Drugs / saproterin dihydrochloride
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hyperphenylalaninemia due to tetrahydrobiopterin deficiency 1 milestone | |||||
| Filed | EU (EMA) | Kuvan | hyperphenylalaninemia due to tetrahydrobiopterin deficiency | 2007-11-08 | “submitted the Marketing Authorization Application (MAA) to the European Medicines Agency (EMEA) for sapropterin dihydrochloride (Kuvan(TM) in the U.S.)” biomarin.com ↗ |
| Filed phenylketonuria 2 milestones | |||||
| Filed | Other | Kuvan | phenylketonuria | 2009-07-29 | “In addition, yesterday, we submitted the Kuvan NDS to Health Canada.” biomarin.com ↗ |
| Filed | US (FDA) | Kuvan | phenylketonuria | 2007-05-24 | “submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Kuvan(TM) (sapropterin dihydrochloride)” biomarin.com ↗ |
| Filed tetrahydrobiopterin (BH4)-deficient hyperphenylalaninemia 1 milestone | |||||
| Filed | EU (EMA) | — | tetrahydrobiopterin (BH4)-deficient hyperphenylalaninemia | — | “In November 2007, Merck Serono submitted a Marketing Authorization Application (MAA) to the EMEA for sapropterin dihydrochloride” biomarin.com ↗ |
| Filed tetrahydrobiopterin-responsive hyperphenylalaninemia/phenylketonuria 1 milestone | |||||
| Filed | EU (EMA) | — | tetrahydrobiopterin-responsive hyperphenylalaninemia/phenylketonuria | — | “In November 2007, Merck Serono submitted a Marketing Authorization Application (MAA) to the EMEA for sapropterin dihydrochloride” biomarin.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 4 | NCT02677870 | Jan → Nov 2018 | phenylketonuria | University Hospitals Cleveland Medical Center | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 15
| Date | Issuer | Release |
|---|---|---|
| 2015-03-30 | BioMarin Pharmaceutical | Results BioMarin Announces One Oral and 7 Poster Presentations at the 2015 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting biomarin.com ↗
There was a statistically significant difference between the baseline and week 13 ADHD-RS total score with Kuvan compared with placebo (p = 0.01). |
| 2014-09-24 | BioMarin Pharmaceutical | Regulatory FDA Receives Paragraph IV Notice Letter for KUVAN(R) (sapropterin dihydrochloride) Tablets biomarin.com ↗ |
| 2013-02-19 | BioMarin Pharmaceutical | Results BioMarin Announces Kuvan Significantly Improves Inattentiveness in Kuvan Responding PKU Patients biomarin.com ↗
Kuvan improved the ADHD-RS (p=0.085), driven by a statistically significant change in the inattention component of the score (p=0.036). |
| 2010-09-01 | BioMarin Pharmaceutical | Results Results From Investigator-Sponsored Trials Presented at SSIEM Conference biomarin.com ↗
Investigators reported that PKU patients taking Kuvan showed improvements in brain functioning, mood and behaviors. |
| 2009-11-05 | BioMarin Pharmaceutical | Regulatory United States Patent Office Issues Patent Covering the Approved Administration of Kuvan With Food biomarin.com ↗
BioMarin Pharmaceutical Inc. announced today that the United States Patent and Trademark Office has issued a patent covering the approved administration of Kuvan® (sapropterin dihydrochloride) with food for the treatment of phenylketonuria (PKU). |
| 2009-06-10 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Priority Review Status From Health Canada biomarin.com ↗
BioMarin Pharmaceutical Inc. announced today that it has received notice of priority review status for Kuvan(R) (sapropterin dihydrochloride) as an oral treatment for patients with phenylketonuria (PKU) from Health Canada. |
| 2009-03-30 | BioMarin Pharmaceutical | Regulatory BioMarin Receives Notice of Allowance for Once Daily Dosing Patent for Kuvan biomarin.com ↗
BioMarin Pharmaceutical Inc. announced today that it has received a notice from the United States Patent Office reporting allowance of claims covering once daily dosing methods for Kuvan(R) (sapropterin dihydrochloride) in the treatment of phenylketonuria (PKU). |
| 2009-02-03 | BioMarin Pharmaceutical | Results Results From Phase 2 Clinical Study of 6R-BH4 in Peripheral Arterial Disease Not Statistically Significant biomarin.com ↗
BioMarin Pharmaceutical Inc. today announced results from its Phase 2 multi-center, randomized, double-blind, placebo-controlled clinical study of 6R-BH4 in patients with symptomatic peripheral arterial disease (PAD). |
| 2008-12-09 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Marketing Approval in the European Union biomarin.com ↗
BioMarin Pharmaceutical Inc. announced today that its partner Merck Serono, a division of Merck KGaA, Darmstadt, Germany, has received marketing approval for Kuvan(R) (sapropterin dihydrochloride) for the treatment of hyperphenylalaninemia (HPA) in phenylketonuria (PKU) or BH4 deficient patients from the European Commission. |
| 2008-09-25 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Positive Opinion From CHMP for European Approval biomarin.com ↗ |
| 2008-07-16 | BioMarin Pharmaceutical | Regulatory Biopten (Sapropterin Dihydrochloride) Approved by Japanese Ministry of Health for the Treatment of PKU biomarin.com ↗ |
| 2007-11-08 | BioMarin Pharmaceutical | Regulatory MAA Submitted to EMEA for European Marketing Authorization of Sapropterin for Hyperphenylalaninemia (HPA) biomarin.com ↗
The MAA filing for sapropterin represents a notable milestone in our joint effort with Merck Serono to bring the first treatment option to PKU and HPA patients around the world," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. |
| 2007-10-17 | BioMarin Pharmaceutical | Results Kuvan, Naglazyme and Aldurazyme Data to Be Presented at the 57th Annual Meeting of the American Society of Human Genetics biomarin.com ↗
Mean daily Phe intake (dietary + supplement) increased from 16.8 to 43.8mg/kg/day with sapropterin (p<0.001) and from 16.3 to 23.5mg/kg/day with placebo (p=0.079). |
| 2007-08-13 | BioMarin Pharmaceutical | Results Positive Kuvan(TM) Pivotal Phase 3 Trial Results Published in The Lancet biomarin.com ↗
Aug 13, 2007 PRNewswire-FirstCall NOVATO, Calif. BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that final results from the Kuvan(TM) (sapropterin dihydrochloride) pivotal Phase 3 clinical trial were published in the August 11, 2007, issue of The Lancet. |
| 2007-07-25 | BioMarin Pharmaceutical | Regulatory Kuvan Receives Priority Review Status from FDA biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing and granted priority review status to the New Drug Application (NDA) for Kuvan(TM) (sapropterin dihydrochloride). |
All press releases naming this drug 29 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | saproterin dihydrochloride | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02677870 ↗ |
| Known as | Kuvan | “The Effectiveness of High-Dose Synthetic BH4 (Saproterin Dihydrochloride or "Kuvan") in Amish PKU Patients” NCT02677870 ↗ |
| Known as | Saproterin | “Saproterin is an approved oral drug developed to reduce blood Phenylalanine (Phe) levels and increase Phe tolerance in BH4 responsive patients with hyperphenylalaninemia.” NCT02677870 ↗ |
| Action | Activate | “activate residual PAH activity” NCT02677870 ↗ |
| Route | Oral | “Medication will be given orally once daily.” NCT02677870 ↗ |
| Target | PAH | “activate residual PAH activity” NCT02677870 ↗ |