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Review
. 2022 Feb 18;11(4):1071.
doi: 10.3390/jcm11041071.

IDELVION: A Comprehensive Review of Clinical Trial and Real-World Data

Miguel Escobar  1 , Maria Elisa Mancuso  2 , Cedric Hermans  3 , Cindy Leissinger  4 , Wilfried Seifert  5 , Yanyan Li  6 , William McKeand  6 , Johannes Oldenburg  7
Affiliations
Review

IDELVION: A Comprehensive Review of Clinical Trial and Real-World Data

Miguel Escobar et al. J Clin Med. .

Abstract

Hemophilia B is a bleeding disorder caused by a deficiency of coagulation factor IX (FIX). Treatment with FIX replacement products can increase FIX activity levels to minimize or prevent bleeding events. However, frequent dosing with standard-acting FIX products can create a high treatment burden. Long-acting products have been developed to maintain bleed protection with extended dosing intervals. Recombinant factor IX-albumin fusion protein (rIX-FP) is a long-acting product indicated for the treatment and prophylaxis of bleeding events and perioperative management in adult and pediatric patients. This review outlines data from all previously treated patients in the Prophylaxis and On-Demand Treatment using Longer Half-Life rIX-FP (PROLONG-9FP) clinical trial program and summarizes real-world data evaluating the use of rIX-FP in routine clinical practice. In the PROLONG-9FP program, rIX-FP demonstrated effective hemostasis in all patients at dose regimens of up to 21 days in patients aged ≥ 18 years and up to 14 days in patients aged < 12 years. rIX-FP has a favorable pharmacokinetic profile and an excellent safety and tolerability profile. Extended dosing intervals with rIX-FP led to high levels of adherence and reduced consumption compared with other FIX therapies. Data from real-world practice are encouraging and reflect the results of the clinical trials.

Keywords: albumin fusion protein; annualized bleeding rates; extended half-life; factor IX; hemophilia B.

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Conflict of interest statement

M.E. reports consulting honoraria for participation in advisory boards and/or consulting for CSL Behring, Genentech, Biomarin, Kedrion, the National Hemophilia Foundation, Novo Nordisk, Pfizer, Roche, Sanofi, Shire, and Takeda; and research funding from Pfizer, NovoNordisk, Takeda, Sanofi, and UniQure. M.E.M. reports consulting honoraria for participation in advisory boards and/or speaker bureaus for Bayer, Biotest, Biomarin, Catalyst, CSL Behring, Grifols, Kedrion, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi, Spark Therapeutics, and Takeda. C.H. reports consulting honoraria for participation in advisory boards and/or speaker bureaus for Bayer, Biogen, CAF-DCF, CSL Behring, Kedrion, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Shire, and SOBI; research funding from Bayer, Shire, and Pfizer; and is a past president of the European Association of Haemophilia and Allied Diseases (EAHAD) and a member of the Board of Directors of the World Federation of Hemophilia (WFH). C.L. reports consulting honoraria for participation in advisory boards for Bayer, Catalyst, CSL Behring, Genentech/Roche, HEMA Biologics, Sanofi, and Takeda. W.S., Y.L., and W.M. are employees of CSL Behring. J.O. has received reimbursement for attending symposia/congresses, honoraria for speaking and/or consulting, and funds for research from Bayer, Biotest, Chugai, CSL Behring, Grifols, Novo Nordisk, Octapharma, Pfizer, Roche, Shire, and Swedish Orphan Biovitrum.

Figures

Figure 1
Figure 1
Overview of the PROLONG-9FP program. PK, pharmacokinetics; PTPs, previously treated patients.
Figure 2
Figure 2
Hemostatic efficacy rating of rIX-FP in surgery. * Hemostatic efficacy ratings provided by the investigator/surgeon Figure adapted from Negrier, C.; et al. Haemophilia 2016, 22, e259–e266 and Pan-Petesch, B.; et al. Thromb. Res. 2020, 193, 139–141.
Figure 3
Figure 3
Efficacy of rIX-FP weekly prophylaxis compared with other rFIX products. * Mean ABR from Lambert et al.’s and Kavakli et al.’s studies. Figure adapted from Davis, J. et al., J. Med. Econ. 2019, 22, 1014–1021. Note, mean AsBR and mean AjBR data are not reported for rFIXFc or IB1001; mean ABR/AsBR/AjBR are not reported for N9-GP. Median doses for each product are as follow: rIX-FP, 40.3 IU/kg (7-day dosing) [8]; rFIXFc, 45 IU/kg (7-day dosing) [26]; rFIX, 100 IU/kg (7-day dosing) [27]; BAX 326, 50.5 IU/kg (2×/weekly dosing) [28]; IB1001, 53 IU/kg (2×/weekly dosing) [29]. ABR, annualized bleeding rate; AsBR, annualized spontaneous bleeding rate; AjBR, annualized joint bleeding rate; rFIX, recombinant factor IX.
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