The Food and Drug Administration on Thursday approved a treatment that uses gene therapy to treat severe hemophilia A, a rare and sometimes fatal blood disorder.
The new drug Roctavian could spare people with the severe form of the disease frequent injections for a lifetime. The drug’s maker estimates that about 2,500 of the estimated 6,500 Americans with severe hemophilia A will be eligible to receive the drug after initial approval.
The treatment uses a single infusion to insert the genes people with hemophilia A lack and can last for years, according to data presented to regulators. It’s also the latest FDA-approved gene therapy, which some scientists believe heralds the future of treating thousands of diseases.
The FDA has approved gene therapies for the following diseases: sickle cell anemia and spinal muscular atrophy. The use of this technology is relatively rare, also because gene therapies are among the most expensive drugs in the world. Roctavian will cost $2.9 million for a single infusion; According to Portal, Hemgenix, which treats another form of hemophilia, costs $3.5 million per application. Still, scientists say the technology will be a crucial part of 21st-century medicine.
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