On Friday, federal officials announced the approval of the first gene therapy treatment for sickle cell disease, which affects close to 100,000 people.
The advancement is good news for the thousands who have inherited blood disorder that causes terrible pain crises and early death.
A second, similar treatment is expected to get the green light before the new year, USA Today reported.
Sickle cell is the most common disease to receive approval by the government for gene therapy treatment, it comes after decades of development and years of approvals for therapy for people who have rarer conditions.
The therapy will be extremely expensive, nearly $2 million per patient, NBC News reported. It will be available only to patients 12 and older who have the most severe form of the disease for the first round.
“We really have to make sure that it is accessible,” said Dr. Rabi Hanna, a pediatric hematologist-oncologist at the Cleveland Clinic who has previously served on the advisory board for Vertex. “This could be an equalizer for people with sickle cell because many patients cannot pursue career options” because of the illness.
“It’s something families have been aware of in the early research stage and they’ve been very patiently waiting for years,” Ferdjallah said. “It’s been eagerly awaited by patients and families, but also by providers and physicians.”
Researchers are still tracking the time to declare the patients “cured” of their sickle cell disease, but after gene therapy, 95% of recipients are symptom-free.
