JAVYGTORTM (sapropterin dihydrochloride) Tablets for Oral Use and Powder For Oral Solution

What is JAVYGTOR?

JAVYGTOR (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). JAVYGTOR is to be used in conjunction with a Phe-restricted diet.1

Treatment with JAVYGTOR should be directed by physicians knowledgeable in the management of PKU.1 All patients with PKU who are being treated with JAVYGTOR should also be treated with a Phe-restricted diet, including dietary protein and Phe restriction.1

At Cycle, we know that a patient’s wellbeing not only depends on life-changing treatments, but also a support system which may help improve quality of life. That’s why we created Cycle Vita, delivering individualized product support every step of the way with JAVYGTOR.

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Support available to you with JAVYGTOR

Start your JAVYGTOR enrollment here

Download and print the enrollment form,
share it with your doctor and discuss why
you think JAVYGTOR may be suited to
your needs.

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 between different parties, such as insurance companies, doctors, and pharmacists to help you with your prior authorization and document requirements.

We will route your JAVYGTOR 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 for insurance coverage may be needed. Where this is the case, our experienced team will work with your insurer and doctor to understand the requirements and help you with any JAVYGTOR 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 the Bridge Program and the New Patient Free Trial etc.).

Important notice

A free supply of JAVYGTOR is available to allow patients to begin therapy immediately while Cycle Vita secures appropriate benefit verification and authorization. Bridge Program may also be requested for existing patients who are temporarily experiencing disruption in therapy due to insurance coverage.

Important notice

If you agree, our proactive team will remind your provider when your JAVYGTOR prescription needs to be renewed, in a timely manner to ensure your therapy is not interrupted.

We provide product support to help you manage every aspect of your JAVYGTOR therapeutic routine, such as clinical education and one-on-one administration training either at home or at your healthcare provider’s office.

Translation services are available upon request.

We provide you with on-going personal guidance and education through the duration of your therapy, to support you in reaching your provider’s nutritional goals and adherence plan.

What is phenylalanine hydroxylase (PAH) deficiency, traditionally called phenylketonuria (PKU)?

Phenylketonuria (PKU) is an inherited disorder caused by a deficiency of the phenylalanine hydroxylase (PAH) enzyme needed to process phenylalanine, which is found in all foods containing protein. High levels of phenylalanine can cause brain damage.2 The incidence of PKU is highest amongst Caucasians, occurring in approximately 1 in 10,000 births.3

Untreated individuals with PKU appear normal at birth, but if they do not restrict the phenylalanine in their diet, they can develop severe intellectual and developmental disabilities. Other signs and symptoms of PKU may include behavioral problems, slow growth, small head size, eczema or skin rashes, seizures, jerking movements of the arms or legs and musty odor of breath, skin or urine. Signs and symptoms also include fair skin and blue eyes, due to the body’s failure to transform phenylalanine into melanin, the pigment responsible for a person’s coloring.4

All 50 US states require newborns to be screened for PKU, which is how nearly all cases of PKU are diagnosed. A test is performed using a few drops of blood from a newborn’s heel to determine the level of phenylalanine in the blood.5

Remember, you are not alone. The Cycle Vita team is here to support you at every step of your treatment process.

Resources

Footnote
*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.

JAVYGTOR is a trademark of Cycle Pharmaceuticals Limited in the United States.

References

  1. JAVYGTOR (sapropterin dihydrochloride). Prescribing Information. Dr Reddy’s Laboratories Ltd.
  2. National Institutes of Health. Phenylketonuria (PKU). Available at: https://www.nichd.nih.gov/health/topics/pku. (Accessed August 21st 2023.)
  3. Williams, R. A. et al. 2008. Phenylketonuria: An Inborn Error of Phenylalanine Metabolism. The Clinical Biochemist Reviews. 29(1) 31-41.
  4. National Institutes of Health. What are common symptoms of phenylketonuria (PKU)? Available at: https://www.nichd.nih.gov/health/topics/pku/conditioninfo/symptoms. (Accessed: August 21st 2023.)
  5. National Institutes of Health. How do health care providers diagnose phenylketonuria (PKU)? Available at: https://www.nichd.nih.gov/health/topics/pku/conditioninfo/diagnosed (Accessed August 21st 2023.)

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Indication and Important Safety Information: JAVYGTOR Tablets for Oral Use and JAVYGTOR Powder for Oral Solution

INDICATION

JAVYGTOR is 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). JAVYGTOR is to be used in conjunction with a Phe-restricted diet.

IMPORTANT SAFETY INFORMATION

Treatment with JAVYGTOR should be directed by physicians knowledgeable in the management of PKU. All patients with PKU who are being treated with JAVYGTOR should also be treated with a Phe-restricted diet, including dietary protein and Phe restriction. Prolonged exposure to elevated blood Phe levels can result in severe neurologic damage in PKU patients.

During treatment with JAVYGTOR, monitor blood Phe levels frequently to ensure adequate blood Phe level control, especially in pediatric patients. Also, active management of dietary Phe intake is required to ensure adequate Phe control and nutritional balance. Biochemical response to JAVYGTOR treatment should be determined through a therapeutic trial.

Patients should be advised to notify their physicians in cases of overdose.

WARNINGS AND PRECAUTIONS

  • Hypersensitivity Reactions Including Anaphylaxis: JAVYGTOR is not recommended in patients with a history of anaphylaxis to SAPROPTERIN DIHYDROCHLORIDE. Hypersensitivity reactions, including anaphylaxis and rash, have occurred. Signs of anaphylaxis include wheezing, dyspnea, coughing, hypotension, flushing, nausea, and rash. Discontinue JAVYGTOR treatment in patients who experience anaphylaxis, and initiate appropriate medical treatment. Continue dietary protein and Phe restrictions in patients who experience anaphylaxis.
  • Upper Gastrointestinal Mucosal Inflammation: Gastrointestinal (GI) adverse reactions suggestive of upper GI mucosal inflammation have been reported with JAVYGTOR. Serious adverse reactions included esophagitis and gastritis. If left untreated, these could lead to severe sequelae including esophageal stricture, esophageal ulcer, gastric ulcer, and bleeding, and such complications have been reported in patients receiving SAPROPTERIN DIHYDROCHLORIDE. Monitor patients for signs and symptoms of upper GI mucosal inflammation.
  • Hypophenylalaninemia: Some patients receiving SAPROPTERIN DIHYDROCHLORIDE have experienced hypophenylalaninemia (low blood Phe) during treatment. Children younger than 7 years old treated with JAVYGTOR doses of 20 mg/kg per day are at an increased risk for low levels of blood Phe compared with older patients.
  • Monitoring Blood Phe Levels During Treatment: Prolonged elevations of blood Phe levels in patients with PKU can result in severe neurologic damage, including severe intellectual disability, developmental delay, microcephaly, delayed speech, seizures, and behavioral abnormalities. Conversely, prolonged levels of blood Phe that are too low have been associated with catabolism and endogenous protein breakdown, which has been associated with adverse developmental outcomes. Active management of dietary Phe intake while taking sapropterin dihydrochloride is required to ensure adequate Phe control and nutritional balance. Monitor blood Phe levels during treatment to ensure adequate blood Phe level control. Frequent blood monitoring is recommended in the pediatric population.
  • Lack of Biochemical Response to JAVYGTOR: Not all patients with PKU respond to treatment with JAVYGTOR. Biochemical response to JAVYGTOR treatment cannot generally be pre-determined by laboratory testing (e.g., molecular testing), and should be determined through a therapeutic trial (evaluation) of JAVYGTOR response.
  • Interactions with Levodopa: There have been reports of interactions with levodopa causing seizures, exacerbation of seizures, over-stimulation, and irritability. Monitor patients who are receiving levodopa for a change in neurologic status during treatment with JAVYGTOR.
  • Hyperactivity: There have been post-marketing reports of hyperactivity with administration of SAPROPTERIN DIHYDROCHLORIDE. Monitor patients for hyperactivity.

ADVERSE REACTIONS

  • Most common: The most common adverse reactions (incidence ≥4%) were headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, and nasal congestion.

The following adverse reactions have been reported during post-approval use of sapropterin dihydrochloride:

  • Hypersensitivity reactions including anaphylaxis and rash. Most hypersensitivity reactions occurred within several days of initiating treatment;
  • Gastrointestinal reactions: esophagitis, gastritis, oropharyngeal pain, pharyngitis, esophageal pain, abdominal pain, dyspepsia, nausea, and vomiting;
  • Hyperactivity

DRUG INTERACTIONS

  • Levodopa – JAVYGTOR may increase the availability of tyrosine, a precursor of levodopa. Neurologic events were reported post-marketing in patients receiving sapropterin and levodopa concomitantly for a non-PKU indication. Monitor patients for a change in neurologic status.
  • Inhibitors of Folate Synthesis – Drugs that inhibit folate synthesis may decrease the bioavailability of endogenous BH4 by inhibiting the enzyme dihydrofolate reductase, which is involved in the recycling (regeneration) of BH4. This reduction in net BH4 levels may increase Phe levels. Frequently monitor blood Phe levels when co-administering JAVYGTOR with medications known to inhibit folate synthesis, such as methotrexate, valproic acid, phenobarbital, trimethoprim.
  • Drugs Affecting Nitric Oxide-Mediated Vasorelaxation – Both JAVYGTOR and PDE- 5 inhibitors (such as sildenafil, vardenafil, or tadalafil) may induce vasorelaxation. A reduction in blood pressure could occur. Monitor patients for hypotension when co-administering JAVYGTOR with medications known to affect nitric oxide–mediated vasorelaxation such as PDE-5 inhibitors.

USE IN SPECIFIC POPULATIONS

  • Pregnancy: There are no well-controlled clinical studies of Sapropterin Dihydrochloride in pregnant women.
  • Lactation: There are insufficient data to assess the presence of sapropterin in human milk and no data on the effects on milk production.
  • Pediatric Use: Pediatric patients with PKU, ages 1 month to 16 years, have been treated with sapropterin dihydrochloride in clinical trials. The efficacy and safety of sapropterin dihydrochloride have not been established in neonates.
  • Geriatric Use: Clinical studies of sapropterin dihydrochloride in patients with PKU did not include patients aged 65 years and older. It is not known whether these patients respond differently than younger patients.

For more detailed information, please refer to the full Prescribing Information at: www.JAVYGTOR.com/PI

To report SUSPECTED ADVERSE REACTIONS, contact Dr. Reddy’s Laboratories, Inc. at 1-888-375-3784 or by email: medinfo@drreddys.com, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch