XTalks

OCTOBER 12, 2022

Perfusion is the passage of fluid through the lymphatic system or the circulatory system to an organ or tissue, and it typically refers to the delivery of blood to a capillary bed in tissue. Anti-angiogenic drugs can slow the growth of tumors and sometimes even shrink tumors by preventing them from growing their own blood vessels.

Clinical Trials 93

Clinical Trials Clinical Trials Trials Drugs 93

pharmaphorum

AUGUST 21, 2022

The FDA has cleared Auvelity (dextromethorphan/bupropion) for the treatment of major depressive disorder (MDD) in adults, setting up a launch in the fourth quarter of this year. In this product, bupropion is designed to increase levels of dextromethorphan in the blood and extend its half-life.

Drugs 91

Drugs FDA Approval Production Marketing 91

pharmaphorum

DECEMBER 12, 2022

Updated results from the ADVANCE IV study of Vyvgart (efgartigimod alfa) showed that more than half of patients with ITP treated with Argenx’ drug showed an improvement in platelet counts, which are dramatically reduced in the disorder. After 24 weeks, 21.8%

Sales 52

Sales Trials Drugs Antibody 52

pharmaphorum

MAY 17, 2021

C5 inhibitor Soliris – which has a list price in the US of more than $500,000 per year – has been a standard therapy for the rare disorder since 2007, and was joined on the market by longer-acting follow-up Ultomiris in 2018 which launched with a price tag of around $450,000 per year. billion and $1.1

Drugs 52

Drugs FDA Approval Marketing Clinical Trials 52

pharmaphorum

FEBRUARY 22, 2021

Last week, Bluebird said it was pausing its Zynteglo (betibeglogene autotemcel) programme in Europe after a phase 1/2 trial in patients with sickle cell disease (SCD) – another red blood cell disorder – turned up two cases of blood cancers, one of acute myeloid leukaemia (AML) and another of myelodysplastic syndrome (MDS).

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

pharmaphorum

JANUARY 11, 2021

While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA. million price tag. Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

Gene Therapy 58

Gene Therapy Gene Genetic Disease Manufacturing 58

XTalks

JUNE 9, 2021

Related: Could A Simple Blood Test Detect Alzheimer’s Disease? Alzheimer’s disease is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills. Aducanumab Controversy and Price Tag. And with a broad-label approval that makes the treatment available to all 6.2

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Drugs Clinical Trials Clinical Trials Trials 98

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. The estimated incidence of the disorder worldwide is one in 100,000.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

Pharmaceutical Technology

JANUARY 27, 2023

On January 18, Sanofi launched a new warranty program for its rare blood disorder drug Cablivi (caplacizumab), which tackles value-based agreements in a slightly different way. The disorder has a reported annual incidence between 1.5 cases per million in Europe.

Manufacturing 130

Manufacturing Drugs Research Analyst FDA Approval 130

pharmaphorum

NOVEMBER 16, 2022

Only a few years later, bluebird bio’s gene therapy, Zynteglo (betibeglogene autotemcel), was approved in the European market for the treatment of the rare blood disorder, beta thalassaemia. However, with a price tag of €1.58 At more than $2.1

Gene Therapy 126

Gene Therapy Gene Marketing Development 126

XTalks

NOVEMBER 30, 2022

In this episode, Ayesha shared news about the FDA approval of the first gene therapy for the rare blood disorder Hemophilia B. Hear more about the therapy and how the high price tag of the new drug is actually lower than the cost of treating Hemophilia B patients over their lifetimes with current conventional treatments.

Gene Therapy 52

Gene Therapy Life Science Gene FDA Approval 52

XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). With a list price of $3.5 Hemophilia B primarily affects men.

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetics 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. With the approval, Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy also became the first CRISPR/Cas9-based therapy approved in the US.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

XTalks

SEPTEMBER 27, 2022

Skysona received FDA approval for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD). The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. Related: Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia.

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

Delveinsight

DECEMBER 3, 2020

Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Ovid showed a phase 2 data on OV101 in Angelman, a genetic nervous system disorder, in 2018.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

XTalks

JANUARY 19, 2024

Late sequela could include pneumonitis, lung fibrosis, fertility disorders and secondary malignancies. This could include the use of blood PK to determine whether the correct exposure has been obtained and whether higher exposures will lead to more side effects or efficacy.

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DNA Radiology FDA Approval Development 66