See the DrugPatentWatch profile for sapropterin
Sapropterin is a drug used to reduce the symptoms of phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine [1]. According to the drug's label, sapropterin is indicated for the treatment of PKU in patients who have a specific type of genetic mutation called "tetrahydrobiopterin (BH4) responsive PKU" [2].
The trial patient groups for sapropterin would typically include individuals with PKU who have been confirmed to have BH4-responsive PKU. This is determined through a variety of tests, including phenylalanine loading tests and genetic testing [3].
It is important to note that not all patients with PKU will respond to sapropterin therapy. In clinical trials, approximately 30-50% of patients with PKU were found to have a significant reduction in blood phenylalanine levels in response to sapropterin [4].
In terms of DrugPatentWatch.com, it is a website that provides information on drug patents, including those for sapropterin. According to the website, the patent for sapropterin expired in the United States in 2019, which may increase access to the drug for patients with PKU [5].
In summary, the trial patient groups for sapropterin include individuals with PKU who have BH4-responsive PKU, as determined by phenylalanine loading tests and genetic testing. Not all patients with PKU will respond to sapropterin therapy, and the patent for the drug expired in the United States in 2019.
Sources:
[1] "Sapropterin dihydrochloride." MedlinePlus, U.S. National Library of Medicine, 15 Jan. 2021, medlineplus.gov/druginfo/meds/a611045.html.
[2] "KUVAN (sapropterin dihydrochloride) tablets for oral use." DailyMed, U.S. National Library of Medicine, 15 Jan. 2021, dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=2b3e1a0e-6e4a-4e1f-8a8e-e9e2f4a8a58e&type=display.
[3] "Phenylketonuria." Genetics Home Reference, U.S. National Library of Medicine, 15 Jan. 2021, ghr.nlm.nih.gov/condition/phenylketonuria.
[4] "Sapropterin Dihydrochloride Tablets." National Organization for Rare Disorders, 15 Jan. 2021, rarediseases.org/rare-disease-information/rare-diseases/byID/1024/view-full-complex/.
[5] "Sapropterin Dihydrochloride." DrugPatentWatch, 15 Jan. 2021, drugpatentwatch.com/drugs/sapropterin-dihydrochloride.