now at least in the US to begin with, the standard of care treatment was plasma for these patients.
Plasma works to some extent. We believe that our study shows that Adzynma works better. The reason for that is that you can only give so much plasma to patients. Normally then you can give maybe 10 or maximum around 15 milliliters per kilogram. A normal person would then get maybe a liter of plasma, body weight, 75, 80 kilos maybe. We know that our supplementation with ADZYNMA gives about four to five times higher exposure of this critical enzyme ADAMTS13.
On top of that, we believe that our study has shown that ADZYNMA has a very beneficial safety profile. There are well-known problems with plasma, and the most common are hypersensitivity or allergic reactions of some form. In some cases, these can be very severe and limit the use of plasma in some patients. The third thing I would like to mention is that plasma infusions can only happen in the hospital. Normally it takes three, four hours, and if you then need to come in to get your infusions every second week or maybe more commonly, it's a significant burden. If you compare it to ADZYNMA, then it's a small volume, 10, maybe 20 milliliters given over a few minutes.
That infusion could also potentially be done in a home setting if the treatment physician saw things. It's a package. I think we are offering something here, which is a package with better efficacy, a very good safety tolerability profile, and convenience. Sometimes convenience can be really, really important for a patient.
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