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Sapropterin Side Effects

In Summary

Commonly reported side effects of sapropterin include: headache and rhinorrhea. See below for a comprehensive list of adverse effects.

For the Consumer

Applies to sapropterin: oral powder for solution, oral tablet disintegrating

Side effects requiring immediate medical attention

Along with its needed effects, sapropterin may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking sapropterin:

More common

  • Fever

Incidence not known

  • Burning feeling in the chest or stomach
  • cough
  • difficulty with swallowing
  • dizziness
  • fast heartbeat
  • fidgeting or moving around too much
  • indigestion
  • puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
  • skin rash, hives, or itching
  • stomach upset
  • talking too much
  • tenderness in the stomach area
  • tightness in the chest
  • unusual tiredness or weakness

Side effects not requiring immediate medical attention

Some side effects of sapropterin may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.

Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Less common

  • Stuffy nose

For Healthcare Professionals

Applies to sapropterin: oral powder for reconstitution, oral tablet soluble

General

The more commonly reported adverse effects include headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, and nasal congestion.[Ref]

Nervous system

Two cases of hyperactivity have been received during the postmarketing period; in 1 case, the patient had inadvertently received an overdose of this drug.[Ref]

Very common (10% or more): Headache (15%)

Common (1% to 10%): Dizziness

Frequency not reported: Convulsions, exacerbation of convulsions, overstimulation

Postmarketing reports: Hyperactivity[Ref]

Respiratory

Very common (10% or more): Rhinorrhea (11%), pharyngolaryngeal pain (10%)

Common (1% to 10%): Cough, nasal congestion, pharyngitis, upper respiratory tract infection

Frequency not reported: Respiratory failure

Postmarketing reports: Pharyngitis[Ref]

Metabolic

Not all patients respond to therapy; in clinical trials, 25% to 44% of pediatric phenylketonuria (PKU) patients receiving 20 mg/kg/day did not respond to therapy and 80% of adult and pediatric PKU patients receiving 10 mg/kg/day did not respond to therapy.[Ref]

Very common (10% or more): Low phenylalanine levels, non-response to therapy

Common (1% to 10%): Hypophenylalaninemia[Ref]

Gastrointestinal

Common (1% to 10%): Diarrhea, vomiting, nausea, abdominal pain, upper abdominal pain

Frequency not reported: Gastrointestinal bleeding, esophageal stricture, esophageal ulcer, gastric ulcer, bleeding

Postmarketing reports: Oropharyngeal pain, esophageal pain, gastritis, dyspepsia, abdominal pain, nausea, vomiting[Ref]

Cardiovascular

Frequency not reported: Myocardial infarction[Ref]

Other

Common (1% to 10%): Peripheral edema

Frequency not reported: Contusion, postprocedural bleeding, pyrexia[Ref]

Psychiatric

Common (1% to 10%): Agitation

Frequency not reported: Irritability

Musculoskeletal

Common (1% to 10%): Arthralgia[Ref]

Genitourinary

Common (1% to 10%): Polyuria[Ref]

Hypersensitivity

Postmarketing reports: Hypersensitivity reactions (including serious allergic reactions, anaphylaxis, rash)[Ref]

Dermatologic

Frequency not reported: Rash[Ref]

Hepatic

Frequency not reported: Increased GGT[Ref]

References

1. "Product Information. Kuvan (sapropterin)." BioMarin Pharmaceutical Inc, Novato, CA.

2. Cerner Multum, Inc. "Australian Product Information." O 0

3. Cerner Multum, Inc. "UK Summary of Product Characteristics." O 0

4. Dubois EA, Cohen AF "Sapropterin." Br J Clin Pharmacol 69 (2010): 576-7

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Some side effects may not be reported. You may report them to the FDA.