As hurricane season made itself felt this week, summer seems over in the east. Kids are back to school and this weekend summer whites and linens have their final appearance before next Memorial Day. Meanwhile it was a very busy week for FDA – there was an approval of the first treatment of Chagas disease as well as a new antibacterial drug. All in all it was actually a rather momentous week and we capture a portion of that here.
- Approval of First Gene Therapy – “We are entering a new frontier in medical innovation…” began a quote from Commissioner Scott Gottlieb in the FDA press release this week announcing the approval of Kymriah (tisagenlecleucel) for the treatment of certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Indeed the approval does usher in a new era where a treatment is manufactured using patient’s own modified T-Cells to kill cancer cells and where in the clinical trial that was the basis for approval, the overall remission rate within three months of treatment was 83 percent. The treatment carries the potential for severe side effects and carries a boxed warning for potential cytokine release syndrome (CRS) which can carry high fever and flu-like symptoms. FDA also announced the expanded approval of tocilzumab to treat CRS. You can see the company digital and multimedia press release here.
- FDA Takes Action on Stem Cell Therapies – From one avenue of promising therapeutic change to another, but with a different direction – FDA announced this week enforcement actions being taken vis a vis the use of unapproved stem cell therapies in the U.S. The agency announced it had issued a warning letter to one manufacturer and a seizure of product associated with another. In a statement issued by FDA Commissioner Gottlieb, the agency also outlined new policy steps and enforcement vis a vis stem cell and regenerative therapies. In it, Commissioner Gottlieb makes clear that while stem cell and regenerative research holds a great deal of promise, the use of unapproved product stands to potentially harm patients and undermine the entire field of science, stating that the agency must separate “the promise from the unscrupulous hype” and that FDA would be stepping up enforcement while simultaneously working to ensure that responsible research is supported.
- Real World Evidence and Devices – Patient centeredness has many facets, and one of them that has been of growing interest has been the use of real world evidence in the approval of treatments for patients. Most of the focus has been on drugs, but this week FDA made available guidance – “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices“. The guidance describes when such evidence can be used, and the criteria that must be fulfilled in order for there to be confidence int he data as well as providing some examples of actual use that have already led to FDA decisions.
That’s it for me this Labor Day Weekend. Have a good one everyone.