See the DrugPatentWatch profile for sapropterin

Sapropterin: A Breakthrough Treatment for Phenylketonuria (PKU)

What is Sapropterin?

Sapropterin, also known as Kuvan, is a prescription medication used to treat phenylketonuria (PKU), a rare genetic disorder that affects the body's ability to break down the amino acid phenylalanine (Phe). PKU is caused by a deficiency of the enzyme phenylalanine hydroxylase, which is necessary for the breakdown of Phe.

Who is Typically Prescribed Sapropterin?

Sapropterin is typically prescribed to patients with PKU who have a mild to moderate form of the disorder. These patients have a residual enzyme activity of 2-10% of normal, which means that their bodies can still produce some phenylalanine hydroxylase, but not enough to effectively break down Phe.

What are the Indications for Sapropterin?

Sapropterin is indicated for the treatment of PKU in patients with a residual enzyme activity of 2-10% of normal. It is used to reduce blood Phe levels and to improve the overall health and well-being of patients with PKU.

How Does Sapropterin Work?

Sapropterin works by increasing the activity of the enzyme phenylalanine hydroxylase, which allows the body to break down Phe more effectively. This leads to a reduction in blood Phe levels and a decrease in the accumulation of toxic metabolites.

What are the Benefits of Sapropterin?

The benefits of sapropterin include:

* Reduced blood Phe levels
* Improved overall health and well-being
* Increased quality of life
* Reduced risk of complications associated with PKU, such as intellectual disability and seizures

Who Should Not Take Sapropterin?

Sapropterin is not recommended for patients with PKU who have a residual enzyme activity of less than 2% of normal, as it may not be effective in these patients. Additionally, sapropterin is not recommended for patients with a history of hypersensitivity to the medication or its ingredients.

What are the Common Side Effects of Sapropterin?

The common side effects of sapropterin include:

* Nausea
* Vomiting
* Abdominal pain
* Diarrhea
* Headache
* Fatigue

What are the Less Common Side Effects of Sapropterin?

The less common side effects of sapropterin include:

* Allergic reactions
* Skin rash
* Hives
* Itching
* Swelling

What are the Precautions and Contraindications for Sapropterin?

The precautions and contraindications for sapropterin include:

* Patients with a history of hypersensitivity to the medication or its ingredients
* Patients with a history of kidney or liver disease
* Patients taking other medications that may interact with sapropterin
* Patients with a history of seizures or other neurological disorders

Conclusion

Sapropterin is a breakthrough treatment for PKU that has been shown to be effective in reducing blood Phe levels and improving the overall health and well-being of patients with the disorder. It is typically prescribed to patients with mild to moderate PKU who have a residual enzyme activity of 2-10% of normal. While it is not recommended for patients with a residual enzyme activity of less than 2% of normal, it can be a valuable treatment option for patients who do not respond to other therapies.

Key Takeaways

* Sapropterin is a prescription medication used to treat PKU
* It is typically prescribed to patients with mild to moderate PKU who have a residual enzyme activity of 2-10% of normal
* It works by increasing the activity of the enzyme phenylalanine hydroxylase
* The benefits of sapropterin include reduced blood Phe levels, improved overall health and well-being, and increased quality of life
* The common side effects of sapropterin include nausea, vomiting, abdominal pain, diarrhea, headache, and fatigue

FAQs

1. What is PKU?
PKU is a rare genetic disorder that affects the body's ability to break down the amino acid phenylalanine (Phe).

2. What is sapropterin?
Sapropterin is a prescription medication used to treat PKU.

3. Who is typically prescribed sapropterin?
Patients with mild to moderate PKU who have a residual enzyme activity of 2-10% of normal are typically prescribed sapropterin.

4. How does sapropterin work?
Sapropterin works by increasing the activity of the enzyme phenylalanine hydroxylase, which allows the body to break down Phe more effectively.

5. What are the benefits of sapropterin?
The benefits of sapropterin include reduced blood Phe levels, improved overall health and well-being, and increased quality of life.

Cited Sources

1. DrugPatentWatch.com. (2022). Sapropterin (Kuvan) Patent Expiration. Retrieved from <https://www.drugpatentwatch.com/patent-expiration-dates/sapropterin-kuvan>
2. Kuvan (Sapropterin) [Prescribing Information]. (2022). Retrieved from <https://www.kuvan.com/prescribing-information>
3. National Institutes of Health. (2022). Phenylketonuria (PKU). Retrieved from <https://www.nichd.nih.gov/health/topics/pku>
4. Orphanet. (2022). Phenylketonuria. Retrieved from <https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Lng=EN&Expert=794>
5. PubMed. (2022). Sapropterin. Retrieved from <https://www.ncbi.nlm.nih.gov/pubmed/?term=sapropterin>