World Federation of Hemophilia

New Study Highlights Benefits of Maintaining High Factor VIII Levels in Hemophilia A Patients

3 months ago

• Maintaining high Factor VIII (FVIII) levels in people with hemophilia A provides significant clinical benefits, including reduced bleeding risk and better joint health preservation. • Despite advances in prophylactic treatment, standard-of-care prophylaxis does not completely prevent joint damage or chronic pain, with approximately 47% of hemophilia patients experiencing ongoing pain. • Recent innovations in hemophilia treatment, including extended half-life therapies, non-factor replacement options, and gene therapy, offer new approaches to sustaining higher FVIII levels and improving patient outcomes.

Sanofi Halts Phase 3 Fitusiran Trials Due to New Safety Concerns in Hemophilia Studies

4 months ago

• Sanofi has voluntarily suspended its phase 3 clinical trials of fitusiran, an RNAi therapeutic for hemophilia A and B, following reports of non-fatal thrombotic events in study participants. • The safety pause, announced by major hemophilia patient organizations, marks the second significant safety setback for fitusiran, following a previous trial halt in 2017 due to a patient death. • The development impacts Sanofi's strategic pipeline, as fitusiran was positioned to compete with Roche's Hemlibra in the hemophilia treatment market.

Pfizer's Gene Therapy Fidanacogene Elaparvovec Shows Promise in Haemophilia B Treatment

5 months ago

• Pfizer's fidanacogene elaparvovec (SPK-9001) gene therapy met its primary endpoint in the Phase 3 BENEGENE-2 study for haemophilia B, demonstrating non-inferiority and superiority in annualized bleeding rate. • The BENEGENE-2 study showed a 71% reduction in annualized bleeding rate (ABR) with fidanacogene elaparvovec compared to the pre-treatment period in adult males with moderately severe to severe haemophilia B. • Secondary endpoints revealed a 78% reduction in treated ABR and a 92% reduction in annualized infusion rate, with a safety profile consistent with earlier phase studies. • Fidanacogene elaparvovec has received breakthrough therapy designations from regulatory agencies, potentially offering a one-time treatment option to reduce the clinical burden for haemophilia B patients.