Baxter International Inc. BAX today announced pivotal Phase I/III study
results evaluating the safety and efficacy of BAX 326, an investigational
recombinant factor IX (rFIX) protein, for the treatment and prophylaxis of
bleeding episodes for patients with hemophilia B over 12 years of age. The
data were presented at the 54^th Annual Meeting and Exposition of the American
Society of Hematology (ASH) in Atlanta. BAX 326 was recently granted
orphan-drug designation by the U.S. Food and Drug Administration (FDA), a
special status given to a product that when approved, will address an unmet
need for people with a rare disease or condition. Data supporting the
prophylaxis indication, which is the basis for the orphan drug designation,
was included in the biologics license application (BLA).
The Phase I/III prospective, controlled, multicenter study investigated the
pharmacokinetics, efficacy and safety of BAX 326 in 73 previously-treated
patients with severe or moderately severe hemophilia B. Results from the study
showed that twice-weekly prophylactic treatment with BAX 326 achieved a median
annualized bleed rate of 1.99 with 43 percent of patients experiencing no
bleeds. The impact of prophylaxis with BAX 326 also translated into
statistically significant improvements in physical health-related quality of
life (HRQoL), as measured by improvements in the Bodily Pain and Role-Physical
domains in the Short Form Health Survey (SF-36v2) tool. Statistically
significant changes were not seen in the other physical health-related domains
or the mental health domains.
No inhibitors were detected and no cases of anaphylaxis were reported in any
patients. Three treatment-related adverse events were reported in 2 of the 73
(2.7%) patients, all of which were mild and transient: dysgeusia (distortion
of the sense of taste) occurred twice in one patient and pain in extremity
occurred once in another patient. More than 70 percent of subjects (56 of 73)
had 50 or more exposure days to BAX 326 during the study.
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