CHICAGO — State Senator Mattie Hunter applauded the FDA’s recent approval of the first gene therapies designed to treat patients with sickle cell disease.
“The FDA's approval of Casgevy and Lyfgenia to treat sickle cell disease is a significant milestone in our ongoing efforts to improve the lives of those affected by this debilitating condition,” said Hunter (D-Chicago). “This is a testament to the progress we can achieve when we prioritize and invest in medical research."
Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the United States, most commonly African Americans. In an affected individual, red blood cells — which are normally round and flexible — are sickled-shaped, stiff and sticky, leading to a variety of problems including blocked blood flow, anemia, organ damage and stroke.
The FDA approved two cell-based gene therapies — Casgevy and Lyfgenia — for the treatment of sickle cell disease in patients 12 and older. Both products are made from the patient’s own blood stem cells, which are modified and given back as a one-time, single-dose infusion. Patients who receive either treatment will take part in a long-term study to evaluate each product’s safety and effectiveness.
Hunter has led legislation to improve care for those affected by sickle cell disease, including the creation of the Sickle Cell Prevention, Care and Treatment Program through the Illinois Legislative Black Caucus’ Health and Human Services Pillar. Additionally, she has served on the Red Cross of Illinois Sickle Cell Task Force.
“This treatment offers hope for improved outcomes and a better quality of life for patients,” said Hunter. “I commend the researchers and scientists who worked tirelessly on these advancements and thank the patients and advocates who helped to make the approval of this treatment possible.”
Read more about the treatments on the FDA’s website.