Clinical trial results

RETHYMIC greatly improved survival for children with congenital athymia

The efficacy and safety of RETHYMIC were evaluated across 10 clinical trials that enrolled a total of 105 children with a follow-up of up to 25.5 years. Of those enrolled, the efficacy of RETHYMIC was evaluated in 95 children.

For children treated with RETHYMIC:

An estimated

77%

were alive after 1 year

An estimated

76%

were alive after 2 years

Those who were alive 1 year after treatment had a survival rate of

94%

with a median follow-up of 10.7 years

RETHYMIC significantly reduced the number of infections over time:

38%

fewer children experienced an infection between 6 and 12 months after treatment with RETHYMIC vs the first 6 months after treatment

In a 2-year analysis, fewer children experienced an infection and the average number of infections per child decreased in the second year after treatment compared to the first year after treatment

The safety of RETHYMIC was demonstrated in 105 children across 10 clinical trials

The most common side effects of RETHYMIC were:

  • Hypertension (high blood pressure)
  • Cytokine release syndrome (overactive immune system)
  • Hypomagnesemia (low magnesium)
  • Rash
  • Renal impairment/failure (decrease of kidney function)
  • Thrombocytopenia (low platelets)
  • Graft versus host disease (a condition in which a person’s T cells attack their own body)

Of the 105 children in the clinical studies, 29 died, including 23 in the first year. The majority of deaths in the first year after receiving RETHYMIC were due to infections.

These are not all the side effects that can or may occur with RETHYMIC treatment. Please see below.

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Indication and Important Safety Information
Important Safety Information

Infection Control: Immune reconstitution sufficient to protect from infection usually develops between 6-12 months after treatment with RETHYMIC. For some children, it may take up to 2 years. Taking medications that prevent infection and other infection control measures, such as hand washing and isolation, should be continued until your child’s doctor confirms that immune function has been reconstituted through immune tests and the criteria for discontinuing certain medications have been met. Immediately report signs and symptoms of infection, such as fever, to your child’s doctor.

Graft versus Host Disease (GVHD): RETHYMIC may cause or make pre-existing GVHD worse. Your child will be monitored for GVHD and treated if needed. Symptoms of GVHD may include fever, rash, swollen lymph nodes, inflammation of the digestive system, and/or diarrhea.

Autoimmune Disorders: Autoimmune-related side effects (when your immune system attacks healthy cells by mistake) occurred in patients treated with RETHYMIC. These included low platelets, white blood cells, or red blood cells; protein in the urine; hair loss; poor thyroid function; inflammation of the liver, joints, or spinal cord; loss of pigment in the skin, eyes and hair; overactive thyroid function; and loss of function of the ovaries. Your doctor will monitor your child regularly.

Kidney Disease: Children with kidney disease have a higher risk of death when treated with RETHYMIC.

Cytomegalovirus (CMV) Infection: In clinical studies, 4 out of 4 patients with CMV infection prior to treatment with RETHYMIC died.

Cancer: Due to your child’s weakened immune system, there is an increased risk of developing blood cancer. Your child’s doctor will monitor your child through testing for Epstein-Barr virus and CMV, which are two viruses that can cause cancer.

Transmission of Serious Infections and Transmissible Infectious Diseases: Because RETHYMIC is made from human tissue, and animal products are used in the manufacturing process, transmission of infectious diseases may occur.

Vaccine Administration: Notify your child’s doctor to evaluate your child’s immune status before receiving vaccinations. Live virus vaccines should not be given until the doctor determines that your child has met criteria for and received inactivated vaccines.

Anti-HLA Antibodies: Before receiving RETHYMIC, your child will be tested for HLA antibodies, which are proteins that may be present in your child’s blood. If your child has these antibodies, your child should receive RETHYMIC from a specific donor, which will be determined by your child’s doctor.

HLA Typing: If your child received a hematopoietic cell transplantation (HCT) or a solid organ transplant, testing to match your child with RETHYMIC from a compatible donor is required. Children who have received an HCT are at an increased risk of developing GVHD after RETHYMIC if the HCT donor does not fully match with RETHYMIC.

Deaths: Of the 105 children who participated in the clinical studies, 29 patients died, including 23 in the first year after implantation of RETHYMIC.

The most common side effects are high blood pressure, cytokine release syndrome, rash, low magnesium, decrease in kidney function, low platelets, and GVHD.

These are not all the possible side effects of RETHYMIC. Talk to your child’s doctor about any side effect that bothers your child or does not go away.

You are encouraged to report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/safety/medwatch.

Indication

RETHYMIC® is indicated for immune reconstitution in pediatric patients with congenital athymia.

RETHYMIC is not for use in patients who have been diagnosed with severe combined immunodeficiency (SCID).

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Sumitomo Pharma is a trademark of Sumitomo Pharma Co., Ltd., used under license. SUMITOMO PHARMA is a trademark of Sumitomo Pharma Co., Ltd., used under license. SUMITOMO is a registered trademark of Sumitomo Chemical Co., Ltd., used under license. Sumitomo Pharma America, Inc. is a U.S. subsidiary of Sumitomo Pharma Co., Ltd. RETHYMIC ®, RETHYMIC Connect™, and its logos are trademarks of Sumitomo Pharma Switzerland GmbH. © 2024 Sumitomo Pharma Switzerland GmbH. All rights reserved. RET-US-0380-24    11/2024