What is RETHYMIC?

RETHYMIC is a regenerative tissue-based therapy that 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).

RETHYMIC is engineered human thymus tissue that is implanted in the thigh muscle to help a child with congenital athymia build a functioning immune system to reduce the number of potentially life-threatening infections. In clinical trials, RETHYMIC demonstrated a survival benefit in some children with congenital athymia. RETHYMIC is a regenerative tissue therapy that was developed to address the ultra-rare condition of congenital athymia, for which, previously, there were no existing treatment options. RETHYMIC has a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA and it is the first and only FDA-approved regenerative therapy for pediatric congenital athymia.

How does RETHYMIC Work?

RETHYMIC is intended to reconstitute immune system function in patients with congenital athymia. The stem cells in the bone marrow migrate to RETHYMIC. Over time, these stem cells in RETHYMIC begin to develop into infection-fighting T-cells. Immune reconstitution sufficient to protect from infection is unlikely to develop prior to 6 to 12 months after treatment with RETHYMIC. Once developed, they leave RETHYMIC and enter the bloodstream where they have the ability to interact with other cells.

How Is RETHYMIC Made?

When an infant has cardiac surgery, the surgeon needs to remove some thymus tissue to access the heart. With consent of the infant donor’s parents or guardians, the thymus tissue from pediatric cardiac surgeries is donated for the engineering process to make RETHYMIC for use in patients diagnosed with congenital athymia. The manufacturing is a precisely timed 12- to 21-day engineering process in a facility dedicated to making RETHYMIC.

How is RETHYMIC Administered?

RETHYMIC is surgically implanted in the thigh muscle of a child with pediatric congenital athymia. Children are put under general anesthesia and the procedure is performed in an in-patient setting. A surgeon implants the tissue in the child’s thigh muscle-a rich source of blood that enables the tissue to get the oxygen and nutrients it needs. RETHYMIC is implanted in one or both thigh muscles. The skin incision is typically approximately 5 cm (2 in) in length.

Clinical Trials

The safety and efficacy of RETHYMIC was evaluated in 105 patients across 10 clinical trials.

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After Treatment

Infection control measures should be in place post-treatment until thymic function is established.

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How to Access

RETHYMIC is currently available at one location in Durham, North Carolina. Here you will find information on accessing RETHYMIC

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"I was so consumed by the anxiety and fear. I then realized that this energy is not helping my daughter and I needed to refocus my thinking and get rid of the fear that is serving no one. I think of my daughter as a little trailblazing princess because she has gifted us and even those outside our family with so many life lessons and so much perspective. She’s taught us to focus on what’s really important in life."

MAGGIE, Aunika’s mom

Aunika

Patient with congenital athymia

Supporting the Treatment Journey

Enzyvant CONNECT is here to provide education, resources and support throughout your child’s treatment journey.

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Indication and Important Safety Information
IMPORTANT SAFETY INFORMATION

Infection Control: Immune reconstitution sufficient to protect from infection is unlikely to develop prior to 6-12 months after treatment with RETHYMIC. Immune reconstitution is needed for the body to produce cells in the immune system to fight infection. Your child’s healthcare provider should advise you of infection control measures which should be followed immediately after treatment and until the immune system starts working at a sufficient level. Monitor your child closely for signs of infection, including fever. Your child should be maintained on immunoglobulin replacement and prophylactic antimicrobials until certain criteria are met as determined by your healthcare provider.

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, enlarged lymph nodes, inflammation of the gastrointestinal system and/or diarrhea.

Autoimmune Disorders: Autoimmune-related adverse events occurred in patients treated with RETHYMIC. These events included: low platelets, low white blood cells, protein in urine, low red blood cells, hair loss, poor thyroid function, inflammation of liver, inflammation of the joints, inflammation of the spinal cord, loss of pigment in the skin, eyes and hair, overactive thyroid function, and loss of function of the ovaries. Your healthcare provider will monitor your child regularly including performing blood tests.

Kidney Disease: Treatment with RETHYMIC is a risk factor for death in patients with pre-existing kidney disease. Cytomegalovirus (CMV) Infection: In clinical studies with RETHYMIC, 3 out of 4 patients with pre-existing CMV infection prior to the implantation with RETHYMIC died. Talk to your healthcare provider about the benefits/risks of treatment if your child has pre-existing CMV infection.

Cancer: Due to your child’s weakened immune system, there is increased risk of developing certain cancers. Your child’s healthcare provider will monitor your child through testing for Epstein-Barr virus (EBV) and cytomegalovirus (CMV), which are two viruses that can cause cancer.

Transmission of Serious Infections: Because RETHYMIC is made from human tissue, and animal products are used in the manufacturing process, transmission of infectious diseases may occur. Vaccinations: Your child should not receive any vaccinations until he or she has met certain requirements set by your healthcare provider. Talk to your child’s healthcare provider prior to any vaccinations.

Anti-HLA Antibodies: Prior to 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, he/she will need to receive RETHYMIC from a donor that does not express those HLA proteins.

HLA Typing: If your child has received a hematopoietic cell transplantation (HCT) or a solid organ transplant, they will have a test to look for specific antibodies that could interfere with the effect of RETHYMIC. If they are present, then it will be necessary to receive RETHYMIC from a certain group of donors that do not have these proteins.

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

What are the most common side effects with RETHYMIC?

The most common side effects with RETHYMIC are hypertension (high blood pressure), cytokine release syndrome, rash, hypomagnesemia (low magnesium), renal impairment / failure (decrease of kidney function), thrombocytopenia (low platelets), and graft versus host disease.

These are not all of the possible side effects of RETHYMIC.

Talk to your child’s healthcare provider 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® (allogeneic processed thymus tissue–agdc) 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).

Indication and Important Safety Information
IMPORTANT SAFETY INFORMATION

Infection Control: Immune reconstitution sufficient to protect from infection is unlikely to develop prior to 6-12 months after treatment with RETHYMIC. Immune reconstitution is needed for the body to produce cells in the immune system to fight infection. Your child’s healthcare provider should advise you of infection control measures which should be followed immediately after treatment and until the immune system starts working at a sufficient level. Monitor your child closely for signs of infection, including fever. Your child should be maintained on immunoglobulin replacement and prophylactic antimicrobials until certain criteria are met as determined by your healthcare provider.

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, enlarged lymph nodes, inflammation of the gastrointestinal system and/or diarrhea.

Autoimmune Disorders: Autoimmune-related adverse events occurred in patients treated with RETHYMIC. These events included: low platelets, low white blood cells, protein in urine, low red blood cells, hair loss, poor thyroid function, inflammation of liver, inflammation of the joints, inflammation of the spinal cord, loss of pigment in the skin, eyes and hair, overactive thyroid function, and loss of function of the ovaries. Your healthcare provider will monitor your child regularly including performing blood tests.

Kidney Disease: Treatment with RETHYMIC is a risk factor for death in patients with pre-existing kidney disease. Cytomegalovirus (CMV) Infection: In clinical studies with RETHYMIC, 3 out of 4 patients with pre-existing CMV infection prior to the implantation with RETHYMIC died. Talk to your healthcare provider about the benefits/risks of treatment if your child has pre-existing CMV infection.

Cancer: Due to your child’s weakened immune system, there is increased risk of developing certain cancers. Your child’s healthcare provider will monitor your child through testing for Epstein-Barr virus (EBV) and cytomegalovirus (CMV), which are two viruses that can cause cancer.

Transmission of Serious Infections: Because RETHYMIC is made from human tissue, and animal products are used in the manufacturing process, transmission of infectious diseases may occur. Vaccinations: Your child should not receive any vaccinations until he or she has met certain requirements set by your healthcare provider. Talk to your child’s healthcare provider prior to any vaccinations.

Anti-HLA Antibodies: Prior to 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, he/she will need to receive RETHYMIC from a donor that does not express those HLA proteins.

HLA Typing: If your child has received a hematopoietic cell transplantation (HCT) or a solid organ transplant, they will have a test to look for specific antibodies that could interfere with the effect of RETHYMIC. If they are present, then it will be necessary to receive RETHYMIC from a certain group of donors that do not have these proteins.

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

What are the most common side effects with RETHYMIC?

The most common side effects with RETHYMIC are hypertension (high blood pressure), cytokine release syndrome, rash, hypomagnesemia (low magnesium), renal impairment / failure (decrease of kidney function), thrombocytopenia (low platelets), and graft versus host disease.

These are not all of the possible side effects of RETHYMIC.

Talk to your child’s healthcare provider 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® (allogeneic processed thymus tissue–agdc) 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).