Sanofi highlights new BTK inhibitor data and strong results for ALTUVIIIO | ASH 2025

Managed Healthcare Executive spoke with Jeff Schaffnit, general manager and head of rare blood disorders at Sanofi, about new data the company presented at ASH 2025 this weekend in Orlando. The discussion focused on the growing role of BTK inhibitors in rare blood diseases beyond immune thrombocytopenia (ITP) and the four-year results for Altuviiio (efanesoctocog alfa), Sanofi’s therapy for hemophilia A.

According to Schaffnit, BTK inhibitors target a key enzyme that helps drive abnormal immune activity. Due to a number of rare blood disorders that share patterns of immune dysregulation, Sanofi is studying whether this approach can work across multiple conditions.

Schaffnit also shared data on Wayrilz (rilzabrutinib), its BTK inhibitor now approved for ITP, that is designed to limit the traditional effects seen with older BTK drugs. Schaffnit described it as “a selective, covalent and reversible BTK.”

Clinicians and patients at ASH made clear that ITP still needs more treatment options, Schaffnit recalled.

Wayrilz can be used in adults with persistent or chronic ITP who did not respond well enough to a previous treatment. This gives providers flexibility to use it after steroids or IVIG or as a later option in those who have failed several therapies, Schaffnit said. Early in the launch, he noted clinicians are already using it both in second-line and in more refractory third- or fourth-line settings. Sanofi is also studying its use earlier in treatment through its ongoing Luna 4 phase 3b trial.

Schaffnit also discussed Altuviiio, a once-weekly factor replacement therapy that aims to keep factor VIII levels higher for longer in hemophilia A.

Sanofi presented four-year results from its trial showing continued improvement in outcomes. For example, patients had low annual bleed rates, with many experiencing no bleeds. Spontaneous and traumatic bleeding decreased over time.

In addition, no inhibitors were detected in the long-term study, and nearly all participants remained on therapy through the four-year period. Overall, data suggests promising protection from bleeding and strong adherence.