Final results from the PUPs A-LONG and PUPs B-LONG studies assessing the efficacy and safety of Elocta® (efmoroctocog alfa) and Alprolix® (eftrenonacog alfa) in previously untreated patients (PUPs) with haemophilia A and B respectively were presented at the ISTH2020 (
"These are the first published final results for extended half-life (EHL) treatments in PUPs demonstrating tolerance and efficacy in this vulnerable patient population," said
PUPs A-LONG (NCT02234323) was the first study evaluating an EHL, recombinant factor VIII Fc-fusion protein (rFVIIIFc) for prevention and treatment of bleeds in PUPs with severe haemophilia
PUPs B-LONG (NCT02234310) was the first study of recombinant factor IX Fc-fusion protein (rFIXFc) for prevention and treatment of bleeds in PUPs with haemophilia B. Alprolix was well tolerated and effective for both prophylaxis and treatment of bleeding episodes. The overall inhibitor incidence was within the expected range. Importantly, only one patient developed a low-titre inhibitor, and no high-titre inhibitors were developed.
Method and endpoints
PUPs A-LONG was an open-label, multicentre, phase 3 study that enrolled male PUPs aged <6 years with haemophilia A (<1 IU/dL endogenous FVIII) to receive rFVIIIFc. The primary endpoint was inhibitor development and secondary endpoints included annualised bleeding rate (ABR) and assessment of response to treatment of bleeding episodes with rFVIIIFc.
PUPs B-LONG was an open-label, multicentre, multinational, phase 3 study including male PUPs aged <18 years with haemophilia B (≤2 IU/dL endogenous FIX) to receive rFIXFc. The primary endpoint was occurrence of inhibitor development and secondary endpoints included annualised bleeding rate (ABR) and assessment of response to treatment of bleeding episodes with rFIXFc.
About Elocta®
Elocta® (efmoroctocog alfa) is a recombinant clotting factor therapy developed for haemophilia A using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Elocta to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). Elocta is manufactured using a human cell line in an environment free of animal and human additives.
Elocta is approved and marketed by Sobi for the treatment of haemophilia A in the EU,
As with any factor replacement therapy, allergic-type hypersensitivity reactions and development of inhibitors may occur in the treatment of haemophilia A. Inhibitor development has been observed with Elocta, including in previously untreated patients. Note that the indication for previously untreated patients is not included in the EU Product Information for Elocta.
About Alprolix®
Alprolix® (eftrenonacog alfa), is a recombinant clotting factor therapy developed for haemophilia B using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling Alprolix to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). Alprolix is manufactured using a human cell line in an environment free of animal and human additives. Alprolix is approved and marketed by Sobi for the treatment of haemophilia B in the EU,
Allergic-type hypersensitivity reactions and development of inhibitors have been observed with Alprolix in the treatment of haemophilia B, including in previously untreated patients. For more information, please see the full
About the Sobi and
Sobi and
About Sobi
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,400 people across
For more information please contact
Paula Treutiger, Head of Communication & Investor Relations
+46 733 666 599
paula.treutiger@sobi.com
+46 708 734 095
linda.holmstrom@sobi.com
1. https://www.cdc.gov/ncbddd/hemophilia/data.html
Postal address SE-112 76
Phone: 46 8 697 20 00 www.sobi.com
https://news.cision.com/swedish-orphan-biovitrum-ab/r/data-presented-at-isth2020-strengthens-evidence-for-both-elocta---and-alprolix---in-previously-untre,c3152881
https://mb.cision.com/Main/14266/3152881/1278279.pdf
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