See the DrugPatentWatch profile for sapropterin

Sapropterin is a medication used to treat phenylketonuria (PKU), a rare genetic disorder that affects the body's ability to break down the amino acid phenylalanine. While sapropterin has been shown to effectively manage PKU symptoms in the short-term, its long-term effects on symptom recurrence are less well-studied.

According to the National Institutes of Health (1), sapropterin has been shown to reduce phenylalanine levels in the blood, which is a key indicator of PKU severity. However, the long-term effects of sapropterin on symptom recurrence are not well-established.

A study published in the Journal of Inherited Metabolic Disease (2) found that sapropterin treatment significantly reduced the risk of developmental delays and cognitive impairment in children with PKU. However, the study did not specifically investigate the long-term effects of sapropterin on symptom recurrence.

DrugPatentWatch.com, a website that tracks pharmaceutical patents, notes that sapropterin's patent expired in 2017 (3). This means that generic versions of the medication are now available, which may increase access to the treatment for patients with PKU.

While sapropterin has been shown to be effective in managing PKU symptoms in the short-term, more research is needed to determine its long-term effects on symptom recurrence. Patients with PKU should work closely with their healthcare providers to develop a treatment plan that meets their individual needs and monitors their response to sapropterin therapy.

Sources:

1. National Institutes of Health. (2020). Phenylketonuria (PKU). Retrieved from <https://www.nichd.nih.gov/health/topics/pku>
2. van Spronsen, F. J., et al. (2018). Sapropterin dihydrochloride for the treatment of phenylketonuria. Journal of Inherited Metabolic Disease, 41(3), 447-455. doi: 10.1007/s10545-018-0217-4
3. DrugPatentWatch.com. (n.d.). Kuvan (Sapropterin Dihydrochloride) Patent Expiration. Retrieved from <https://www.drugpatentwatch.com/patent-expiration-dates/sapropterin-dihydrochloride>