Comming Soon Kuvan (Sapropterin) Copay Assistance Program |QuickRx

Without insurance or copay assistance, Kuvan (sapropterin) can be very expensive. As a specialty medication for the rare metabolic disorder PKU, Kuvan costs can be significant for patients and families.

Factors That Affect Kuvan Cost:

• Weight-based dosing: Doses are calculated based on body weight, so costs vary by patient
• Generic availability: Generic sapropterin is available and may cost less than brand Kuvan
• Formulation: Available as tablets (100 mg) and powder packets (100 mg, 500 mg)
• Insurance coverage: Many plans cover Kuvan with varying copay or coinsurance amounts

Contact QuickRx at (917) 830-2525 to explore copay assistance options that may help reduce your Kuvan costs.

Several copay assistance programs may help reduce Kuvan (sapropterin) costs depending on your insurance status and financial situation.

Available Kuvan Copay Assistance Options:

• Manufacturer copay cards: For commercially insured patients
• Patient assistance programs: For uninsured or underinsured patients meeting income requirements
• Independent foundation grants: Organizations supporting patients with PKU and rare metabolic disorders
• State pharmaceutical assistance programs: Varies by state

QuickRx can help determine which programs you qualify for and assist with enrollment at no cost to you.

Medicare patients cannot use manufacturer copay cards due to federal regulations. However, other assistance options may be available for Medicare beneficiaries.

Medicare Patient Options:

• Independent foundation grants: Some foundations provide assistance regardless of insurance type
• State pharmaceutical assistance programs: Many states offer additional help for Medicare patients
• Medicare Extra Help: Low-Income Subsidy program for prescription costs
• Generic sapropterin: May be a lower-cost option compared to brand Kuvan

QuickRx can help Medicare patients explore all available options for reducing Kuvan costs. Call (800) 496-6111 to speak with a patient navigator.

No, QuickRx provides completely FREE copay assistance services for all patients. Our patient navigators help you find and enroll in Kuvan copay assistance programs at no charge.

What QuickRx Does For Free:

• Reviews your insurance coverage and identifies available programs
• Completes copay card and patient assistance applications
• Coordinates with your healthcare provider for required documentation
• Manages ongoing renewals and insurance changes
• Provides continuous support throughout your PKU treatment

Our goal is to help you access the Kuvan you need at the lowest possible cost.

Yes, generic sapropterin is available. Generic versions may cost less than brand-name Kuvan.

Available Sapropterin Products:

• Brand Kuvan: 100 mg tablets, 100 mg and 500 mg powder packets
• Javygtor: Another brand of sapropterin (100 mg tablets, 100 mg and 500 mg powder packets)
• Zelvysia: Another brand of sapropterin (100 mg and 500 mg powder packets)
• Generic sapropterin: Available in both tablet and powder formulations

Your healthcare provider can help determine which formulation is best for you or your child.

Kuvan (sapropterin) is a prescription medication used to treat phenylketonuria (PKU). It is a synthetic form of a natural substance called BH4 (tetrahydrobiopterin).

About Kuvan:

• Drug class: Enzyme Cofactor, Phenylalanine Hydroxylase Activator
• Mechanism: Activates the enzyme phenylalanine hydroxylase (PAH), which helps the body process phenylalanine normally
• FDA approved: December 13, 2007
• Manufacturer: BioMarin Pharmaceutical Inc
• Available forms: 100 mg tablets, 100 mg and 500 mg powder packets for oral solution

Kuvan helps reduce blood phenylalanine levels in patients with BH4-responsive PKU when used along with a phenylalanine-restricted diet.

Kuvan is FDA-approved to treat hyperphenylalaninemia caused by BH4-responsive phenylketonuria (PKU).

About Phenylketonuria (PKU):

• What is PKU: A rare inherited metabolic disorder where the body cannot properly break down phenylalanine, an amino acid found in protein
• Cause: A deficiency in the enzyme phenylalanine hydroxylase (PAH)
• Why treatment matters: Untreated high phenylalanine levels can cause severe neurological damage, including intellectual disability, developmental delays, behavioral problems, and seizures
• Diagnosis: PKU is typically detected through newborn screening

Important:

• Kuvan is used along with a phenylalanine-restricted diet, not instead of it
• Only 25% to 50% of PKU patients respond to sapropterin treatment
• Approved for patients as young as 1 month of age

Kuvan dosing is based on body weight.

Dosing Information:

• Initial dose: 10 to 20 mg/kg once daily
• Maintenance range: 5 to 20 mg/kg once daily
• Frequency: Once daily

Treatment Evaluation:

• Blood phenylalanine levels are checked after 1 week and periodically during the first month
• Dose is adjusted after 1 month based on phenylalanine levels
• If starting at 10 mg/kg and no response, dose may be increased to 20 mg/kg
• If no response after 1 month at 20 mg/kg/day, Kuvan should be discontinued (patient is a “nonresponder”)

Important: Do not change your diet during the initial evaluation period. Your healthcare provider will guide any dietary changes after determining your response to Kuvan.

Kuvan should be taken with food, preferably at the same time each day.

Tablets:

• Swallow tablets whole, OR
• Dissolve tablets in 4-8 oz (120-240 mL) of water or apple juice
• May crush or stir to help dissolution (may take several minutes)
• Tablets may not dissolve completely – this is normal; drink any remaining residue with more water or juice
• Tablets can also be crushed and mixed with applesauce or pudding
• Take within 15 minutes of dissolving

Powder for Oral Solution:

• Dissolve powder in 4-8 oz water or apple juice, OR
• Mix with a small amount of soft foods like applesauce or pudding
• Powder should dissolve completely
• Take within 30 minutes of mixing
• For infants: Can be mixed with phenylalanine-free formula

Missed Dose:

Take as soon as possible, but do NOT take 2 doses on the same day.

Like all medications, Kuvan can cause side effects. Most side effects are mild.

Most Common Side Effects (more than 10% of patients):

• Headache: 15%
• Runny nose (rhinorrhea): 11%

Common Side Effects (1-10% of patients):

• Throat pain: 10%
• Diarrhea: 8%
• Vomiting: 8%
• Cough: 7%
• Nasal congestion: 4%

Tell your doctor about any side effects you experience.

Contact your doctor immediately if you experience any serious side effects while taking Kuvan.

Serious Side Effects Requiring Medical Attention:

• Upper GI problems (esophagitis, gastritis): Severe stomach pain, trouble swallowing, vomiting blood, black/tarry stools, lack of appetite, nausea
• Allergic reactions (including anaphylaxis): Rash, hives, itching, swelling of face/lips/tongue/throat, difficulty breathing, wheezing
• Hyperactivity: Fidgeting, excessive talking, agitation (rare)
• Low phenylalanine levels: Can occur especially in children under 7 on higher doses

Important Warning – GI Inflammation:

Kuvan has been associated with upper GI mucosal inflammation. If untreated, this can lead to esophageal stricture, ulcers, and bleeding. Monitor for signs of stomach or throat problems.

There are no absolute contraindications to Kuvan listed in the US labeling. However, certain patients should use caution.

Use Caution or Avoid If:

• History of anaphylaxis to sapropterin: Not recommended; allergic reactions including anaphylaxis have occurred
• Nonresponders: If blood phenylalanine does not decrease after 1 month at 20 mg/kg/day, Kuvan should be discontinued

Special Considerations:

• Children under 7: At increased risk for low phenylalanine levels (hypophenylalaninemia) at doses of 20 mg/kg/day
• Diet: Must continue phenylalanine-restricted diet while taking Kuvan

Pregnancy:

Kuvan may be used during pregnancy for women with BH4-responsive PKU. Controlling phenylalanine levels during pregnancy is critical to prevent harm to the developing baby. Discuss with your healthcare provider.

Kuvan can interact with several medications. Always tell your doctor about all medications you take.

Medications Requiring Monitoring:

• Levodopa: Kuvan may increase side effects; monitor neurologic status
• PDE-5 inhibitors (sildenafil, tadalafil, vardenafil): Kuvan may increase low blood pressure effects; monitor blood pressure
• Phenobarbital: May decrease Kuvan effectiveness
• Primidone: May decrease Kuvan effectiveness
• Valproic acid: May decrease Kuvan effectiveness
• Dihydrofolate reductase inhibitors (methotrexate, trimethoprim): May decrease Kuvan effectiveness

Your healthcare provider should review all your medications before starting Kuvan.

Regular blood phenylalanine monitoring is essential during Kuvan treatment.

Phenylalanine Monitoring Schedule:

• Baseline: Before starting treatment
• After 1 week: Check response
• First month: Check periodically
• Newly diagnosed infants: Frequently until levels stabilize, then weekly until age 1
• Children 1-12 years: Every 2-4 weeks
• Adolescents and stable adults: Monthly

Target Phenylalanine Range:

Blood phenylalanine goal: 2 to 5.9 mg/dL (120 to 360 micromol/L)

Other Monitoring:

• Signs of upper GI inflammation
• Hyperactivity (especially in children)
• Blood pressure (if taking PDE-5 inhibitors)
• Neurologic status (if taking levodopa)

Yes, Kuvan can be used during pregnancy in women with BH4-responsive PKU. In fact, controlling phenylalanine levels during pregnancy is critical.

Why Phenylalanine Control Matters in Pregnancy:

High maternal phenylalanine levels are associated with serious problems for the developing baby, including:

• Congenital heart disease
• Developmental delay
• Growth retardation
• Microcephaly (small head size)
• Facial abnormalities
• Learning difficulties

Recommendations:

• Normalize phenylalanine levels at least 3 months before conception
• Maintain phenylalanine levels of 2-6 mg/dL throughout pregnancy
• Testing response to Kuvan before pregnancy is recommended when possible
• Close monitoring is required during pregnancy
• Doses may need to decrease in the third trimester

Pregnancy outcomes are comparable to the general population when phenylalanine levels are well controlled.

No, only about 25% to 50% of PKU patients respond to Kuvan treatment.

Understanding Kuvan Response:

• Response to Kuvan is determined by whether blood phenylalanine levels decrease after starting treatment
• Response cannot be predicted by lab testing before treatment – a trial is needed
• If phenylalanine levels do not decrease after 1 month at 20 mg/kg/day, the patient is considered a “nonresponder” and Kuvan should be stopped

What Happens if You Don’t Respond?

• Continue with phenylalanine-restricted diet as your primary treatment
• Other treatment options may be available – discuss with your healthcare provider
• QuickRx can help with copay assistance for other PKU medications as well

Questions about Kuvan copay assistance? Contact QuickRx at (917) 830-2525 or (800) 496-6111.