First Gene Therapy for Hearing Loss Approved
The FDA approved a new treatment called Otarmeni on Thursday. It is the first gene therapy for genetic hearing loss.
The treatment targets a very rare form of genetic deafness. It helps people with a faulty OTOF gene.
This affects about 50 babies in the United States each year. After the approval Regeneron made a deal with President Trump.
The therapy will be offered for free. Reports say the free pricing was part of a larger drug pricing deal at the White House.
Sources say patients may still pay out-of-pocket costs for the surgery needed to get the therapy. One parent said their child was born deaf. After treatment the child can now hear.
Klear Note — Gene therapy treats disease by fixing faulty genes. This is the first gene therapy approved for hearing loss caused by genetic mutations.
Key Terms 5
- FDA United States Food and Drug Administration; approves new medicines and treatments
- gene therapy treatment that fixes or replaces a faulty gene in the body
- OTOF gene a gene that when faulty can cause a person to be born deaf
- out-of-pocket costs money a patient pays themselves that insurance does not cover
- Regeneron a United States company that makes medicines and treatments