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

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).

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Gene Therapy Gene FDA Approval Dermatology 97

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. Ultragenyx Pharmaceuticals has an orthinine transcarbamylase (OTC) activator drug in Phase III trials. Future Possibilities.

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XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Rett syndrome is characterized by a distinctive pattern of symptoms that gradually emerge after a period of seemingly normal development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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pharmaphorum

SEPTEMBER 28, 2022

The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market. “We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease,” said Nia Tatsis, Vertex’ chief regulatory and quality officer, in a statement.

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XTalks

FEBRUARY 28, 2022

Related: UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD. Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. How is a Rare Disease Defined? Are Most Rare Diseases Genetic?

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XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Verifying the biomarker’s clinical validity for use as a surrogate endpoint in rare disease research is another hurdle which is generally a longer-term goal.

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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.

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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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XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.

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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 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. Typically, it is children who suffer from the disease.

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The Pharma Data

DECEMBER 20, 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. Approval demonstrates our ability to execute upon the entirety of the drug development and registration process.

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XTalks

DECEMBER 22, 2021

The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. They can be developed and adapted quickly. This did the trick and set the stage for the development of viable mRNA-based therapeutics. RNA Therapeutics.

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The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Sigilon Therapeutics

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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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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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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).

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pharmaphorum

JANUARY 10, 2022

With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies. Both biotechs focus on mRNA therapies but not just for infectious diseases.

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Drug Discovery World

FEBRUARY 22, 2024

For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. years it takes to complete an entire cancer clinical trial. The organisation also notes that only 1% of the people that can take part in dementia clinical trials actually do so. This compares to the 2.3

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The Pharma Data

NOVEMBER 25, 2020

The mRNA vaccine candidate developed by Pfizer and Germany-based BioNTech demonstrated 95% efficacy in clinical trials. Food and Drug Administration (FDA) will review the data on Dec. The vaccine approvals are the proverbial light at the end of the tunnel that is COVID-19.

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