Sapropterin — Membership Pricing

Other medications in Metabolic

• Tafamidis
• Vestronidase Alfa
• Asfotase Alfa
• Cerliponase Alfa
• Eliglustat
• Inotersen
• Miglustat
• Nitisinone

What Sapropterin is approved for (FDA label)

Sapropterin dihydrochloride tablets 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 tablets are to be used in conjunction with a Phe- restricted diet. Sapropterin dihydrochloride tablets are a phenylalanine hydroxylase activator 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 tablets are to be used in conjunction with a Phe-restricted diet. ( 1 )

Source: Read the full FDA label on DailyMed. Label effective date: August 6, 2026.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Dosage forms and strengths for Sapropterin (FDA label)

Sapropterin dihydrochloride tablets are for oral use. Each tablet contains 100 mg of sapropterin dihydrochloride. Tablets are off white to light yellow, mottled, round shaped tablets debossed with ‘ZS’ on one side and plain on other side. • Tablets: 100 mg sapropterin dihydrochloride. ( 3 )

Source: FDA prescribing information (DailyMed). Set ID: c4d92cce-e899-4f14-b19c-5e17f9922215. SPL document ID: a92c2177-440b-4da4-92f4-e8c809ac64c5. Label effective date: August 6, 2026. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Adverse reactions for Sapropterin (FDA label)

Most common adverse reactions (≥4%) are: headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, and nasal congestion. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Hikma Pharmaceuticals USA Inc. at 1-800-962-8364 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to the rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice.

Source: FDA prescribing information (DailyMed). Set ID: c4d92cce-e899-4f14-b19c-5e17f9922215. SPL document ID: a92c2177-440b-4da4-92f4-e8c809ac64c5. Label effective date: August 6, 2026. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Warnings and precautions for Sapropterin (FDA label)

• Hypersensitivity Reactions Including Anaphylaxis : Sapropterin dihydrochloride tablets are not recommended in patients with a history of anaphylaxis to sapropterin dihydrochloride tablets; discontinue treatment in patients who experience anaphylaxis and initiate appropriate medical treatment. Continue dietary Phe restrictions. ( 5.1 ) • Upper Gastrointestinal Mucosal Inflammation : Monitor patients for signs and symptoms of these conditions including esophagitis and gastritis. ( 5.2 ) • Hypophenylalaninemia : Pediatric patients younger than 7 years treated with sapropterin dihydrochloride tablets doses of 20 mg/kg per day are at increased risk for low levels of blood Phe compared with patients 7 years and older. ( 5.3 ) • Monitoring Blood Phe Levels During Treatment : Ensure adequate blood Phe control and nutritional balance during treatment with sapropterin dihydrochloride tablets.

Source: FDA prescribing information (DailyMed). Set ID: c4d92cce-e899-4f14-b19c-5e17f9922215. SPL document ID: a92c2177-440b-4da4-92f4-e8c809ac64c5. Label effective date: August 6, 2026. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.

Drug interactions for Sapropterin (FDA label)

Table 4 includes drugs with clinically important drug interactions when administered with sapropterin dihydrochloride and instructions for preventing or managing them. Table 4: Clinically Relevant Drug Interactions Levodopa Clinical Impact Sapropterin dihydrochloride 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 [see Warnings and Precautions ( 5.5 )] Intervention Monitor patients for a change in neurologic status. Inhibitors of Folate Synthesis (e.g., methotrexate, valproic acid, phenobarbital, trimethoprim) Clinical Impact In vitro and in vivo nonclinical data suggest that 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. Intervention Consider monitoring blood Phe levels more frequently during concomitant administration.

Source: FDA prescribing information (DailyMed). Set ID: c4d92cce-e899-4f14-b19c-5e17f9922215. SPL document ID: a92c2177-440b-4da4-92f4-e8c809ac64c5. Label effective date: August 6, 2026. Read the full label.

Excerpt from the FDA-approved prescribing information. This is not medical advice; talk to your doctor or pharmacist.