FDA approves 2 new gene therapies for sickle cell disease, a ‘functional cure’ for many patients

ABC NEWS

The U.S. Food and Drug Administration (FDA) on Friday announced that it has approved Casgevy, the first CRISPR gene-editing therapy for sickle cell disease, paving the way for thousands of patients in the U.S. to receive a treatment that has been described as a “functional cure” for eligible patients.

The FDA also approved a gene therapy called Lyfgenia. Collectively, these two therapies represent two “milestone” treatments for sickle cell disease, according to the FDA announcement.

Sickle cell disease is a genetic condition that affects approximately 100,000 Americans – primarily Black Americans with African ancestry, according to the Centers for Disease Control and Prevention (CDC).

Researchers estimate that roughly 20-25% of those with the disease are sick enough that they would be good candidates for the newly approved treatments, which were approved for people aged and older.

Although historic, the new treatments come with significant hurdles and potential risks. The treatment is difficult to manufacture and requires months of preparation for patients and their families. Patients will need to stay in the hospital for several weeks, and will receive preemptive chemotherapy that can place fertility at risk. Because of this, patients will likely be offered preemptive fertility preservation.

Still, in a world with few options, doctors and patients say this is a historic step forward.

“We’ve had nothing in our field for decades,” said Dr. Sharl Azar, Medical Director of the Comprehensive Sickle Cell Disease (SCD) Treatment Center at Mass General Hospital. “So this is part of the reason why this is such a large, seismic shift for us.”

According to the CDC, one out of every 365 Black or African American babies born in the U.S. is born with sickle cell disease, which is characterized by abnormal ‘sickle’-shaped red blood cells that can clog blood flow, causing severe pain episodes, fatigue, infections, stroke and sometimes organ damage and other complications. The average life expectancy for those living with sickle cell disease is roughly 52 years old.

The only cure is a risky bone marrow transplant – an option that is out of reach for most patients because it requires a donor match, typically an unaffected sibling.

The new treatments are technically not a cure in the same way a bone marrow transplant would be.

“What we are calling it is a ‘functional cure,'” said Dr. Haydar Frangoul, Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute and HCA Healthcare’s The Children’s Hospital At TriStar Centennial. “I think it is better described by the fact that patients do not have any symptoms.”

Many of the clinical trial volunteers who have already undergone treatment say they have a new lease on life.

“I’m literally a different person,” said Jimi Olaghere, an early clinical trial volunteer for the Casgevy CRISPR trial, who was treated at the Sarah Cannon Research Institute in Nashville. “Before, my life was me most of the time in bed writhing in pain, not present because of all the pain medications.”