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Supporting the treatment journey

Enzyvant CONNECT is here to provide personalized support throughout your child's treatment journey with RETHYMIC.

For more information, call 844-ENZCNCT (844-369-2628), Monday through Friday, 8:00 AM to 8:00 PM ET. or download the Patient Enrollment Form and take it to your next appointment with your doctor. Your signature along with your doctor's, will be needed to complete the enrollment process.

Meet your dedicated Patient Access Specialist

When you enroll in Enzyvant CONNECT, you will be connected with a Patient Access Specialist who will assist you throughout the treatment journey including:

Find information, downloads and links to patient organizations to help you and your family.

Read More

a guide through the
congenital athymia journey
for caregivers

Here you’ll find information from the time of diagnosis to what your child may experience after treatment with RETHYMIC® (allogeneic processed thymus tissue–agdc). Families that have been in the same place as you have shared their experiences so we can share them with you.

Click the buttons below to start your journey and learn more.



The diagnosis of congenital athymia
can be overwhelming.

Learn the facts about congenital athymia. Here you will find answers to common questions families had at the time of diagnosis.


While not the typical course, Brynlee had open-heart surgery when she was five days old and that’s when they noticed she didn’t have a thymus. When the newborn screening test came back, she had zero T cells.

Michelle, Brynlee’s mom

Congenital Athymia Diagnosis

What is congenital athymia?

Congenital athymia is an ultra-rare condition in which a child is born without a thymus.

How is congenital athymia diagnosed?

Congenital athymia is usually first detected in newborn screening for Severe Combined Immunodeficiency (SCID) which is required in all 50 states. This SCID test will show if there is a low number of T cells. Congenital athymia is not SCID, but the test for SCID will show if there is a low number of T cells, which is seen in congenital athymia. Based on the results of this newborn screening, your doctor will determine what additional testing may be needed. After a positive newborn SCID screening result, a laboratory technique called flow cytometry is used to verify low naive T cells and strengthen the diagnosis of congenital athymia.

What is a thymus and why is it important?

The thymus is an organ that sits on top of the heart and plays an important role in how the immune system works. The immune system is made of organs, cells and proteins that work together throughout the body to fight infections. The thymus helps the body produce T-cells, which are white blood cells that fight off viruses, bacteria, fungi, and parasites. These cells also help the body remember the invaders it fights, which is how the body builds immunity to illnesses.

What happens when the thymus is missing?

Children with congenital athymia can face repeated infections because they do not have enough working T cells to fight the invaders off. Without working T cells, children can also be affected by autoimmune conditions, when the body’s immune system attacks and destroys healthy body tissue by mistake. These infections and autoimmune conditions can be fatal.

What causes congenital athymia?

Congenital athymia usually occurs in babies who have certain genetic problems but for some there is no known cause. Here are conditions that have been associated with congenital athymia:

DiGeorge syndrome- This syndrome results when a part of chromosome 22 is missing.

CHARGE syndrome – This syndrome consists of medical and physical conditions that differ child to child. The CHARGE acronym comes from the first letter of some of the more common features seen in children:

  • CColoboma or defects in the way the eye looks
  • HHeart defects
  • AAtresia of the choanae or blocked nasal breathing passages
  • RRetardation of growth and development
  • GGenital underdevelopment
  • EEar abnormalities

FOXN1 deficiency –This condition affects the production of critical proteins which results in a severely compromised immune system and problems with the growth of hair and nails.

Understanding Congenital Athymia

Download

Congenital Athymia Questions to Ask your Doctor

Download

Indication and Important Safety Information

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).

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 doctor 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 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, 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 doctor 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 doctor 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 doctor 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 doctor. Talk to your child’s doctor 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 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.

US–1900023

US–2100094


Supportive Care

Upon diagnosis of congenital athymia, isolation measures are important for your child to avoid contact with germs that can cause infection. Here are recommendations on what to do to minimize risk of infection.


The isolation precautions we took were significant. As soon as we found out she didn’t have T cells, the hospital put her in isolation. At home we did everything we could to keep her in that bubble. Anytime we would go out, when we came home, we would change, shower and disinfect. Anything that was bought from the store we would wipe down with disinfectant wipes.

Jason, Aunika’s dad

Supportive Care

Children with congenital athymia need special care. One of the most important things that your doctor will discuss with you is isolation—both in the hospital and at home.

How do I minimize the chance of infection?

Always talk to your doctor to create a plan for what is right for you and your family. Here are some things that your doctor may recommend:

  • Restrict visitors in the home
  • Encourage frequent handwashing in the home
  • Wipe down any items coming into the home
  • Remind family members that they should shower and change clothes upon re-entry to the home from work or school
  • If possible, work with your doctor to obtain masks, gowns, and gloves for family members and visitors to wear
  • Homeschool other children in the family and work from home, if possible – If siblings must attend school, ask the school to notify you of any outbreaks or illnesses that siblings might bring home
  • Work with doctors to prevent exposure to sick children at medical appointments
How do I minimize infection risk outside our home?

Talk to your doctor on recommended practices but here are a few to consider:

  • Should anyone in your household get sick, have a plan in place for that family member to stay outside of the home, such as with a friend/family member until they are well enough to come home
  • Talk to those around you, family, friends, teachers, about what congenital athymia is and what it means for your family
Caring for a child with Congenital Athymia

Download

Indication and Important Safety Information

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).

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 doctor 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 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, 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 doctor 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 doctor 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 doctor 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 doctor. Talk to your child’s doctor 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 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.

US–1900023

US–2100094


Congenital athymia treatment

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).


Be patient. It’s going to be a journey, lean on your family and friends…and advocate for your child as best as you can.

Amanda, Jada’s mom

Congenital athymia treatment

About RETHYMIC® (allogenenic processed thymus tissue-agdc)

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 the RETHYMIC processed tissue 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 for a surgery that takes about 2 hours.
  • 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.

RETHYMIC Clinical Trials

The safety and efficacy of RETHYMIC was evaluated in 105 patients across 10 clinical trials. The effectiveness of RETHYMIC was evaluated in 95 of those patients with congenital athymia in 10 clinical trials with follow-up of up to 25.5 years.

The RETHYMIC clinical trials measured survival after treatment. Results showed:


For patients who were alive at 1 year after RETHYMIC treatment, the survival rate was 94% at a median follow-up time of 10.7 years.

RETHYMIC significantly reduced the number of infections over time.

What are the most common side effects?

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

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)
  • Graft versus host disease

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

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

A Caregivers Guide To RETHYIMC

Download

What to Expect After Treatment

How long will it take for my child to start making T cells?

Immune reconstitution is the creation of naïve T cells. Immune reconstitution sufficient to protect from infection is unlikely to develop prior to 6-12 months after treatment with RETHYMIC. Your child will remain immune compromised during the process of immune reconstitution. Infection control measures should be followed until the development of thymic function can be established.

When can current medications be discontinued?

Your child may be on several medications, including some for the prevention of infections related to congenital athymia.These medications may include immunoglobulin, antibiotics, and antifungals to prevent or treat infections. Some children are also on immunosuppressants to reduce symptoms of immune dysregulation due to congenital athymia.

Once T cells reach certain levels, your doctor will conduct tests to understand if and when your child can discontinue certain medications.

When can my child be vaccinated?

Your child should not receive any vaccinations until he or she has met certain requirements set by your doctor. Talk to your child’s doctor prior to any vaccinations.

When will strict isolation end for my family?

Relaxing strict isolation is a decision your child’s doctor will help you make based on your child’s specific needs. Infection control measures, including strict isolation should continue until immune reconstitution is achieved. Your doctor will monitor T cell levels and other markers of immune reconstitution and advise you of when it is safe to relax strict isolation.

Indication and Important Safety Information

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).

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 doctor 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 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, 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 doctor 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 doctor 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 doctor 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 doctor. Talk to your child’s doctor 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 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.

US–1900023

US–2100094


How To Access Treatment

RETHYMIC® (allogeneic processed thymus tissue–agdc) is currently available at one location in Durham, North Carolina. Here you will find information on accessing RETHYMIC.


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

How To Access Treatment

If you and your child’s doctor decide that RETHYMIC® (allogeneic processed thymus tissue–agdc) is right for your child, your doctor should contact the treatment location.

You and your doctor can enroll your child in Enzyvant CONNECT, a program that provides support through the treatment journey. Click here for more information.

Where can my child receive RETHYMIC?

RETHYMIC is available at one location in Durham, North Carolina.

Why is there only one location that can administer RETHYMIC?

The FDA approval allowed for RETHYMIC to be available at one approved treatment location.

Why is the supply of RETHYMIC limited?

Supply is limited by the availability of donor thymus tissue. Each lot of RETHYMIC is made one patient at a time.

When will my child be treated?

Enzyvant does not make treatment decisions. Enzyvant’s role is to supply RETHYMIC. Please talk to your physician to determine if RETHYMIC is right for your child and, if so, the timing of treatment.

What happens while waiting for RETHYMIC?

While waiting for product supply, your healthcare team will ensure that the single site of treatment has all the lab tests and information they need. During this time it is critical to follow your physician’s recommendations on medications and isolation.

Work with your physician to enroll in Enzyvant CONNECT, which is a program that provides support through the treatment journey. Click here for more information.

Indication and Important Safety Information

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).

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 doctor 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 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, 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 doctor 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 doctor 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 doctor 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 doctor. Talk to your child’s doctor 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 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.

US–1900023

US–2100094


Supporting the treatment journey

Enzyvant CONNECT is here to provide personalized support throughout your child’s treatment journey with RETHYMIC® (allogeneic processed thymus tissue–agdc).

The Enzyvant CONNECT team will assist families with:
  • Navigating insurance information
  • Connecting to financial assistance programs
  • Providing educational resources
Meet Your Enzyvant CONNECT Team

Patient Access Specialist – Your dedicated Patient Access Specialist can assist you with:

  • Insurance information – Benefit investigation/verification to determine insurance coverage, any related requirements, and your out-of-pocket responsibility.
  • Financial assistance programs – Identify and put you in touch with programs that may be able to help with financial and/or support assistance.
  • Educational resources – Provide you with information about congenital athymia and treatment, and advocacy resources to support you along the treatment journey.

Support Liaison –The Support Liaison is another resource available to everyone enrolled in Enzyvant CONNECT. The Support Liaison can provide you with information about congenital athymia and available educational resources. The Support Liaison is here to help you before and after RETHYMIC treatment. The Support Liaison can also connect families with advocacy organizations.

How to Enroll

To enroll in Enzyvant CONNECT, you will fill out the Patient Enrollment Form with your healthcare provider. Your healthcare provider will fax the form to Enzyvant CONNECT. Once received, your Patient Access Specialist will contact you to provide an overview of the program and get started.

Patient Enrollment Form Download

For more information, call 844-ENZCNCT (844-369-2628), Monday through Friday, 8:00 AM to 8:00 PM ET. or download the Patient Enrollment Form and complete it with the help of your doctor to get started. Your signature along with your doctor’s, will be needed to complete the enrollment process.


Bring your own version of fire in the belly to the situation. Be your baby’s number one advocate and be in it, really in it as much as you can give. Keep doing your research, keep investigating and pay attention to what your child is saying, even though they may not be speaking. Never stop fighting for your child because of what might be possible.

Melyza, Sidney’s mom

Caregiver Resources

Find information, downloads and links to patient organizations to help you and your family.

Congenital athymia resources

  • Congenital Athymia: Questions to Ask your Doctor

    Download

  • Information for Friends and Family

    Download

  • Understanding Congenital Athymia

    Download

  • Caring for a Child with Congenital Athymia

    Download

RETHYMIC resources

  • A Caregivers Guide to RETHYMIC

    Download

  • RETHYMIC: Questions to Ask your Doctor

    Download

Organizations

There are many resources available to help you take care of yourself and your family. You should always talk with your healthcare provider if you have any questions. The following organizations are independent of Enzyvant and may also be helpful:

Jeffrey Modell Foundation

This nonprofit organization helps families and caregivers affected by immunodeficiency disorders find support, education, awareness,
advocacy and care.

Visit Site

Global Genes

This organization pursues positive change and aims to connect, empower, and inspire the rare disease community.

Visit Site

Immune Deficiency Foundation

Here you can find valuable information and insights related to immunodeficiency disorders, including the Patient and Family Handbook for Primary Immunodeficiency Diseases.

Visit Site

National Organization of Rare Disorders

This patient advocacy organization, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through the programs of education, advocacy, research and patient services.

Visit Site

Indication and Important Safety Information

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).

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 doctor 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 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, 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 doctor 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 doctor 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 doctor 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 doctor. Talk to your child’s doctor 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 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.

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Indication and Important Safety Information

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).

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 doctor 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 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, 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 doctor 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 doctor 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 doctor 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 doctor. Talk to your child’s doctor 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 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.

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