CSL’s innovative approach ensuring Hemgenix delivers for patients

CSL (ASX:CSL) presented new HOPE-B Phase III data confirming the five-year durability of Hemgenix (etranacogene dezaparvovec), the only gene therapy for hemophilia B, at the American Society of Hematology (ASH) Annual Meeting earlier this month.

These data, which were also published in the New England Journal of Medicine (NEJM), showed sustained near-normal factor IX (FIX) activity, bleed protection and favorable safety in adults with severe or moderately severe hemophilia B.

Indeed, as many as 94% of patients (51 of 54) remained free from the burden of continuous prophylaxis treatment through five years following a single infusion of Hemgenix, demonstrating prolonged therapeutic benefit.

At year five, mean factor IX activity levels remained strong at 36.1% and Hemgenix continued to demonstrate a favourable safety profile

The results—building on the four-year post-infusion data—further increase confidence in a therapy that has won regulatory approval in the US, Europe and other regions,  and been used to treat more than 75 people across eight countries.

‘Makes a huge difference’

Monica De Abadal Salsas, vice president, head of medical affairs, International, CSL, spoke to The Pharma Letter about the importance of the data, the company’s innovative outcomes-based agreements to bring Hemgenix to patients, and how the roll-out has gone so far.

“The data published in NEJM delivers five years durability and sustained near to normal FIX activity levels, greater bleed protection compared to prophylaxis, and significant decrease of FIX prophylaxis consumption, while maintaining a favorable safety profile,” she explained.

“It highlights the meaningful reduction in joint bleeds that are a major cause of pain and disability in people living with hemophilia. For them, chronic joint pain and long-term joint damage are very common complications. And that translates into reduced mobility and much lower quality of life. 

“By decreasing the bleeds, Hemgenix has the potential to offer these patients a reduced physical burden of hemophilia. Many people with hemophilia find themselves having to think about their disease all the time because they need to think about “what if I bleed?” That’s an emotional and mental impact for them. 

“So, the fact that they now don’t have to think about having to have their prophylaxis treatment, makes a huge difference.”

Outcomes-based agreements 

Since launch, feedback from treatment centers has been positive, and CSL continues to work closely with healthcare systems to support the roll-out as Hemgenix is a gene therapy that represents a paradigm shift in the field of rare disease treatment. 

“Adoption requires naturally some time for health care systems to establish the infrastructure and the appropriate pathways,” Dr De Abadal Salsas said. “That means that CSL is working very closely with physicians, patient groups, treatment centers and health care systems on medical education, on management of hemophilia, but also on supporting that treatment process. 

“The health care systems need also to be prepared to identify those eligible patients, administer gene therapy and have the appropriate infrastructure for those specialized centers.” 

CSL is confident that, as the company continues to secure positive reimbursement decisions for Hemgenix, it can continue to expand patient access globally, reflecting growing momentum and recognition of its innovative therapy.

It is not just with Hemgenix itself where CSL is delivering innovation. The company is pioneering innovative outcomes-based agreements and working with healthcare systems to implement these in real-world settings.

“It being the first gene therapy, this is reshaping the treatment landscape and how we are interacting with healthcare systems,” Dr De Abadal Salsas said. “Up until now, national reimbursements have been granted in 11 markets. In many of these, it’s the first product of its type, and each country has its own unique healthcare system. That means that negotiations need to be tailored to each healthcare system, so we need to engage in an early and iterative dialogue with regulators, payers and governments to pilot this outcomes-based agreement.”

Tailored approach

CSL has to ensure that within those healthcare systems, there is recognition of the unmet need and the requirement for innovative payment models as well as insurance readiness and expertise. 

“The contracting solutions can be very different,” Dr De Abadal Salsas said. “For example, in Spain we obtained national reimbursement in September 2024, which was based on an outcomes-based agreement that addressed the apparent durability of Hemgenix, while also recognizing the value that it brings to patients, carers and healthcare professionals, as a once-in-a-lifetime therapy. That national agreement entails that the health care authorities will only pay for as long as the gene therapy works. 

“In Spain, each region has their own way of setting up the centers to make Hemgenix accessible. In the case of Germany, CSL secured the first national success-based reimbursement agreement with the National Association of Statutory Health Insurance Funds (GKV-Spitzenverband), includes a pay for performance element of Hemgenix.

“These are the type of different discussions we're engaging in, which are very tailored to the healthcare systems in different countries. We’re looking forward to continuing to make progress, so more patients can access Hemgenix.” 

Thankfully, CSL’s stellar data from the HOPE-B program is a compelling reminder of the benefits of this work for patients. 

“The HOPE-B five-year data really is landmark data in terms of marking the final analysis for this study, showing that five-year durability,” Dr De Abadal Salsas said.