Drugs / BMN 307
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | phenylketonuria | 2021-09-06 | “BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study.” biomarin.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04480567 | Sep 2020 → Dec 2027 expected | phenylketonuria | BioMarin Pharmaceutical | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2022-02-17 | BioMarin Pharmaceutical | Regulatory BioMarin Provides Updates on Progress in Gene Therapy Programs biomarin.com ↗
In February 2022 , the Company received additional requests from the Food and Drug Administration (FDA) for information needed to resolve the clinical hold of the PHEARLESS Phase 1/2 study of BMN 307 issued in September 2021. |
| 2021-09-06 | BioMarin Pharmaceutical | Regulatory U.S. FDA Placed a Clinical Hold on BMN 307 Phearless Phase 1/2 Gene Therapy Study in Adults with PKU Based on Interim Pre-clinical Study Findings biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study. |
| 2020-10-02 | BioMarin Pharmaceutical | Regulatory BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 biomarin.com ↗
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BMN 307, an investigational gene therapy for the treatment of individuals with PKU. |
| 2020-01-13 | BioMarin Pharmaceutical | Regulatory BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy biomarin.com ↗
Both the FDA and European Medicines Agency have granted BMN 307 Orphan Status. |
| 2019-09-26 | BioMarin Pharmaceutical | Regulatory BioMarin, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in U.K. for Investigational Gene Therapy for PKU biomarin.com ↗
today announced that it has submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN 307 |
| 2018-11-07 | BioMarin Pharmaceutical | Results BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on November 7th in New York biomarin.com ↗
BioMarin shared pre-clinical data of its investigational gene therapy program for PKU using an AAV5 PAH vector, which demonstrated lifetime Phe corrections in mouse models. |
All press releases naming this drug 7 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BMN 307 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04480567 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-Associated Virus Vector-Mediated Gene Transfer” NCT04480567 ↗ |
| Route | Other | “AAV Gene Therapy Infusion” NCT04480567 ↗ |