Recently, we learnt that the Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for IntraBio’s supplemental New Drug Application (sNDA) for AQNEURSA™ (levacetylleucine) for the treatment of GM2 Gangliosidosis.
We calling on the GM2 community to come together and request an Advisory Committee for the Supplementary New Drug Application (sNDA) for AQNEURSA for the treatment of GM2 Gangliosidosis.
To support this initiative, we are asking anyone connect to our community to complete 4 quick questions that supports this request and our community statement below. The questions you will be asked are:
- Your name first name and initial of your last name
- Where you live (plus your State if you live in the US)
- Your connection to the Tay-Sachs and Sandhoff community
- Optional - the opportunity to share your experience or personal story with GM2
Any member of our GM2 community is welcome to participate - from patients to friends and loved ones. While GM2 may affect a small patient populations, its impact is profound and extends beyond the patients alone and affects a much bigger community. The voices of all of us affected are extremely important to share.
Thank you for providing answers to all questions to the best of your ability and if you have any questions please contact dan@cats-foundation.org, diana@ntsad.org, flynn@ntsad.org or rick.karl@curetay-sachs.org.