Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues.

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BioSpace

OCTOBER 22, 2023

The label for the achondroplasia drug, which promotes endochondral bone growth, now covers children under five years of age with the rare genetic disease causing the most common form of dwarfism.

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XTalks

MARCH 19, 2025

Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. DMD is a rare, progressive and fatal disease. Sarepta is reviewing all available data. It will update Elevidys prescribing information and adjust monitoring protocols as necessary.

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XTalks

OCTOBER 16, 2024

Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year.

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XTalks

SEPTEMBER 12, 2022

After approvals in Japan and Europe, Sanofi’s Xenpozyme has secured one from the US Food and Drug Administration (FDA) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Patients in the trial received Xenpozyme or placebo for 52 weeks.

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XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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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.

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XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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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.

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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

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

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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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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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).

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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

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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

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.

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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.

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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.

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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.

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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

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.

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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.

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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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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.

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XTalks

SEPTEMBER 25, 2024

In a significant regulatory milestone, the US Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for treating neurological symptoms associated with Niemann-Pick disease type C (NPC). NPC, caused by mutations in the NPC1 or NPC2 gene, typically shortens life expectancy to around 13 years.

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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.

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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.

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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

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XTalks

SEPTEMBER 25, 2024

It is estimated that 900 people are living with NPC in the US, of which approximately one-third have been formally diagnosed with this ultra-rare, relentlessly progressive and fatal neurodegenerative disease. NPC is a rare genetic disorder that leads to progressive neurological symptoms and organ dysfunction.

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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.

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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.

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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. .

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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

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XTalks

JANUARY 3, 2025

For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option. FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels.

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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.

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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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