News

CSL’s HEMGENIX ® (etranacogene dezaparvovec-drlb) provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion. With the approval of HEMGENIX, CSL now offers an even more comprehensive portfolio of treatments for people living with hemophilia B, ushering in a new era of treatment options. The FDA approval is supported by results from the ongoing HOPE-B trial, the largest gene therapy trial in hemophilia B to date. Results from the study demonstrated that HEMGENIX allowed patients to produce mean factor IX activity of 39 percent at six months and 36.7 percent at 24 months post infusion.

Patient Advocacy Groups are calling on South Carolina Legislators to support bipartisan legislation to address rising out-of-pocket costs from an emerging insurance practice called “Accumulator Adjustment Programs (AAP).” This legislation will help patients better access their medicine and stay adherent. Unfortunately, health insurers and PBMs have adopted polices, often referred to as “accumulator adjustment programs” that block assistance towards a patient’s deductible and maximum out-of- pocket limits. AAPs further shift cost to patients, putting patients at risk of not being able to afford their medicines. South Carolinians need health insurers to count ALL payments and not discriminate against those patients living with chronic conditions.

The onset of childbirth and the postpartum period are times when women with von Willebrand disease (VWD) are at an increased risk for excessive bleeding, exposing them to further, and in some instances, serious complications. While there exist therapies with VWD-specific indications, it is not uncommon for these patients to still experience excessive bleeding while receiving treatment. These scenarios are challenging as there is sparce clinical data and a subsequent lack of clear guidance on the optimal management of bleeding in these particular settings. 

Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec. Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate compared to baseline FIX prophylactic therapy. Stable and durable FIX levels with mean FIX activity of 36.9 percent of normal in full study population at 18-months, compared to a mean of 39.0 percent of normal at 6 months. Manufacturing operations supporting process validation of etranacogene dezaparvovec successfully completed by uniQure.

More than 140 groups representing patients with serious and chronic conditions this week sent a letter to House Representatives Donald McEachin (D-VA) and Rodney Davis (R-IL) voicing support of their recent introduction of H.R. 5801, the Help Ensure Lower Patient Copays Act (HELP Copays Act). The legislation, which included seven additional co-sponsors from both sides of the aisle, is a two-part solution that helps ensure patients can cover their out-of-pocket costs and access needed medications.

Washington, D.C. – Today, Congressman A. Donald McEachin (VA-04) and Congressman Rodney Davis (IL-13) introduced the Help Ensure Lower Patient (HELP) Copays Act to protect patients from increased out-of-pocket prescription drug costs.
The bipartisan bill builds on previous efforts to lower out-of-pocket (OOP) costs for patients by prohibiting the use of copay accumulator programs, ensuring insurers cannot exclude the value of pharmaceutical manufacturer cost-sharing assistance from counting toward an enrollee’s annual cost-sharing limit.

GREENVILLE, S.C.— Prisma Health has expanded its Hemophilia Treatment Center to include an office in Greenville as well as Columbia. Prisma Health has South Carolina’s only federally supported comprehensive hemophilia treatment center, serving a growing number of both children and adults across the state. “Our mission