Interim funding secured for new bleeding disorder therapies

State and federal Health Ministers have agreed to interim funding arrangements for three new therapies for bleeding disorders, expanding access to treatment for Australians living with serious and potentially life-threatening conditions.

The arrangements cover Takeda’s Obizur (susoctocog alfa), CSL’s Hemgenix (etranacogene dezaparvovec) and Novo Nordisk’s Alhemo (concizumab), with the therapies to be funded through the National Blood Arrangements.

The therapies have received recommendations from the Medical Services Advisory Committee (MSAC), supporting their access for Australians with bleeding disorders. At the Health Ministers Meeting on 4 September, it was noted that a time-limited working group has been established to consider future access arrangements for emerging therapeutic products.

Haemophilia is a rare, genetic bleeding disorder caused by a deficiency or dysfunction of clotting factor VIII (FVIII) or factor IX (FIX). Reduced levels of these clotting factors can leave people vulnerable to spontaneous or recurrent bleeding, with potentially significant and debilitating consequences.

Alhemo provides a new prophylactic treatment option. Administered daily by subcutaneous injection, it is indicated for patients with haemophilia A with FVIII inhibitors or haemophilia B with FIX inhibitors.

Public consultation identified several anticipated benefits from Alhemo, including the availability of subcutaneous rather than venous administration, fewer bleeding episodes and improvements in quality of life.

The Haemophilia Foundation Australia (HFA) said that living with congenital haemophilia B and FIX inhibitors was debilitating and, in some cases, could be fatal because of uncontrolled bleeding. HFA added that individuals live with significant pain, have poor mobility, are often wheelchair bound, depend on pain relief and miss out on daily activities.

Healthcare professionals and people who had accessed Alhemo through clinical trials also reported positive experiences with the treatment. They said the pen device was easy to use, treatment was less painful, and bleeding episodes were greatly reduced.

The other two therapies address distinct bleeding disorder needs. Hemgenix is a one-time gene therapy for adults with haemophilia B (congenital factor IX deficiency) who have no history of FIX inhibitors. Obizur, meanwhile, is indicated for the treatment of bleeding episodes in adults with acquired haemophilia A.

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