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Purpose: Hemophilia B is an X-linked congenital bleeding disorder caused by a deficiency of coagulation factor IX (FIX) clotting activity. This study evaluated safety and efficacy of nonacog alfa, a recombinant human blood coagulation FIX replacement product, in males aged 12-65 years with hemophilia B (FIX activity ≤ 2%) with or without inhibitors in India., Methods: In this multicenter, open-label, post-approval phase 4 study, participants were treated for up to 8 weeks, with up to a 4-week screening period and a subsequent post-treatment 28-day safety observation period. Intravenous nonacog alfa 40 IU/kg (range 13-78 IU/kg) was administered at intervals of 3-4 days, in accordance with the approved local product document., Results: A total of 25 participants were enrolled and completed the study. No participants developed FIX inhibitors during the study, experienced treatment-related adverse events (AEs) or serious AEs, or developed a thrombotic event and/or hypersensitivity reaction. No participants experienced bleeding events requiring on-demand treatment with nonacog alfa. Seventeen bleeding episodes (16 spontaneous and 1 traumatic) were reported in 10 participants; all occurred post treatment, with the exception of a minor gum-bleeding event, and were managed without treatment. The mean (SD) annualized total factor consumption (TFC) per patient was 224,582 (75,527) IU; the mean (SD) annualized TFC by weight per patient was 3639 (573) IU/kg., Conclusion: Nonacog alfa was safe and effective for the prevention of hemorrhagic episodes in Indian males with congenital, severe hemophilia B. No participants developed FIX inhibitors, and no new safety signals were reported., Competing Interests: Competing InterestsNirmalkumar Choraria and Shashikant Apte have no conflicts to disclose. Savita Rangarajan has served on an advisory board for Pfizer and for Sanofi Sigilon; has served on a speakers’ bureau for Pfizer and Takeda; and has served as a consultant for Reliance Life Sciences. M. Joseph John has served as a speaker/consultant or advisory board member, and/or received research support for attendance at educational meetings from Dr Reddy’s Lab, Grifols, Janssen, Mylan, Novo Nordisk, Pfizer, Roche, and Takeda. Pritam Gupta, Shyam Parvatini, Rohit Chand, Seema Pai, G. S. H. Ramakanth, Jeremy Rupon, Chhabra Amit, Hitesh Bhaskarrao Muley, and Damien Simoneau are employees of Pfizer Inc. and may own stock/options in the company., (© The Author(s) 2022.)

Purpose: Hemophilia A is an X-linked congenital disorder, characterized by factor VIII (FVIII) deficiency. Globally, India has the highest population of patients with hemophilia, and there is a clear unmet need for appropriate and effective treatment for this patient population. This multicenter, open-label, post-approval study evaluated the safety and efficacy of moroctocog alfa in patients with moderate or severe congenital hemophilia A in India., Methods: Intravenous moroctocog alfa was administered 30 ± 5 IU/kg 3 times weekly for bleeding prophylaxis, according to the local product document. Participants were treated for up to 8 weeks, with an up to 4-week screening period and a subsequent post-treatment, 28-day safety observation period. Patients continued in the study until at least 24 exposure days or a period of up to 8 weeks on moroctocog alfa., Results: A total of 50 participants were enrolled, and 48 (85.7%) completed the study. No participants developed FVIII inhibitors during the study. The mean (SD) annualized bleeding rate during moroctocog alfa prophylaxis was 0.79 (2.0) with a median (range) of 0.00 (0.0, 6.8). The mean (SD) annualized total factor consumption (TFC) per participant was 287,432 (93,866) IU; the mean (SD) annualized TFC by weight per participant was 4176 (858) IU/kg. Moroctocog alfa was well tolerated with no reported treatment-emergent adverse event-related dose reductions, discontinuations, or serious adverse events., Conclusion: Moroctocog alfa was safe, effective, and well tolerated in Indian participants with congenital moderate to severe hemophilia A. No participant developed FVIII inhibitors during the study., Competing Interests: Competing InterestsNirmalkumar Choraria and Shashikant Apte have no conflicts to disclose. Savita Rangarajan has served on advisory boards for Pfizer and Sanofi Sigilon; on speakers’ bureaus for Pfizer and Takeda; and as a consultant for Reliance Life Sciences. M. Joseph John has received honoraria as a speaker/consultant and/or advisory board member, and has received research grants for attendance at educational meetings from Dr Reddy’s Lab, Grifols, Janssen, Mylan, Novo Nordisk, Pfizer, Roche, and Takeda. Pritam Gupta, Shyam Parvatini, Rohit Chand, Seema Pai, G. S. H. Ramakanth, Jeremy Rupon, Chhabra Amit, Hitesh Bhaskarrao Muley, and Damien Simoneau are employees of Pfizer Inc. and may own stock/options in the company., (© The Author(s) 2022.)