The Pharma Data

AUGUST 9, 2021

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research.

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XTalks

NOVEMBER 10, 2023

“As we strive to help patients with limited or no treatment options, developing innovative treatments in rare diseases is an inspiring challenge and one we have taken on for 70-plus years as a leader in hematology. The study evaluated both prophylactic and on-demand Adzynma ERT compared to plasma-based therapies in cTTP patients.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

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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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XTalks

MAY 17, 2023

The diseases include a type of retinal degeneration caused by mutations to the NPHP5 gene; a neurological disorder called Charcot-Marie-Tooth disease type 4J that damages the peripheral nerves; and multiple sulfatase deficiency that affects the breakdown of sugars and fats.

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XTalks

FEBRUARY 28, 2022

In the official Rare Disease Day 2022 video above, patients from all around the world are heard sharing their stories in their native languages about living with rare diseases like Duchenne muscular dystrophy, cystic fibrosis and Gaucher’s disease among others. Are Most Rare Diseases Genetic?

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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.

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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.

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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.

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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.

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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 FDA approved the drug on November 24. It was accepted for priority review on June 2. The submission was based on the safety and efficacy data of the pivotal Phase II trials 201 and 12-230.

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The Pharma Data

DECEMBER 20, 2020

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. The FDA approval of Klisyri is a significant milestone for Athenex,” said Johnson Lau, chairman and chief executive officer of Athenex. and Europe, including Russia. Most Read Today.

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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

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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XTalks

SEPTEMBER 25, 2024

“NPC is a serious disease that leads to enormous adverse impacts on patients and families. The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”

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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.

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XTalks

SEPTEMBER 17, 2020

While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.

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XTalks

JANUARY 6, 2025

Whether its the integration of nanotechnology in medicine, the evolution of point-of-care (POC) diagnostics or the transformative impact of CRISPR and regenerative medicine, these biotech trends are pushing scientific boundaries and creating new opportunities for businesses and researchers alike.

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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. COVID research has been a primary focus, but that has not put a halt to the development of treatments for other diseases, including rare diseases.

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