Rebinyn is a glycoPEGylated rebombinant factor IX (rfIX) therapy developed by Novo Nordisk for the treatment of hemophilia B. The PEGylation of proteins is believed to prolong their half-life by interfering with renal clearance, protecting against enzymatic degradation, and blocking interaction with clearance receptors. In Rebinyn, the activation peptide attached to the PEG moiety is cleaved off during the coagulation process to leave activated fIX. This allowed the drug to be dosed at weekly intervals in its pivotal clinical trials.
Rebinyn was first approved in the US in May 2017, with subsequent EU approval under the Refixia brand name in June 2017.
The long-acting rfIX therapy is the third such product to gain approval for hemophilia B patients. Although it can be dosed at weekly intervals, it does not have any known advantages over other competing long-acting rfIXs, and its success will be dependent on Novo Nordisk’s extensive experience in the hematology market. Rebinyn will be further hindered by the lack of routine prophylaxis on its product label, until Novo Nordisk is able to adequately demonstrate the lack of toxicity associated with the PEG modification. Owing to the improvement that long-acting products are able to offer over conventional treatments, especially within the hemophilia B market, Rebinyn will be part of the trend towards increased prophylaxis, fewer injections, and the erosion of former gold-standard drug BeneFIX (rfIX; Pfizer).
LIST OF FIGURES 9 Figure 1: Rebinyn for hemophilia – SWOT analysis
10 Figure 2: Datamonitor Healthcare’s drug assessment summary of Rebinyn for hemophilia
11 Figure 3: Datamonitor Healthcare’s drug assessment summary of Rebinyn for hemophilia
13 Figure 4: Rebinyn sales for hemophilia across the US, Japan, and five major EU markets, by country, 2016–25
LIST OF TABLES
6 Table 1: Rebinyn drug profile
7 Table 2: Rebinyn pivotal trial data in hemophilia
14 Table 3: Rebinyn sales for hemophilia across the US, Japan, and five major EU markets, by country ($m), 2016–25
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