Skip to Content

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

As well as its needed effects, sapropterin may cause unwanted side effects that require medical attention.

Major Side Effects

If any of the following side effects occur while taking sapropterin, check with your doctor immediately:

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

Minor Side Effects

Some sapropterin side effects may not need any medical attention. As your body gets used to the medicine these side effects may disappear. Your health care professional may be able to help you prevent or reduce these side effects, but do check with them if any of the following side effects continue, or if you are concerned about them:

More common:
  • Diarrhea
  • headache
  • runny nose
  • sore throat
  • vomiting
Less common:
  • Stuffy nose

For Healthcare Professionals

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

General

In phenylketonuria (PKU) trials, the most common side effects were headache, rhinorrhea, pharyngolaryngeal pain, diarrhea, vomiting, cough, and nasal congestion.

In clinical trials for non-PKU indications, a different formulation was used in patients with disorders other than PKU; some had underlying neurologic disorders or cardiovascular disease. Common side effects were headache, peripheral edema, arthralgia, polyuria, agitation, dizziness, nausea, pharyngitis, abdominal pain, upper abdominal pain, and upper respiratory tract infection. Serious and severe side effects included convulsions, exacerbation of convulsions, dizziness, gastrointestinal bleeding, postprocedural bleeding, headache, irritability, myocardial infarction, overstimulation, and respiratory failure.[Ref]

Nervous system

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

Very common (10% or more): Low phenylalanine levels
Common (1% to 10%): Hypophenylalaninemia[Ref]

Gastrointestinal

Common (1% to 10%): Diarrhea, vomiting, nausea, abdominal pain, upper abdominal pain
Frequency not reported: Gastrointestinal 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. Dubois EA, Cohen AF "Sapropterin." Br J Clin Pharmacol 69 (2010): 576-7

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

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

It is possible that some side effects of sapropterin may not have been reported. These can be reported to the FDA here. Always consult a healthcare professional for medical advice.

Disclaimer: Every effort has been made to ensure that the information provided is accurate, up-to-date and complete, but no guarantee is made to that effect. In addition, the drug information contained herein may be time sensitive and should not be utilized as a reference resource beyond the date hereof. This material does not endorse drugs, diagnose patients, or recommend therapy. This information is a reference resource designed as supplement to, and not a substitute for, the expertise, skill , knowledge, and judgement of healthcare practitioners in patient care. The absence of a warning for a given drug or combination thereof in no way should be construed to indicate safety, effectiveness, or appropriateness for any given patient. Drugs.com does not assume any responsibility for any aspect of healthcare administered with the aid of materials provided. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the substances you are taking, check with your doctor, nurse, or pharmacist.

Hide