The Alabama Department of Public Health announced Monday that a child enrolled in ADPH’s ALL Kids program is eligible to receive a newly approved gene therapy for a rare form of inherited deafness.
ALL Kids is a low-cost health care coverage program administered by ADPH for eligible children under 19.
The child, who has profound hearing loss caused by mutations in the OTOF gene, which encodes the otoferlin protein produced in the inner ear, is among the first patients eligible to receive lunsotogene parvec-cwha, marketed as Otarmeni, the first and only FDA-approved gene therapy for hearing loss.
The therapy will be provided at Boston Children’s Hospital, one of the original clinical trial sites for the gene therapy.
“At ADPH, the mission of our ALL Kids program is to ensure Alabama’s children have access to the care they need, including highly specialized treatments that may not be available close to home,” said Dr. Karen Landers, Alabama chief medical officer. “We are grateful this child has the opportunity to receive a therapy that offers new hope for children and families affected by this rare genetic condition.”
The FDA approved Otarmeni in April. The therapy is the first approved drug designed to address the underlying genetic cause of hearing loss rather than simply treating its effects.
The drug’s manufacturer, Regeneron Pharmaceuticals, has announced it will provide the gene therapy at no cost to clinically eligible U.S. patients. According to ADPH, families still may be responsible for costs associated with the procedure and related medical care, depending on their insurance coverage.
ALL Kids is Alabama’s Children’s Health Insurance Program, or CHIP, which provides low-cost health care coverage to children in families with incomes too high to qualify for Medicaid but too low to afford private insurance. Through a partnership with Blue Cross and Blue Shield of Alabama, ALL Kids connects families with a network of health care providers to ensure enrolled children have access to preventive and specialty care services, as well as other medically necessary treatments.
ADPH wrote that the Alabama Early Hearing Detection and Intervention Program collaborates with the Alabama Early Intervention System to ensure that all infants with hearing loss are identified “as early as possible and provided with timely and appropriate audiological, educational, and medical intervention.”
The program works with the Alabama Early Intervention System to ensure all infants with hearing loss are identified by 3 months of age and enrolled in the system before they are 6 years old.
“This milestone demonstrates the importance of ensuring children have access to innovative medical care as new therapies become available,” Landers said.
“Through a shared commitment between the ADPH and Blue Cross and Blue Shield of Alabama, we are proud that ALL Kids can help Alabama families access life-changing treatment options when medically appropriate,” she continued. “ADPH is grateful to the entire team at Boston Children’s and its Department of Otolaryngology and Communication Enhancement for caring for this patient and others who will benefit in the future.”
