Since Mesoblast made this announcement below in July, the FDA have come back and asked for 'additional information' and we are awaiting a meeting this quarter under the RMAT framework .....
"Mesoblast now intends to meet with FDA under the RMAT framework to discuss the totality of the data and the evidence of a common rexlemestrocel-L MOA across the broader HFrEF spectrum."View attachment 4885573..... here are some links outlining the complexities involved in this process, which some might find interesting to read while we await the FDA / OTAT feedback on the matter ?
This is what Mesoblast went through to get it in the first place:https://www.fda.gov/vaccines-blood-...erative-medicine-advanced-therapy-designationAs 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
These are some of the examples of the RMAT process Mesoblast is now following to enable guidance on how they proceed:https://www.fda.gov/vaccines-blood-...d-breakthrough-therapy-bt-designated-productsSubmission of Initial Comprehensive Meeting (Type B) requests to OTATThe sponsor should submit the meeting request as an amendment to the existing IND. The meeting request should include a list of the specific objectives of the meeting and a list of questions [grouped by discipline; e.g., Chemistry, Manufacturing, and Controls (CMC), pharmacology / toxicology, clinical, statistical].
Courtesy emails to the Regulatory Project Manager (RPM) for the IND and to
[email protected] regarding submission of the meeting request to the file is greatly appreciated.
OTAT does not send an acknowledgement email or letter following OTAT’s receipt of a meeting request. However, by the timelines described in the
Table, OTAT will send the decision to grant or deny the meeting request, along with the meeting date, if granted, or reasons for denial.
View attachment 4885633RMAT/BT – End of Phase and Pre-BLA (Type B) MeetingsOTAT will grant these milestone meetings if, upon evaluation, the stage of development matches the requested milestone. Please refer to the specific milestone meetings, e.g.,
EOP,
pre-BLA, described elsewhere on the website.
Only one meeting will be granted per milestone (e.g., one EOP2). Milestone meetings should be multidisciplinary. OTAT will not grant separate “clinical” and “CMC” EOP mtgs. Refer to the milestone meeting section above.
It's complex so not a 5 minute / quick process - just sayin'
GLTAH
(20min delay)