XTalks

MAY 12, 2023

In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

Drug Discovery World

NOVEMBER 14, 2023

“The FDA remains deeply committed in our efforts to help facilitate the development and approval of safe and effective therapies for patients with rare diseases,” said Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research. Without treatment, cTTP is ultimately fatal.

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

XTalks

AUGUST 6, 2020

The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

MARCH 3, 2021

The approval was granted to BridgeBio Pharma, Inc. BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. The announcement was made on Rare Disease Day, which raises awareness about the impact of rare diseases on patients and their caregivers. Nulibry Trial Results.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

The Pharma Data

NOVEMBER 29, 2020

The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. The submission was based on the safety and efficacy data of the pivotal Phase II trials 201 and 12-230. The FDA approved the drug on November 24. It was accepted for priority review on June 2.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

XTalks

DECEMBER 29, 2022

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

DECEMBER 20, 2020

It was approved on December 15, 2020. The submission included data from two positive pivotal Phase III trials. Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. and Europe, including Russia. It results in patches of thick, scaly or crusty skin.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Drugs 52

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

DECEMBER 22, 2021

percent, according to a report by BCC Research. In attempts to mask RNA from immune attack, researchers Drew Weissman and Katalin Karikó at the University of Pennsylvania switched out uridine, which is one of the four bases of the RNA code, for pseudouridine. billion by 2026 at a compound annual growth rate (CAGR) of 16.8

Life Science 98

Life Science RNA Vaccination Vaccine 98

Drug Discovery World

MARCH 19, 2024

Lenmeldy (atidarsagene autotemcel) has become the first Food and Drug Administration (FDA)-approved gene therapy for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetic Disease 52

pharmaphorum

JANUARY 10, 2022

The first mRNA-based vaccines were brought onto the market in 2020, with Pfizer/BioNtech and Moderna receiving emergency use authorisation from the US Food and Drug Administration (FDA) for the prevention of COVID-19. The breakthrough was hailed as a revolution for disease treatment and particularly a major success against the pandemic.

RNA 107

RNA Vaccination Vaccine Pharma Companies 107

Drug Discovery World

FEBRUARY 22, 2024

Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research. ARUK 3 adds that while the UK government increased funding in dementia research from £28.2m years it takes to complete an entire cancer clinical trial.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

NOVEMBER 25, 2020

Ongoing research was put on the backburner and scientists began to focus on understanding the virus and assessing what medications could be used against it. The mRNA vaccine candidate developed by Pfizer and Germany-based BioNTech demonstrated 95% efficacy in clinical trials.

Vaccination 52

Vaccination Vaccine Genetic Disease Genetics 52