Pharmaceutical Technology

SEPTEMBER 5, 2022

The US Food and Drug Administration (FDA) has approved the expanded usage of Vertex Pharmaceuticals ’ Orkambi (lumacaftor/ivacaftor) to treat cystic fibrosis (CF) in children aged 12 months to under 24 months. It enrolled 46 children aged one to under two years with the F/F genotype.

FDA Approval 130

FDA Approval Genotype Clinical Trials Clinical Trials 130

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia 0 genotypes. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Genotype In-Vivo 246

XTalks

JANUARY 21, 2025

TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Date of FDA approval : March 29, 2024 Approved for : To detect exposure to human parvovirus B19. TriClip size comparison photo. Photo courtesy of Abbott Medical.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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FDA Approval Production RNA Medicine 52

XTalks

MARCH 3, 2021

Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. Joffe, MD, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, in a press announcement from the FDA. “The

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

DECEMBER 12, 2023

Additionally, the potential for accelerated approval of therapies via biomarker surrogate endpoints is introducing new complexities into the mix. “In This approval was based on the observed reduction of plasma neurofilament light chain (NfL) in patients who received tofersen.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111

Roots Analysis

APRIL 28, 2024

Absence of Biomarkers for Diagnosis: Due to the absence of genotype-phenotype correlations and prognostic markers, there have been challenges in the diagnosis and treatment of rare kidney diseases. Insufficient Model Organisms: Limitations of these model organisms includelong generation time and strain effects.

Genetics 40

Genetics Genotype Development Research 40