The CNPV program leveraged to push groundbreaking results of gene therapy treatment.
The Food and Drug Administration (FDA) has announced approval for Otarmeni (lunsotogene parvec-cwha), a first-of-its-kind treatment for hearing loss. Otarmeni can be used for patients with severe to profound sensorineural hearing loss “associated with molecularly confirmed biallelic variants in the OTOF gene.”
Without the otoferlin (OTOF) protein, the inner ear’s tiny hair cells are unable to transmit sound signals to the brain, resulting in total deafness pre-birth. This treatment is described as “the first-ever dual adeno-associated virus (AAV) vector-based gene therapy.” Eighty percent of the children in the study experienced hearing improvement in the clinical trial.
After a publication in the New England Journal of Medicine was released, highlighting the groundbreaking results of Otarmeni, the FDA granted an accelerated review of the gene therapy, under a national priority voucher. This is the sixth approval under the Commissioner’s National Priority Voucher (CNPV) pilot program.
As the Lord Leads, Pray with Us…
- For Commissioner Makary to be led by the Lord as he oversees the Food and Drug Administration and its approvals.
- For FDA officials and researchers as they conduct trials and studies to measure the risks and benefits of new treatments.
- For the children and their families who have a possibility of reversing hearing loss through this new therapy.
Sources: Department of Health and Human Services
