It truly is laughable how they turn themselves in knots trying to walk stuff back .....
.... when Remestemcel-L received another CRL, according to the docs on here the FDA knew exactly what they were doing and made the right decision(s) but now that the FDA are entertaining an AA for CHF / LVAD through the RMAT provisions all sorts of criticisms of the FDA are forthcoming including now that they won't approve it .... you seriously can't make this stuff up
IMO ... People should do their own research instead of relying on information posted by self proclaimed doctors and the like on an anonymous stock forum
https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-program
Accelerated Approval Program
The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval.
https://www.fda.gov/vaccines-blood-...erative-medicine-advanced-therapy-designation
Regenerative Medicine Advanced Therapy Designation
As described in Section 3033 of the 21
st Century Cures Act, a drug is eligible for regenerative medicine advanced therapy (RMAT) designation if:
- The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
- The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
- Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition
Based on FDA’s interpretation of Section 506(g) of the Federal Food, Drug, and Cosmetic Act (as added by Section 3033 of the 21
st Century Cures Act), certain human gene therapies and xenogeneic cell products may also meet the definition of a regenerative medicine therapy. For more information on FDA’s definition of regenerative medicine therapies, refer to the
Guidance for Industry, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.
The request for RMAT designation must be made either concurrently with submission of an Investigational New Drug application (IND) or as an amendment to an existing IND. We will not grant a RMAT designation if an IND is on hold or is placed on hold during the designation review.
(20min delay)