SILVER SPRING, Md., October 8, 2021 / PRNewswire / – Today, the United States Food and Drug Administration approved Rethymic for the treatment of pediatric patients with congenital athymia, a rare immune disorder. Rethymic is the first thymus tissue product approved in the United States
“Today’s action marks the first FDA approval of a therapy to treat this very rare and devastating disease in children,” said Pierre Marc, MD, Ph.D., Director of the Center for Biologics Evaluation and Research, FDA. “We remain committed to helping advance the development of safe and effective medical products for patients with rare diseases – an area where the needs are so critical. ”
Congenital athymia is a rare immune disorder in which a child is born without a thymus, an organ that plays a vital role in helping the body learn to fight infections. Children affected by this disease usually die within the first two years of life and can have repeated, often fatal, infections because they lack adequate functional T cells (a kind of white blood cell that fights infection).
Rethymic is made up of human allogeneic thymic tissue (derived from a donor) which is processed and cultured and then implanted into patients to help restore immunity (improve immune function) in athymic patients. The dosage is individualized by the patient, determined by the area of the Rethymic slices and the patient’s body surface area. Rethymic is not indicated for the treatment of patients with severe combined immunodeficiency (SCID).
The safety and efficacy of Rethymic have been established in clinical studies which included 105 patients, aged 1 month to 16 years, each of whom received a single administration of Rethymic, from 1993 to 2020. Rethymic improved performance survival of children with congenital athymia, and most children treated with this product survived at least two years. Children treated with Rethymic who survive beyond the first year usually survive long term. Rethymic also reduced the frequency and severity of infections over time.
The most common side effects in patients who have received Rethymic include high blood pressure, cytokine release syndrome, low levels of magnesium in the blood, rash, low platelets, and graft versus host disease. .
Because Rethymic is derived from human tissue, it carries a risk of transmitting infectious diseases. Based on effective donor selection procedures and product manufacturing processes, the risk of infectious disease transmission is low, but not completely eliminated.
It takes six months or more to restore immune function in treated patients; therefore, it is important that until immune reconstitution occurs, patients continue to take strict precautions to prevent infection and healthcare providers should treat accordingly.
This app has received a voucher for rare pediatric diseases by the FDA. A description of the Rare Pediatric Disease Designation and Voucher programs can be found in the Industry Guide: Rare Pediatric Rare Disease Priority Review Vouchers.
The FDA has granted Rethymic approval to Enzyvant Therapeutics, Inc.
Healthcare professionals should report all serious adverse events suspected of being associated with the use of any drug, biologic, or device to the FDA’s MedWatch reporting system or by calling 1-800-FDA-1088.
Media contact: Veronika Pfaeffle, 310-301-2576
Consumer inquiries: Email or 888-INFO-FDA
The FDA, an agency of the U.S. Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and safety of drugs, vaccines, and other biologicals for human and veterinary use, as well as medical devices. . The agency is also responsible for the safety and security of our country’s food supply, cosmetics, dietary supplements, products that emit electronic radiation, and the regulation of tobacco products.
SOURCE US Food and Drug Administration