By Dr. Jason Mercer, RAC

On March 7, the CBER updated the list of biologic products that have been approved in 2024.  So far, there have been two:

  • Casgevy (exagamglogene autotemcel (exa-cel)) from Vertex Pharmaceuticals was approved in January for the treatment of patients 12 years and older with sickle cell disease with recurrent vaso occlusive crises with or without transfusion-dependent β-thalassemia.
  • Amtagvi (lifileucel) from Iovance Biotherapeutics was approved for treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor.

Both products represent exciting advances in medicine.  Casgevy is the first gene therapy approved to treat patients with sickle cell disease and joins the short list of fewer than 20 approved gene therapies.  Amtagvi is the first FDA-approved tumor-infiltrating lymphocyte (TIL) based therapy.  Amtagvi uses lymphocytes harvested from a patient’s tumor and expanded ex-vivo before re-introduction into the patient to target the tumor.  This is a novel approach for immune-oncologic cell therapies as the other currently approved products utilize ex-vivo introduction of a transgenic T cell receptor into T cells harvested from the patient to target the tumor.  We look forward to more advanced therapy approvals for treatment of rare diseases during the coming year!

RARE DISEASE DAY – March 1, 2024
FDA’s virtual public meeting was another success!   If you missed it, you can still view the recorded event on YouTube here: