Anavex Life Sciences Corp (AVXL)

[nidan7500]

If AVXL has been granted RMAT for Rett Syndrome trials then tis looks like a slam dunk, based on previous trial results. All he has to say is he has RMAT and game over.

some text deleted...directly from FDA page on new trials rules

RMAT...NOTE ...I believe ANAVEX has IND for RETT syndrome, see PR

.
HHS
U.S. Department of Health and Human Services
U.S. Food and Drug Administration
Search FDA

As described in Section 3033 of the 21st 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
We refer you to the Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics, section III.A., for FDA’s interpretation of whether a disease or condition is serious or life-threatening and whether a drug is intended to treat a serious disease or condition.
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.(NOTE A2-&# has been granted IND for RETT) We will not grant a RMAT designation if an IND is on hold or is placed on hold during the designation review.
We generally do not expect you to submit primary data (data sets), but your request for regenerative advanced therapy designation should describe the preliminary clinical evidence. Please include a brief description of any available therapies for the disease or condition, the study design, the population studied, and the endpoint(s) used; and a description of the study results and statistical analyses (e.g., subgroup analyses).
You may submit a request for RMAT designation to:
Food and Drug Administration
Center for Biologics Evaluation and Research
Office of Tissues and Advanced Therapies
Document Control Center
10903 New Hampshire Avenue
WO71, G112
Silver Spring, MD 20993-0002
If the RMAT designation request is submitted to your IND as an amendment, the cover letter should specify that the submission contains a REQUEST FOR REGENERATIVE MEDICINE ADVANCED THERAPY DESIGNATION in bold, uppercase letters. If the request is submitted with an initial IND, the cover letter should specify that the submission contains both an INITIAL INVESTIGATIONAL NEW DRUG SUBMISSION and REQUEST FOR REGENERATIVE MEDICINE ADVANCED THERAPY DESIGNATION in bold, upper case letters.
No later than 60 calendar days after receipt of the designation request, the Office of Tissues and Advanced Therapies (OTAT) will notify the sponsor as to whether RMAT designation has been granted. If OTAT determines that the RMAT designation request was incomplete or that the drug development program does not meet the criteria for RMAT designation, OTAT will include a written description of the rationale for such determination.

Cellular & Gene Therapy Products
Approved Products
Cloning
Page Last Updated: 03/13/2017
Note: If you need help accessing information in different file formats, see Instructions for Downloading Viewers and Players.
Language Assistance Available: FDA
Accessibility
10903 New Hampshire Avenue
Silver Spring, MD 20993
1-888-INFO-FDA (1-888-463-6332)
Contact FDA
Subscribe to FDA RSS feeds

Follow FDA on Twitter

Follow FDA on Facebook

View FDA videos on YouTube

View FDA photos on Flickr

FDA Archive
Combination Products
Advisory Committees
Regulatory Information
Safety
Emergency Preparedness
International Programs
News & Events
Training & Continuing Education
Inspections & Compliance
Federal, State & Local Officials
Consumers
Health Professionals
Science & Research
Industry