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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FDA Approval Clinical Trials Clinical Trials Trials 104

The Pharma Data

DECEMBER 3, 2020

The addition of berotralstat through the EAMS will bring a much needed option for HAE patients suffering with this debilitating disease,” said Dr. Sorena Kiani, Consultant Immunologist at Royal London Hospital, London. At the moment, additional doses or dosages of ORLADEYO that surpass 150mg once daily are not recommended.

FDA Approval 52

FDA Approval Medicine Genetic Disease Trials 52

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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Clinical Trials Clinical Trials Genetics Trials 98

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. 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.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

XTalks

MAY 17, 2023

In the past several years, gene therapies have finally been hitting the market after decades of setbacks and uncertainties. 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.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

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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FDA Approval Genetics Trials Drugs 97

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. ” Trading in Reata’s shares was halted ahead of the meeting, but the stock was down 38% pre-market this morning. .”

Drugs 52

Drugs Clinical Trials Clinical Trials Genetic Disease 52

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

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.

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Drug Delivery Vaccination Vaccine Research 147

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

XTalks

APRIL 1, 2025

Clinical Trials Backing Qfitlias Approval Two Phase III studies involving adult and pediatric male patients with hemophilia A or B supported Qfitlias FDA approval. Qfitlia is the second RNA interference therapy to receive a highly anticipated FDA approval recently. Sanofi is not new to the hemophilia market.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

The Pharma Data

DECEMBER 8, 2020

The fully integrated pharmaceutical company creates value through China’s specialty pharmaceutical markets with focus on iron deficiency, pain management and respiratory. Cornering the untapped Chinese CNS market, SciNeuro launched with $100 million in their pocket. SciNeuro Pharmaceuticals .

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RNA Antibody Life Science Protein 52

XTalks

SEPTEMBER 25, 2024

While Miplyffa will be the only treatment for NPC with neurological manifestations on the market, Sanofi has an infusion called Xenpozyme for non-central nervous system manifestations of Niemann-Pick, also known as acid sphingomyelinase deficiency (ASMD), which got an FDA green light in 2022.

Genetics 52

Genetics Drugs Trials Pharmacy 52

Pharmaceutical Technology

SEPTEMBER 30, 2022

These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

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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Trials Gene Hormones Clinical Trials 98

XTalks

NOVEMBER 26, 2024

A Marketing Authorization Application has been submitted to the European Medicines Agency (EMA), with a decision anticipated in 2025. BridgeBio has been highly active in the genetic diseases space. From a regulatory standpoint, BridgeBio has taken steps to ensure global access to Attruby.

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Genetic Disease Genetics Protein Clinical Development 105

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.”

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

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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FDA Approval Production RNA Medicine 52

XTalks

MARCH 19, 2025

Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. This drop likely reflects market concerns over the therapys safety profile. Sarepta promptly notified both prescribing physicians and study investigators. on March 18, 2025, where more than 8.2

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Gene Therapy Gene Genetics Clinical Trials 80

XTalks

DECEMBER 29, 2022

This highlights potentially new marketing trends that life science companies may have to start looking into if they haven’t already. As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. 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

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. How Can Study Protocols Be More Effective?

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

DECEMBER 22, 2021

The market for mRNA-based vaccines and therapeutics is projected to grow from $46.7 billion (including $25 million upfront) with UK-based MiNA Therapeutics to develop small activating RNA (saRNA) drugs for up to five targets across Lilly’s key therapeutic areas of cancer, diabetes, immunology, neurodegenerative diseases and pain.

Life Science 98

Life Science RNA Vaccine Vaccination 98

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. Currently, the company is researching two potential therapies in immuno-oncology, both at the phase 1 stage.

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RNA Vaccination Vaccine Pharma Companies 111

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

JANUARY 6, 2025

Notably, the ASO drug Vitravene (fomivirsen) received US Food and Drug Administration (FDA) approval for treating cytomegalovirus (CMV) retinitis in AIDS patients. Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets.

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