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

Delveinsight

DECEMBER 13, 2020

This discovery led to the development of diagnostic tests and an effective vaccine, and Blumberg was awarded the Nobel Prize in Physiology or Medicine in 1976 for this discovery. It was approved as a part of therapy for HCV GT1 and GT4 with PegINF-? Epclusa) was developed as the first drug therapy to treat all hepatitis C genotypes.

Genotype 52

Genotype FDA Approval DNA Drugs 52

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 FDA Approval 40

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.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

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.

FDA Approval 52

FDA Approval Production RNA Medicine 52

XTalks

APRIL 24, 2023

According to Dr. Melanie Blank, clinical team leader for General Medicine Branch 1 at the US Food and Drug Administration’s (FDA) Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), the agency is seeing one or two new applications coming in every week for new gene therapies for different diseases.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

MARCH 1, 2024

The FDA has approved many HPV DNA tests that can be used alone (primary HPV testing) or along with Pap tests (co-testing) for cervical cancer screening. Merck’s Gardasil was the first HPV vaccine to be approved by the FDA, and it was approved in 2006.

Vaccination 52

Vaccination Vaccine Life Science FDA Approval 52