- Center on Health Equity and Access
- Clinical
- Health Care Cost
- Health Care Delivery
- Insurance
- Policy
- Technology
- Value-Based Care
CHMP Recommends First Treatment of FCS for Marketing Approval
The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion that an orphan drug, volanesorsen (Waylivra), receive a conditional marketing authorization. The drug would be the first medication approved for the treatment of the familial chylomicronemia syndrome (FCS).
The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion that an orphan drug, volanesorsen (Waylivra), receive a conditional marketing authorization. The drug would be the first medication approved for the treatment of the familial chylomicronemia syndrome (FCS).
FCS is a rare genetic disease that prevents the body from breaking down lipids; therefore, patients with this condition have extraordinarily high levels of triglycerides in their blood. Having such high levels of triglycerides can cause adverse events such as severe abdominal pain, potentially fatal attacks of acute pancreatitis, and memory loss.
“This positive CHMP opinion is an important step forward as we work to bring Waylivra to people living with FCS who currently have no treatment options. Once approved, Waylivra will be the first and only therapy for people living with the devastating challenges of FCS. We are now anticipating approval in Europe in the coming months,” Paula Soteropoulos, chief executive officer of Akcea Therapeutics, said in a statement.
The marketing application was submitted based on results from the phase 3 APPROACH study and the ongoing Approach Open Label Extension study. Results from the APPROACH trial, which enrolled 66 patients, making it the largest trial conducted in patients with FCS to date, found that treatment with Waylivra reduced triglycerides by 77% after 3 months compared with an increase of 18% in the placebo arm. The most common adverse events in the APPROACH trial were injection site reactions and reductions in platelet levels.
“The FCS community is encouraged by the positive CHMP opinion and we remain very hopeful that people living with FCS will soon have an approved treatment available in the [European Union]. Without a treatment, people living with FCS have debilitating daily symptoms as well as the constant fear of pancreatitis,” said Jill Prawer, founder and chair of the LPLD Alliance. “This is a positive step for people living with FCS who currently have no treatment options.”
The positive opinion will now be sent to the European Commission to make a final decision on a European Union-wide marketing authorization.
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis
January 23rd 2024Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
Read More
Oncology Onward: A Conversation With Dr Shereef Elnahal, Under Secretary for Health
April 20th 2023Shereef Elnahal, MD, MBA, under secretary for health at the Veterans Health Administration (VHA), sat for a conversation with our hosts Emeline Aviki, MD, MBA, Memorial Sloan Kettering Cancer Center, and Stephen Schleicher, MD, MBA, Tennessee Oncology, that covered the cancer footprint of the VHA.
Listen
Interventions Needed to Increase DMT Uptake in Sickle Cell Disease
December 26th 2023A recent study found that uptake of disease-modifying therapies (DMTs) has been low among patients with sickle cell disease, suggesting that more interventions that consider individual patient characteristics are needed to improve adoption.
Read More
Exploring Payer Coverage Decisions Following FDA Novel Drug Approvals
May 3rd 2022On this episode of Managed Care Cast, Ari D. Panzer, BS, lead author and researcher, then at Tufts Medical Center—now at Duke University—discusses the findings from his team’s investigation into coverage decisions by health plan insurers of the 66 drugs approved by the FDA in 2018.
Listen
Exagamglogene Autotemcel Meets End Points in Severe Sickle Cell Disease, β-Thalassemia
December 7th 2023Two posters set to be presented at the 65th American Society of Hematology Annual Meeting & Exposition met their primary and secondary end points regarding exagamglogene autotemcel therapy for sickle cell disease and β-thalassemia.
Read More
