(CNN) — The U.S. Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era of treatments for genetic conditions.
The medicines, called Casgevy and Lyfgenia, are potential cures for people with sickle cell, a debilitating and life-shortening inherited red blood cell disorder that disproportionately affects African Americans.
Casgevy is the CRISPR-based treatment, made by Vertex Pharmaceuticals and Crispr Therapeutics, while Lyfgenia, made by Bluebird Bio, uses an older gene therapy approach. Both were cleared for people age 12 and older with histories of vaso-occlusive crises, painful events caused by the disease.
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need,” said Dr. Nicole Verdun, director of the FDA’s Office of Therapeutic Products within its Center for Biologics Evaluation and Research. “We are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today.”
Johnny Lubin is one of the youngest people to have had the CRISPR treatment, as part of a clinical trial. Before he went through it, he recalls, he had one main concern.
“I was worrying that I might get, like, superpowers,” said Johnny, now 15, who lives in Trumbull, Connecticut, about 60 miles north of New York City.
For many in the sickle cell community, the approvals have been a long time coming. The disease afflicts about 100,000 people in the US, including an estimated 1 of every 365 Black babies born, according to the US Centers for Disease Control and Prevention, and has long been considered neglected by the pharmaceutical industry. About 20,000 people in the US are thought to have a severe enough form of the disease to potentially qualify for a treatment like this.
“To have sickle cell suddenly be the focus of this dramatic new approach to therapy development is, on the one hand, great, because hopefully it will undo some of that history of neglect and really give the sickle cell community the attention that is always needed,” said Mayo Clinic bioethicist Megan Allyse. She notes, however, that access to such a cutting-edge treatment remains a major question.
>>Read more from CNN here.
CNN’s Meg Tirrell contributed to this story
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