What is HT-1?
Tyrosinemia Type 1, often referred to as HT-1, is an ultra-rare disorder that leaves a person unable to fully break down the amino acid tyrosine.
This causes a dangerous build-up of toxic metabolites in the body – which can lead to serious liver and kidney problems – those affected require a special diet and medication (nitisinone, sometimes known as NTBC) to control the disorder. It is estimated that there are less than 200 patients in the US.
Tyrosinemia Type 1 is a lifelong disorder. It is an autosomal recessive genetic condition, which means the baby inherits a defective gene from each parent. Unaffected adults are often unaware they carry a defective gene until they have an affected child. People born with Tyrosinemia Type 1 cannot metabolize the amino acid tyrosine, meaning they are missing an essential enzyme that helps to break down protein in the food they eat.
In the US Tyrosinemia Type 1 is mostly diagnosed at birth. Newborn screening typically involves a heel stick blood test. However, a positive screening result may not be definitive. Your healthcare provider may order additional blood and urine tests to confirm the diagnosis. Early testing gives the opportunity to start treating your baby as early as possible, minimizing the risk of further complications.
Monitoring and testing
What to expect
While managing Tyrosinemia Type 1 can be challenging, you should know that you are not alone.
Your healthcare team, made up of your doctor, dietitian, genetic counselors, social workers and other clinicians will help you or your child thrive at every stage of development.
- Immediately upon diagnosis start therapy with NTBC (e.g., NITYR) and recommend dietary therapy with medical foods and a protein-restricted diet to limit phenylalanine and tyrosine intake while supporting normal growth1
- Monitor health, including growth and body weight, draw blood and collect urine to ensure lab values are within expected bounds1
- Recommend a diet that helps restrict phenylalanine and tyrosine to maintain concentrations within the treatment range1
- Adjust the diet as appropriate as you or your child grows
- Ensure your diet and nutrition supports normal growth and development
Depending on the stage you are at in your journey with Tyrosinemia Type 1, other providers may be added to your healthcare team (e.g., ophthalmologists, neurologists).
How NITYR can help
We understand that a new diagnosis or transitioning to a new medicine may be concerning, but you are not alone. We are here for you to provide continuous support, answer any questions and ensure your NITYR Experience is seamless throughout.
Cycle Vita support for eligible* patients includes:
- Quick Start Program: A free supply of NITYR that allows you to begin therapy immediately while Cycle Vita secures appropriate benefit verification and authorization. Quick Start may also be requested if you’re an existing patient temporarily experiencing disruption in therapy due to insurance coverage
- Co-pay Assistance: Cycle Vita Co-pay Assistance is available to eligible commercially insured patients, who may pay as little as $0
- Patient Nutrition Program: Our Patient Nutrition Program reinforces your clinic’s dietary plan with product support that contributes to better outcomes
NITYR helps children to be treated,
from the start
Like many parents and caregivers before you, the first few days, weeks and months after a Tyrosinemia Type 1 diagnosis can be challenging.
Please keep in mind that babies born with Tyrosinemia Type 1 can be treated right from the start by being compliant with their medication and diet. This is not always going to be easy, but you are not alone, we will be here every step of the way to help you.
NITYR has administration options to suit every need.
Most children are able to swallow tablets from the age of three. Talk to your doctor before changing how your child takes NITYR.
- Give as suspension
- Crush and mix with applesauce
- Swallow whole
With NITYR you can create a suspension whenever and wherever you want:
- Less medication complexities with no refrigeration required
- Greater flexibility with on the go preparation
- Accurate dosing each and every time
When your child is ready, they can swallow the very small baby aspirin-sized tablet too.2 Talk to your doctor about which option is best suited to your child’s needs.
Gaining independence. NITYR helps children transition seamlessly to tablets
Now that your child is a little older, he or she may be ready to start swallowing tablets whole. In fact, many children prefer baby aspirin-sized tablets over solutions and suspensions.3
This marks one of the first steps in giving your child a little more responsibility so they can begin controlling their condition for themselves.
NITYR Tablets:
- Are very small, easy to carry, and can be discreetly carried in a backpack2
- Can be taken with an empty stomach or with your meal, you choose
- Can be taken once daily for qualified patients 5 years and older, which may provide additional flexibility to your day***
Of course if you and your child feel more comfortable, you can use the medication to create a suspension or mix with applesauce.
*** For patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stabledosage of nitisinone. Ask your Healthcare Provider for more information.
Embracing independence. NITYR helps teenagers to become healthy adults
The teenage years are marked by increasing freedom, the start of real independence, responsibility, and hectic schedules that include plenty of time spent with friends, at school, and doing extracurricular activities.
Teenagers also want to control what they eat and how they manage their Tyrosinemia Type 1. NITYR may make it easier for them to control their condition.
NITYR can help teenagers to start self-managing and become more independent with:
- Very small and discreet tablets, giving teenagers the possibility to take NITYR on the go2
- Stable at room temperature, so teenagers don’t have to worry about refrigeration – ideal for school life, trips to the beach or holidays with friends
- Can be taken with an empty stomach or with your meal, you choose to suit your schedule
- Qualified patients can take NITYR tablets once daily, which may give teenagers more flexibility in their lives***
*** For patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage of nitisinone. Ask your Healthcare Provider for more information.
Embracing responsibility. NITYR gives adults one less thing to worry about
Balancing college, work, home life, children, bills… The responsibilities of adulthood can seem overwhelming.
Designed for convenience, NITYR may help make things easier by providing the flexibility adults need to spend more time with their studies, family or advancing their careers, without the burden of “take with food” restrictions or refrigeration requirements.
NITYR can help adults with:
- The convenience of once-daily dosing for qualified patients, may give more flexibility in their busy schedule***
- Very small tablets which may provide discretion when taking medication around friends and colleagues on the go2
- Stable at room temperature, so there’s no need to worry about refrigeration
- Can be taken with an empty stomach or with your meal, its their choice
*** For patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage of nitisinone. Ask your Healthcare Provider for more information.
+1 (888) 360-8482
References
1. Chinsky JM, Singh R, Ficicioglu C, van Karnebeek CDM, Grompe M, Mitchell G, Waisbren S, Gucsavas-Calikoglu M, Wasserstein MP, Coakley K, Scott CR, Diagnosis and treatment of tyrosinemia type I: a US and Canadian consensus group review and recommendations. Genet Med. 2017 Dec;19(12):1-16. doi: 10.1038/gim.2017.101
2. Data on file: REF-00041.
3. van Riet-Nales DA, Schobben AF, Vromans H, Egberts TC, Rademaker CM. Safe and effective pharmacotherapy in infants and preschool children: importance of formulation aspects. Arch Dis Child. 2016 Jul;101(7):662–9. doi: 10.1136/archdischild-2015-308227
* Some areas of support may not be accessible to all patients. Eligibility criteria may apply to ensure compliance with all applicable federal and state requirements, and benefits may be limited to commercially insured patients only. For more detailed information about eligibility, terms and conditions, please contact the Cycle Vita team at +1 (888) 360-8482.