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What safety measures were taken in sapropterin clinical trials?

See the DrugPatentWatch profile for sapropterin

How were participants screened for eligibility in sapropterin trials?

Screening included PKU patients aged 4 years and older who had a blood phenylalanine concentration of at least 450 µmol/L. Patients also underwent a part-day visit before enrollment to confirm that they responded to sapropterin with a 30% reduction in blood phenylalanine after receiving a single 10 mg/kg dose [1].

What screening tests were monitored during the trials?

During the trials, patients underwent blood phenylalanine measurements at every visit. Blood biopterin levels were checked to exclude tetrahydrobiopterin deficiency. Liver function tests were also performed along with monitoring for adverse events [1].

What happened to the 10% of patients who did not respond?

The 10% of patients who showed less than 30% blood phenylalanine reduction after the initial dose were not included in the efficacy analysis but were still included in the safety population [1].

What side effects did patients report?

Patients reported upper respiratory tract infections, headache, and rhinorrhea. These events occurred at similar rates in the drug-treated and placebo-treated groups [1].

How did researchers handle adverse events?

Researchers recorded every adverse event that occurred during the trials. They attributed some events to the dietary protein intake required in the trials, not to the drug itself [1].

When did patents expire for sapropterin?

The U.S. patent for sapropterin dihydrochloride (Kuvan) expires in 2026 [2].

When does sapropterin exclusivity expire?

Exclusivity for sapropterin ended in 2015 [2].

Who manufactures sapropterin?

BioMarin Pharmaceuticals manufactures sapropterin under the brand name Kuvan [2].

Can biosimilars enter before patent expiry?

Yes. Generic versions of sapropterin dihydrochloride are available in the U.S., generics entered after exclusivity ended but before patent expiry [2].

Why are companies challenging this patent?

Some companies have filed abbreviated new drug applications with paragraph IV certifications challenging the remaining patents [2].



Other Questions About Sapropterin :

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