An Open-Label Study of the Safety and Efficacy of Morotocog Alfa (AF-CC) in Previously Untreated Patients Aged <6 Years in Usual Care Settings
Makaleler > Tam Makale · 2017
Yazarlar: Kaan Kavaklı, James A. Baumann, Francois Huard, Lynne M. Smith, Christine Alvey, Joan Korth‐Bradley, Pablo Rendo, Jeremy Rupon
Blood, Uluslararası, Wos, 2017, cilt 130, ss 1091-1091
DOI: 10.1182/blood.v130.suppl_1.1091.1091
Özet
Abstract Background: Moroctocog alfa (AF-CC) (ReFacto AF), a purified recombinant B-domain deleted factor VIII (FVIII) protein, is indicated for the treatment and prophylaxis of bleeding events in patients (pts) with hemophilia A and is manufactured in the absence of human albumin and purified using an affinity chromatography purification process along with additional filtration. It replaced the predecessor product moroctocog alfa, which was manufactured with human serum album and utilized murine monoclonal antibody purification. Data in previously untreated patients aged Aims: To fulfill a European Medicines Agency requirement for post-authorization safety surveillance and risk management that moroctocog alfa (AF-CC) was acceptable in terms of efficacy and safety (including inhibitor development) in the usual care setting. Methods: This was a nonrandomized, open-label study that enrolled male patients with severe hemophilia A (FVIII activity Results: Twenty-three patients aged Conclusions: The clinical inhibitor rate along with the overall safety and efficiacy of moroctocog alfa (AF-CC) is similar to its predecessor product supporting that the change in manufacturing did not alter the overall clinical benefit. These data also support the use of moroctocog alfa (AF-CC) for the treatment of previously untreated hemophilia A patients less than 6 years of age. Disclosures Kavakli: Shire: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bayer: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees. Baumann: Inventiv: Other: Contractor working for Pfizer on the submitted study. Huard: Pfizer: Employment. Smith: Pfizer: Employment. Alvey: Pfizer: Other: Retired former employee. Korth-Bradley: Pfizer: Employment. Rendo: Pfizer: Employment. Rupon: Pfizer: Employment.