turoctocog alfa pegol SC (NN7170) / Novo Nordisk - LARVOL DELTA

Validation of the Hemophilia Treatment Experience Measure (Hemo-TEM): a New Haemophilia-specific Patient-reported Outcome Measure (ISTH 2022) - " Patients from 3 clinical trials (NN7170-4213, NN7415-4255, NN7415-4310) currently taking an injection for haemophilia (n=88) completed a validation battery (demographics and PRO measures needed for the cross-sectional validation analysis) at a screening visit, at baseline (retest), and at 24 weeks post-baseline (n=56, sensitivity to change). Item Reduction dropped 4 items resulting in a final 26-item measure (Figure 1). Factor analysis generated 5 domains in the Hemo-TEM [injection difficulties (3 items), physical impact (6 items), treatment bother (7 items), interference with daily life (4 items), and emotional impact (6 items)] and a total score. All scores were reliable [internally consistent (0.84 to 0.88) and reproducible (0.80 to 0.92)]."

Clinical • Patient reported outcomes • Hematological Disorders • Hemophilia • Rare Diseases

Preclinical pharmacokinetics and biodistribution of subcutaneously administered glycoPEGylated recombinant factor VIII (N8-GP) and development of a human pharmacokinetic prediction model. (PubMed, J Thromb Haemost) - "SC administered N8-GP may provide effective hemophilia A prophylaxis. A phase I clinical trial is underway to investigate this possibility."

Journal • PK/PD data • Preclinical

Immunogenicity, Safety, Pharmacokinetics and Preliminary Efficacy Results from the First Human Subcutaneous Factor VIII Trial (Subcutaneous Turoctocog Alfa Pegol, SC N8-GP) in Previously Treated Patients with Hemophilia A (Alleviate 1) (ISTH 2019) - P1; "SC N8-GP demonstrated clinically relevant FVIII levels with dose linearity in PTPs with severe hemophilia A. The immunogenicity findings in part B may relate to the subcutaneous route of administration because no immunogenicity concerns were reported with N8-GP administered intravenously in previous studies."

Clinical • PK/PD data