SCDAA Statement About Gene Therapy Approval
According to Sickle Cell Disease Association of America, SCDAA
“On Dec. 8, 2023, the Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), Casgevy from CRISPR/Vertex and Lyfgenia from bluebird bio. These are the first treatments of their kind available to individuals with SCD in the United States. SCDAA welcomes the approval of these potentially curative therapies which mark major advances in the treatment of sickle cell disease; however, there are valid concerns about accessibility and the potential for adverse effects.”
Biden-Harris Administration Announces Action to Increase Access to Sickle Cell Disease Treatments
According to Sickle Cell Disease Association of America, CMS.gov press release
“The Biden-Harris Administration announced today that sickle cell disease (SCD) will be the first focus of the Cell and Gene Therapy (CGT) Access Model, which was initially announced in February 2023. The model is designed to improve health outcomes, increase access to cell and gene therapies, and lower health care costs for some of the nation’s most vulnerable populations.”
CMMI Announces the Cell and Gene Therapy Access Model
According to Sickle Cell Disease Association of America, SCDAA
“Today, the Centers for Medicare and Medicaid Innovation (CMMI) announced the launch of their new Cell and Gene Therapy Access Model, a demonstration aimed at improving access to cell and gene therapy products in the Medicaid program. CMMI chose sickle cell disease (SCD) as the first indication for the model. The intent of the model is to provide access to the two recently approved gene therapy products for SCD for individuals insured by Medicaid. Participation by state Medicaid programs will be optional.”
SCDAA and MedicAlert Pilot Program
According to Sickle Cell Disease Association of America, SCDAA
“During a sickle cell pain crisis, time is of the essence. SCDAA and MedicAlert have launched a pilot program designed to improve the emergency room experience for sickle cell warriors. Learn more about the program and how to get involved.”
Dr. Lametra Scott Interview
New Novartis medicine Adakveo® (crizanlizumab) approved by FDA to reduce frequency of pain crises in individuals living with Sickle Cell Disease
November 15, 2019 – Novartis announced today that the US Food and Drug Administration (FDA) approved Adakveo® (crizanlizumab), previously known as SEG101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease. Adakveo represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin –a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion. The medicine is expected to be available to patients in the coming weeks.
What Doctors Should Ignore
Sickle Cell Anemia was first described in 1910 and was quickly labeled a “black” disease. At a time when many people were preoccupied with an imagined racial hierarchy, with whites on top, the disease was cited as evidence that people of African descent were inferior. But what of white people who presented with sickle cell anemia?
