Remove Antibody Remove FDA Approval Remove Gene Therapy Remove Protein
article thumbnail

Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

article thumbnail

Beqvez Becomes Pfizer’s First Approved Gene Therapy After Nod from Health Canada

XTalks

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. Beqvez is also being reviewed by the US Food and Drug Administration (FDA) with a decision anticipated by mid-2024.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Sarepta’s Elevidys Reaches Finish Line as First Gene Therapy Approved for Duchenne Muscular Dystrophy

XTalks

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.

article thumbnail

Roctavian Becomes First Gene Therapy for Severe Hemophilia A to Get FDA Nod

XTalks

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

article thumbnail

Genmab’s Silver Anniversary: Reflecting on 25 Years of Breakthroughs in Antibody Therapeutics

XTalks

Genmab, a trailblazer in the field of antibody therapeutics, celebrates this milestone by reflecting on its impressive journey from a small-scale startup to a global leader in cancer treatment and beyond. Since then, innovative science has been at Genmab’s core, as we harness the power of human antibodies to improve the lives of patients.

article thumbnail

J&J’s Carvykti and BMS’ Abecma Win FDA Approvals for Earlier Use in Multiple Myeloma

XTalks

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

article thumbnail

Amtagvi (Lifileucel) Scores Landmark Win as First FDA-Approved T-Cell Therapy for a Solid Tumor

XTalks

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.