See the DrugPatentWatch profile for sapropterin
Sapropterin, also known as tetrahydrobiopterin, is a drug used to reduce the symptoms of phenylketonuria (PKU), a genetic disorder that can cause intellectual disability [1]. The development of sapropterin has been marked by several milestones:
1. **Discovery of Tetrahydrobiopterin**: In the 1950s and 1960s, scientists discovered tetrahydrobiopterin, the active form of biopterin, which is a cofactor for several enzymes involved in the metabolism of neurotransmitters and amino acids [2].
2. **Understanding of PKU**: In the 1960s, PKU was found to be caused by a deficiency in the enzyme phenylalanine hydroxylase, which requires tetrahydrobiopterin as a cofactor [3].
3. **Clinical Trials**: In the late 1990s and early 2000s, clinical trials were conducted to test the efficacy of sapropterin in treating PKU [4]. These trials showed that sapropterin could reduce blood phenylalanine levels in some PKU patients.
4. **FDA Approval**: In 2007, the US Food and Drug Administration (FDA) approved sapropterin for the treatment of PKU [5].
5. **Patent Expiration**: The patent for sapropterin expired in 2019, which may lead to increased accessibility and affordability of the drug [6].
Sources:
1. [Mayo Clinic. Phenylketonuria.](
https://www.mayoclinic.org/diseases-conditions/phenylketonuria/symptoms-causes/syc-20353994)
2. [NIH. Tetrahydrobiopterin.](
https://www.ncbi.nlm.nih.gov/books/NBK535435/)
3. [Genetics Home Reference. Phenylketonuria.](
https://medlineplus.gov/genetics/condition/phenylketonuria/)
4. [ClinicalTrials.gov. Sapropterin Dihydrochloride in the Treatment of Phenylketonuria.](
https://clinicaltrials.gov/ct2/show/NCT00005275)
5. [FDA. KUVAN (sapropterin dihydrochloride) Tablets.](
https://www.fda.gov/drugs/postmarket-drug-safety-information-patients-and-providers/kuvan-sapropterin-dihydrochloride-tablets)
6. [DrugPatentWatch. Sapropterin.](
https://www.drugpatentwatch.com/drugs/sapropterin)