Actually, JB the Head of the FDA appears more interested in durability of response ….and Mesoblast has just provided them with some excellent data.
PeterMarks:” Indeed, one of the big questions for gene therapies is the durability of the treatments—that is, will these treatments work for a few months, a few years, or provide a functional cure? Patients expect to know how long their treatment will work. Because it is relevant for benefit–risk considerations, from a regulatory perspective we want to know this information as well. We also understand that durability will be an important consideration for payers as they conduct their health economic analyses. So, I think the expectations are set that manufacturers will need to gather these data over the long term. However, this doesn’t imply regulatory approvals will be delayed for decades until we have long-term durability data.“
Please spare us your attempt at empathy. You are obviously clueless about the cost of using Jakafi per Complete Response which is the only currently approved therapy for adult sr aGVHD …Don’t forget the conclusion of the Reach2 trials showing that Jakafi on average delayed mortality over Best Available Therapy by only 3 months..and showed very unpromising results in the most severe patient categories where it’s efficacy is marginal. You have now seen the 4 year mortality data for Remestemcel and I should not have to spell out to you the Cost Economic Analysis (CEA). If you had studied the current costs of paediatric intensive care you would know that billing costs regularly exceed over $500k for the more acute grades of refractory aGVHD….and which invariably result in the death of an innocent child.
You have ignored the real world evidence shown in Kurtzburg’s EAP for treatmentrefractory patients and continue to rely on your main assumption that regulatory authorities are somehow far more advanced in their research than commercial organisations spending substantially greater sums of money. In my experience that is seldom the case.
Let me speak plainly. Compared to Kurtzberg you know nothing . The latest data linking in vitro potency assays of IL2R to in vivo outcomes clearly shows that Mesoblast has achieved astonishing survival data in the “non responders” with MAP scores exceeding 0.29. You cannot dismiss that. OP
Please do not rely on the opinions or facts expressed in the above post when making an investment decision.
MSB Price at posting:
94.0¢ Sentiment: Buy Disclosure: Held
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