Advancing Targeted Therapies for Rare Diseases Act (S.2030)

A EURORDIS and NORD Member Organization 

As a NORD member organization, MEBO has signed on to a NORD coalition letter in support of the Advancing Targeted Therapies for Rare Diseases Act (S.2030).

As background, this is bill is a result of the efforts of Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association (MDA), Duchenne Alliance, and more within the rare disease patient advocacy community.


This legislation clarifies FDA’s existing authority to leverage data previously used in the approval of a targeted drug when approving a new therapy that “incorporates or utilizes the same or similar genetically targeted technology, or the same variant protein targeted technology”.

This is particularly important to drug development for patients with diseases that contain various genetic sub-populations. This is also important for diseases that may be genetically related to other diseases, and could potentially benefit from drug development in those other diseases.

The bill language for S.2030 can be found here: https://www.congress.gov/bill/114th-congress/senate-bill/2030/text

María

María de la Torre
Founder and Executive Director

A Public Charity
maria.delatorre@meboresearch.org
www.meboresearch.org
www.mebo.com.br (MEBO Brasil)
diretoria@mebo.com.br
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A EURORDIS and NORD Member Organization