The first domestic recombinant coagulation factor VIII clinical study was officially published in "Haemophilia"

Article source: Medical Rubik's Cube Info

Hemophilia is a bleeding disorder with inherited abnormal blood coagulation function.

Coagulation factor VIII is the main treatment drug for hemophilia A.

In the past, domestic rVIII could only rely on imports, the price was high, and the supply was difficult to guarantee

SCT800 is an rFVIII independently developed by Shenzhou Cell Engineering Co.

On June 5, 2021, the results of SCT800's pivotal clinical trial "Pharmacokinetic, efficacy and safety evaluation of B-domain-deleted recombinant FVIII (SCT800) for prophylactic treatment in adolescent and adult patients with severe haemophilia A" were published online in hemophilia A well-known journal in the field of diseases-"Haemophilia" magazine

The study was initiated by Shenzhou Cell Engineering Co.

Twelve domestic clinical trial institutions participated in the study, and a total of 73 adolescents and adults with severe (FVIII:C<1%) hemophilia A patients who had previously received factor VIII replacement therapy (exposure day ≥150 days) were enrolled.

The results of the study showed that the average estimated ABR of SCT800 for the prevention and treatment of severe hemophilia A patients was 2.

During the treatment visit, a total of 94 breakthrough bleeding events were observed.

b.

The ultimate goal of preventive treatment is to maintain/improve the patient’s muscle and joint function and improve the patient’s quality of life

In the process of factor replacement therapy, the production of inhibitors is the most clinically concerned complication

In terms of safety, 53.

Note: The original text has been deleted

Reference

[1] Yang Renchi, editor-in-chief.

[2]WFH Guidelines for the Management of Hemophilia, 3rd edition.

[3]Hermans Cedric, et al.