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.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

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.

FDA Approval 52

FDA Approval Genetic Disease Genetics Drugs 52

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.

FDA Approval 52

FDA Approval Genetic Disease Genetics Clinical Trials 52

Delveinsight

AUGUST 17, 2021

FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDA approved the combination of Keytruda and Lenvima produced by Merck and Eisai, as a first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The rarity of disease – 1.36

FDA Approval 98

FDA Approval Medicine Vaccination Vaccine 98

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).

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

FDA Approval 104

FDA Approval Clinical Trials Clinical Trials Trials 104

The Pharma Data

DECEMBER 3, 2020

HAE is a potentially life-threatening, rare genetic disease characterized by episodes of acute swelling of the skin, pharynx, larynx, gastrointestinal tract, genitals and other extremities. With this decision by the MHRA, the wait for many HAE patients in the UK can end sooner,” said Jon Stonehouse, chief executive officer of BioCryst.

FDA Approval 52

FDA Approval Medicine Genetic Disease Trials 52

XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). ” In March 2022, Nobelpharma received approval from the US Food and Drug Administration (FDA) for its topical treatment for facial angiofibroma called Hyftor (sirolimus topical gel) 0.2

Life Science 98

Life Science Genetic Disease FDA Approval Marketing 98

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. It affects around one in 10,000 girls born each year.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

XTalks

FEBRUARY 27, 2023

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients.

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

World of DTC Marketing

JANUARY 24, 2022

” “More than 50% of FDA-approved medicines now have their start in venture-backed private biotech companies in the United States. These are the groundbreaking treatments for cancer, genetic diseases, and other scourges,” said Arjun Goyal, M.D., co-founder, and managing director at Vida Ventures.

Life Science 225

Life Science Drugs Genetic Disease Development 225

XTalks

SEPTEMBER 22, 2022

On September 1, 2022, Boehringer Ingelheim Pharmaceuticals announced in a press release that the US Food and Drug Administration (FDA) approved Spevigo (spesolimab-sbzo) intravenous injections for GPP flares in adults. Spevigo is the first approved treatment option for GPP in adults. A Promising Treatment for a Rare Disease.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Dermatology 52

pharmaphorum

NOVEMBER 27, 2020

The new EU approval comes after the FDA approved the expanded label last year, and provides the first treatment option that tackles the underlying cause of CF in patients aged six to 11 with these mutations. It is caused by a defective or missing CFTR gene, and there are around 75,000 people worldwide with the disease.

Gene 134

Gene Genetic Disease FDA Approval Genetics 134

XTalks

MAY 17, 2023

In 2022, the FDA approved four new gene therapies, and more than a handful are looking to get approval this year for diseases including Duchenne muscular dystrophy (DMD), sickle cell disease (SCD) and hemophilia A. In the US, more than 30 million people live with a rare disease.

Gene Therapy 98

Gene Therapy Clinical Trials Clinical Trials Gene 98

XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes. The orphan drug designation was.

Clinical Trials 98

Clinical Trials Clinical Trials Genetics Trials 98

pharmaphorum

SEPTEMBER 28, 2022

. “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.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

pharmaphorum

DECEMBER 9, 2021

An FDA advisory committee has delivered a blow to Reata Pharma, after voting unanimously that the drugmaker’s data on bardoxolone – a drug for kidney disease – did not show it is effective. In severe firms, the disease leads to progressive kidney damage with patients often needing dialysis.

Drugs 52

Drugs Clinical Trials Clinical Trials Genetic Disease 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 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.

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

The Pharma Data

NOVEMBER 29, 2020

The FDA approved the drug on November 24. PH1 is an ultra-rare genetic disease marked by overproduction of oxalate, which causes deposits of calcium oxalate crystals in the kidneys and urinary tract and can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and system organ dysfunction.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

Drug Discovery World

NOVEMBER 14, 2023

“In recent decades, significant progress has been made to better understand the link between ADAMTS13 deficiency and cTTP, ultimately leading to this moment where we finally have an FDA-approved treatment option for patients living with this rare disease,” said Spero Cataland, Professor of Internal Medicine at the Wexner Medical Center at The Ohio (..)

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

DECEMBER 20, 2020

The FDA approval of Klisyri is a significant milestone for Athenex,” said Johnson Lau, chairman and chief executive officer of Athenex. CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. Most Read Today. Source link.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Drugs 52

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).

Gene Therapy 111

Gene Therapy Gene Drugs FDA Approval 111

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

The Pharma Data

DECEMBER 8, 2020

The impact of CNS diseases extends beyond patients—to their families and society as well.” The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. .

RNA 52

RNA Antibody Life Science Protein 52

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

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

XTalks

SEPTEMBER 17, 2020

Janssen’s Nizoral (ketoconazole), Bristol Myers Squibb’s Lysodren (mitotane), Novartis’ Metopirone (metyrapone) and Corcept Therapeutics’ Korlym (mifepristone) are all cortisol-inhibiting medications that may be prescribed to treat Cushing’s disease. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.

Trials 98

Trials Gene Hormones Clinical Trials 98

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.

RNA 109

RNA Vaccination Vaccine Pharma Companies 109

XTalks

DECEMBER 22, 2021

RNA therapeutics hold promise as powerful treatments for diseases, including those that are genetic-based. Non-invasive or minimally invasive medical devices that can diagnose, monitor and treat disease is entering a promising era, and will be a key life science trend next year. RNA Therapeutics. The deal is worth $1.5

Life Science 98

Life Science RNA Vaccination Vaccine 98

Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

NOVEMBER 25, 2020

Also this week, the FDA approved Eiger Pharmaceuticals ‘ Zokinvy , the first drug approved to treat Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies. The two genetic diseases cause premature, rapid aging that dramatically decreases the lifespan of children affected.

Vaccination 52

Vaccination Vaccine Genetic Disease Genetics 52

Pharmaceutical Technology

APRIL 24, 2023

Covid-19 vaccines from Moderna and Pfizer /BioNTech, which use lipid nanoparticles, became the only two FDA-approved vaccines for almost all ages. However, after a period of slow movement in the field, the FDA approved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147