It has finally happened….. the decades’ long wait for the approval of the holy grail in hemophilia treatment….. a gene therapy that replaces the need for frequent blood factor infusions.
BUT….. the approval of this new ‘cure’ needs some explaining….. including why the new therapy was approved with….. count ‘em….. 39 NDCs.
First, this new one-and-done therapy is called Hemgenix (etranacogene dezaparvovec-drlb) and was developed by CSL Behring.
Second, Hemgenix is indicated only for Hemophilia Type B (congenital Factor IX deficiency).
Type B represents 8,000 individuals in the US vs. Type A (congenital Factor VIII deficiency) at over 25,000.
CSL trials showed that 94% of individuals with Type B had a positive response and realized a 54% reduction in the annual bleed rate…. (not too shabby but far from a total cure).
Third, is the little sniggly thing related to cost.
Hemgenix will hit the market at a sizzling $3.5 million making it the world’s most expensive drug. That may stall access for patients as payers struggle to deal with the expense burden.
Fourth, here is what you’ve been waiting for……
Hemgenix was approved with 39 NDCs.
It is available in multi-vial kits providing 100mL – 480 mL (10mL per vial) as Hemgenix requires weight-based dosing….. hence the 39 unique NDCs.
It remains to be seen how quickly (if?) Hemgenix will see strong uptake. Individuals with hemophilia are very cautious when considering any change in the factor they use due to a historical correlation with triggering inhibitor reactions. As we know, blood factor dispensing is predominantly provided by a small sub-set of specialty pharmacies nationally. It is likely that Hemgenix will be distributed directly to leading hemophilia treatment centers / hospitals due to cost and infusion management.