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Sapropterin: A Breakthrough Treatment for Phenylketonuria (PKU)

What is Phenylketonuria (PKU)?

Phenylketonuria (PKU) is a rare genetic disorder that affects the body's ability to process the amino acid phenylalanine. If left untreated, PKU can lead to severe intellectual disability, seizures, and behavioral problems. The disorder is caused by a deficiency of the enzyme phenylalanine hydroxylase, which is necessary for the breakdown of phenylalanine.

What is Sapropterin?

Sapropterin is a medication used to treat PKU by increasing the production of phenylalanine hydroxylase. It is a synthetic form of tetrahydrobiopterin (BH4), a co-factor that is essential for the enzyme's function.

When was Sapropterin Approved for Use?

Sapropterin was approved by the United States Food and Drug Administration (FDA) in 2007 for the treatment of PKU in patients with mild hyperphenylalanemia (elevated levels of phenylalanine in the blood).

How Does Sapropterin Work?

Sapropterin works by increasing the production of phenylalanine hydroxylase, which allows the body to break down phenylalanine more efficiently. This leads to a decrease in the levels of phenylalanine in the blood, reducing the risk of complications associated with PKU.

Benefits of Sapropterin

Sapropterin has been shown to be effective in reducing phenylalanine levels in the blood, improving cognitive function, and reducing the risk of seizures and behavioral problems. It is also well-tolerated and has a low risk of side effects.

Who Can Take Sapropterin?

Sapropterin is approved for use in patients with mild hyperphenylalanemia, which is typically defined as a blood phenylalanine level of 120-360 μmol/L. It is not approved for use in patients with severe hyperphenylalanemia or those who require a diet that is very low in phenylalanine.

Dosing and Administration

Sapropterin is available in tablet form and is typically taken orally, usually with food. The recommended dose is 5-20 mg/kg/day, divided into two or three doses.

Monitoring and Side Effects

Patients taking sapropterin should have their blood phenylalanine levels monitored regularly to ensure that the medication is effective and to detect any potential side effects. Common side effects of sapropterin include headache, nausea, and vomiting.

Conclusion

Sapropterin is a breakthrough treatment for PKU that has been shown to be effective in reducing phenylalanine levels in the blood and improving cognitive function. It is an important addition to the treatment options available for patients with PKU and has the potential to improve the quality of life for those affected by this disorder.

FAQs

1. What is the recommended dose of sapropterin?
Answer: The recommended dose of sapropterin is 5-20 mg/kg/day, divided into two or three doses.

2. Who can take sapropterin?
Answer: Sapropterin is approved for use in patients with mild hyperphenylalanemia, which is typically defined as a blood phenylalanine level of 120-360 μmol/L.

3. How often should blood phenylalanine levels be monitored?
Answer: Blood phenylalanine levels should be monitored regularly to ensure that the medication is effective and to detect any potential side effects.

4. What are the common side effects of sapropterin?
Answer: Common side effects of sapropterin include headache, nausea, and vomiting.

5. Is sapropterin available in other forms besides tablets?
Answer: No, sapropterin is only available in tablet form.

Cited Sources:

1. Food and Drug Administration. (2007). Kuvan (sapropterin dihydrochloride) tablets, for oral use. Retrieved from <https://www.accessdata.fda.gov/drugsatfda_docs/label/2007/021843s000lbl.pdf>
2. DrugPatentWatch.com. (n.d.). Sapropterin dihydrochloride patents. Retrieved from <https://www.drugpatentwatch.com/patent/US-7345154-B2>
3. National Institutes of Health. (n.d.). Phenylketonuria (PKU). Retrieved from <https://ghr.nlm.nih.gov/condition/phenylketonuria>