Pharmaceutical Technology

SEPTEMBER 5, 2022

The treatment is indicated for CF patients who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Earlier, the therapy had obtained FDA approval for usage in CF patients aged two years and above with two F508del mutation copies.

FDA Approval 130

FDA Approval Genotype Clinical Trials Clinical Trials 130

XTalks

APRIL 24, 2023

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. Reference: Chung DC, et al.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Delveinsight

DECEMBER 13, 2020

and ribavirin, were officially approved for HCV GT1 treatment. Even though clinical trial results were promising, adverse events and serious complications were observed. 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

XTalks

DECEMBER 12, 2023

The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinical trials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinical trials.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111

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. Nulibry Trial Results.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

Pharmaceutical Technology

AUGUST 18, 2022

The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study. The open-label, single-arm, 24-month Phase III trials enrolled 41 subjects of the age four to 34 years with non-?0/?0 0 genotypes.

Gene Therapy 246

Gene Therapy Gene Genotype Genetics 246

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