Sapropterin Support Program for Sapropterin Dihydrochloride
What is Sapropterin Dihydrochloride?
Sapropterin Dihydrochloride is a synthetic form of the naturally occurring cofactor, tetrahydrobiopterin (BH4). Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution are indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Sapropterin Dihydrochloride is to be used in conjunction with a Phe-restricted diet.
Treatment with Sapropterin Dihydrochloride should be directed by physicians knowledgeable in the management of PKU. All patients with PKU who are being treated with Sapropterin Dihydrochloride should also be treated with a Phe-restricted diet, including dietary protein and Phe restriction.1
At Cycle, we know that life-changing treatments need life-improving support to match. That’s why we created Cycle Vita, delivering individualized product support every step of the way with the Sapropterin Support Program.
Support available to you from the Sapropterin Support Program
Start your Sapropterin Support Program enrollment here
Download, complete and fax a PDF
The first 24hrs
Your Cycle Vita team will quickly get to work with your insurance provider to help you understand your coverage.
We will coordinate seamlessly between different parties, such as insurance companies, doctors, and pharmacists to help you with your prior authorization and document requirements
We will route your Sapropterin Dihydrochloride therapy to the appropriate specialty pharmacy and follow-up to ensure swift shipment of your medication.
Additional assistance, when required, throughout your journey
In some cases, an appeal may be needed. Where this is the case, our experienced team will work with your insurer and doctor to understand and help you with any Sapropterin Dihydrochloride reimbursement gaps.
If you have commercial insurance, you may be eligible for the Cycle Vita Co-Pay Assistance Program. Commercially insured eligible patients may pay as little as $0. Please contact our Access Specialists to find out more about this and other programs that may be available to you (such as Quick Start, Bridge Program, etc.).
If you agree, our proactive team will remind your doctor when your Sapropterin Dihydrochloride prescription needs to be renewed, in a timely manner, to ensure an uninterrupted supply of your therapy.
We provide product support to help you manage every aspect of your Sapropterin Dihydrochloride therapeutic routine, such as clinical education and 1-to-1 administration training either at home or in-clinic.
Translation services are available on request.
We provide you with on-going personal guidance and education through the duration of your therapy, to support you in meeting your clinic’s nutrition and adherance plan.
What is phenylalanine hydroxylase (PAH) deficiency, traditionally called phenylketonuria (PKU)?
Phenylketonuria (PKU) is an inherited metabolic disorder caused by a deficiency of the phenylalanine hydroxylase (PAH) enzyme needed to process phenylalanine, leading to phenylalanine build up in the blood. It affects roughly 16,500 people in the United States today.2
The disease affects the brain through increased levels of an amino acid called phenylalanine in the blood.2
Untreated individuals with PKU may have severe intellectual and developmental disabilities. It is also estimated that 90% of people with PKU have light coloration, such as blue eyes. Other signs and symptoms of PKU may include having behavioral problems, a small head, delayed growth or short stature, flat feet, eczema or skin rashes, seizures, jerking movements of the arms or legs, tremors, and musty odor of breath, skin or urine. 2,3,4
Newborns at birth with PKU may appear normal..3,
In the United States, PKU is primarily diagnosed around birth, via the Newborn Screening Program. It involves a heel stick blood test taken at least 12 hours, and generally 24-28 hours, after birth.2
Early testing gives the opportunity to start treating affected babies as early as possible.2
Remember, you’re not alone. The Cycle Vita team is here to support you for life.
*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.
- Vockley, J. et al. (2014) ‘Phenylalanine hydroxylase deficiency: diagnosis and management guideline’ Genetics in Medicine 16(2), 188-200. DOI: https://doi.org/10.1038/gim.2013.157
- National PKU Alliance. What is PKU – About PKU. Available at: https://www.npkua.org/What-is-PKU/About-PKU. (Accessed: February 16th 2022.)
- National Institutes of Health. What are common symptoms of phenylketonuria (PKU)? Available at: https://www.nichd.nih.gov/health/topics/pku/conditioninfo/symptoms. (Accessed: February 16th 2022.)
- NORD. Phenylketonuria. Available at: https://rarediseases.org/rare-diseases/phenylketonuria/. (Accessed: February 16th 2022.)
Important Safety Information: Sapropterin Dihydrochloride Tablets for Oral Use and Sapropterin Dihydrochloride Powder for Oral Solution
What Important Information Should I Know About Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution?
- Sapropterin Dihydrochloride can cause serious side effects including:
Severe allergic reactions, including anaphylaxis – Sapropterin Dihydrochloride is not recommended in patients with a history of anaphylaxis to Sapropterin Dihydrochloride. Stop taking Sapropterin Dihydrochloride and get medical help right away if you develop any of these symptoms of severe allergic reaction:
- Wheezing or trouble breathing
- Feeling lightheaded or you faint
Inflammation of the lining of the stomach (gastritis) or esophagus (esophagitis) – Gastritis or esophagitis can happen with Sapropterin Dihydrochloride and may be severe. Patients should be monitored by their doctor for signs and symptoms of upper gastrointestinal mucosal inflammation. Call your doctor right away if you have any of these signs or symptoms:
- Severe upper stomach area (abdominal) discomfort or pain, nausea and vomiting
- Blood in your vomit or stool
- Black, tarry stool
- Difficulty swallowing
- Loss of appetite
- Pain in the throat
Phenylalanine (Phe) levels that are too low – Some PKU patients have experienced low blood Phe during treatment. Some children under the age of 7 years who take high doses of Sapropterin Dihydrochloride each day may experience low Phe levels
Too much or constant activity (hyperactivity) can happen with Sapropterin Dihydrochloride – Tell your doctor if you have any signs of hyperactivity, including:
- Fidgeting or moving around too much
- Talking too much
- It is not possible to know if Sapropterin Dihydrochloride will work for you until you start treatment. Your doctor will check your blood Phe levels when you start taking Sapropterin Dihydrochloride to see if the medicine is working
- All patients with PKU who are being treated with Sapropterin Dihydrochloride should also be treated with a Phe-restricted diet, including dietary protein and Phe restriction
- Your doctor should continue to monitor your blood Phe levels often during your treatment with Sapropterin Dihydrochloride to make sure that your blood Phe levels are not too high or too low. Frequent blood monitoring is recommended in the pediatric population
- Avoid any dietary changes which may affect your blood Phe level. Follow your doctor’s instructions carefully and do not make any changes to your dietary Phe intake without first talking with your doctor. Even if you take Sapropterin Dihydrochloride and your Phe blood levels are not well controlled, you can develop severe neurologic problems
- Tell your doctor as soon as possible if you are sick or have fever as this may increase your blood Phe level
- Take Sapropterin Dihydrochloride exactly as your doctor tells you to
- Do not take 2 doses in a day. If you take too much Sapropterin Dihydrochloride, call your doctor for advice or seek medical attention
Who Should Not Use Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution?
- Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution is not recommended in patients with a history of anaphylaxis to Sapropterin Dihydrochloride or any of the ingredients in Sapropterin Dihydrochloride
What Should I Tell My Doctor Before Taking Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution?
Before taking Sapropterin Dihydrochloride Tablets for Oral Use or Powder for Oral Solution, tell your doctor if you:
- Are allergic to Sapropterin Dihydrochloride or any of the ingredients in Sapropterin Dihydrochloride
- Have poor nutrition or have loss of appetite
- Are pregnant or plan to become pregnant
- Are breastfeeding or plan to breastfeed
Provide your doctor with a complete list of medications you take, including prescription and over-the-counter medications, vitamins, and dietary or herbal supplements. Sapropterin Dihydrochloride can interact with other medications that you are taking. Especially tell your doctor if you take:
- A medication that contains levodopa
- An antifolate medication, such as methotrexate, phenobarbital, trimethoprim, valproic acid
- Sildenafil (Revatio, Viagra), tadalafil (Adcirca, Cialis), vardenafil (Staxyn, Levitra)
Tell your doctor if you are unsure if your medication is one of the above listed. Always share your complete list of medications with your doctor and pharmacist
- What Are the Most Common Side Effects of Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution?
- Runny nose and nasal congestion
- Sore throat
These are not all of the possible side effects of Sapropterin Dihydrochloride. Call your doctor for medical advice about side effects.
For more information, ask your doctor or pharmacist. You are encouraged to report negative side effects of prescription drugs. To report suspected side effects, call Dr. Reddy’s Laboratories Medical Information Call Center at 1-888-DRL-DRUG (1-888-375-3784) or via email to email@example.com or contact the US FDA at 1-800-FDA-1088 (1-800-332-1088) or online at http://www.fda.gov/safety/medwatch
Please refer to the Sapropterin Dihydrochloride Tablets for Oral Use and Sapropterin Dihydrochloride Powder for Oral Solution Package Inserts for full Prescribing Information and Instructions for Use
Indication and Usage for Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution: Sapropterin Dihydrochloride Tablets for Oral Use and Powder for Oral Solution are indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-(BH4-) responsive Phenylketonuria (PKU). Sapropterin Dihydrochloride is to be used in conjunction with a Phe-restricted diet