What's next for hemophilia treatments?

The latest gene therapy pipeline update 4Q 2022–1Q 2027 highlights several new and notable drugs in development for hemophilia.

Hemophilia is a rare, genetic bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery.¹ Hemophilia occurs in about 1 of every 5,000 male births. Females can also have hemophilia, but this is much rarer. The two most common types are:

• Hemophilia A (classic hemophilia), caused by a lack or decrease of clotting factor VIII
• Hemophilia B (Christmas disease), caused by a lack or decrease of clotting factor IX

Some people with hemophilia will produce antibodies or inhibitors. Inhibitors increase the risk for complications and associated treatment costs are on average three to four times higher and can cost more than $1 million annually per patient. These patients must receive additional therapy to help them overcome this resistance.

Hemophilia is complicated and costly to manage.

Notable drugs in the pipeline

There is no cure for hemophilia, but the treatment pipeline is rich with clotting factor and non-factor replacement therapies. Therapeutic advances include treating hemophilia with less frequent injections, which could increase adherence and reduce complications.³ Meanwhile, the first hemophilia gene therapy may enter the market in late 2022.

New drugs for hemophilia expected to gain approval between now and the end of 2023 include:

• EtranaDez (etranacogene dezaparvovec), which would be the first hemophilia gene therapy (specifically for severe hemophilia B). It is a one-time IV injection and is expected to be approved later this year.
• Roctavian (valoctocogene roxaparvovec), a one-time IV injection gene therapy for severe hemophilia A, which may be approved around Spring 2023.
• Efanesoctocog alfa, a factor replacement therapy for severe hemophilia A with once-weekly IV injections. This dosing makes it more convenient than the current factor replacement products, which are administered two to four times a week; it is expected to be equally effective at preventing bleeds.
• Concizumab, which has a novel mechanism of action and may be approved for a broad indication, specifically for the prevention of bleeding episodes in patients with hemophilia A and B – with or without inhibitors. Patients receive once-daily subcutaneous injections.

Raising the bar for managing the high cost of hemophilia care

CVS Health has pioneered care for hemophilia patients by providing management services since 1978. More than 40 years of experience has taught us that successful management of complex bleeding disorders calls for a comprehensive care approach as flexible and unique as each patient. That means:

• a sound clinical approach that goes beyond treating bleeds and protecting joints in the short term. This helps promote physical and psychological health for improved long-term outcomes.
• safe and clinically appropriate use of factor replacement therapies while maintaining or improving optimal clinical outcomes.
• resources to educate and empower members so they can stay motivated and informed about their care.

Some of the solutions CVS Health uses to manage hemophilia cost and trend include formulary with preferred hemophilia agents and appropriate utilization through evidence-based care. Our dose and assay management are key to reduce waste and avoid unnecessary costs as we help ensure the right amount of clotting factor at the right time for the member to control or prevent bleeds – achieving industry-leading results. And we’ve developed new ways to smooth actuarial risk with stop-loss coverage and provide financial flexibility with payments over time.