Drugs / Sapropterine
Sapropterine
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hyperphenylalaninemia due to tetrahydrobiopterin deficiency 1 milestone | |||||
| Filed | EU (EMA) | — | hyperphenylalaninemia due to tetrahydrobiopterin deficiency | 2007-11-08 | “has submitted the Marketing Authorization Application (MAA) to the European Medicines Agency (EMEA) for sapropterin dihydrochloride” biomarin.com ↗ |
| Filed phenylketonuria 2 milestones | |||||
| Filed | Other | Kuvan | phenylketonuria | 2009-07-30 | “In addition, yesterday, we submitted the Kuvan NDS to Health Canada.” biomarin.com ↗ |
| Filed | US (FDA) | Kuvan | phenylketonuria | 2007-05-24 | “announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Kuvan(TM) (sapropterin dihydrochloride)” biomarin.com ↗ |
| Filed tetrahydrobiopterin-responsive hyperphenylalaninemia/phenylketonuria 1 milestone | |||||
| Filed | EU (EMA) | Kuvan | 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 |
|---|---|---|---|---|---|---|
| — | NCT07574034 | Jun 2026 → Jun 2028 expected | multisystemic smooth muscle dysfunction syndrome | Gregor Andelfinger | Not yet recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 16
| 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 ↗ |
| 2014-04-14 | BioMarin Pharmaceutical | Regulatory FDA Extends Market Exclusivity Six Months for BioMarin’s Rare Disease Therapy KUVAN(R) (sapropterin dihydrochloride) Powder for Oral Solution and Tablets biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN), a global leader in the development and commercialization of therapies for rare genetic diseases, today announced that the Food and Drug Administration (FDA) granted KUVAN® (sapropterin dihydrochloride) Powder for Oral Solution and Tablets a six-month pediatric exclusivity extension. |
| 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. |
| 2009-06-10 | 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). |
| 2007-05-24 | BioMarin Pharmaceutical | Regulatory BioMarin Submits New Drug Application for U.S. Marketing Authorization of Kuvan for Phenylketonuria (PKU) biomarin.com ↗
BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Kuvan(TM) (sapropterin dihydrochloride), an investigational oral small molecule for the treatment of patients with phenylketonuria (PKU) being developed in partnership with Merck Serono. |
All press releases naming this drug 28 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Sapropterine | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07574034 ↗ |
| Known as | Kuvan | “The molecule we will be using, sapropterin (Kuvan), is already approved and available for other indications.” NCT07574034 ↗ |
| Known as | sapropteride | “We plan to repurpose sapropteride, a synthetic form of tetrahydrobiopterin (BH4), an essential cofactor of phenylalanine hydroxylase (PAH).” NCT07574034 ↗ |
| Known as | sapropterin | “The molecule we will be using, sapropterin (Kuvan), is already approved and available for other indications.” NCT07574034 ↗ |
| Known as | sapropterin dihydrochloride | “Participant will receive KUVAN® (sapropterin dihydrochloride) 100 mg Powder Packets once daily.” NCT07574034 ↗ |