Ten founding companies have joined together to form the Rare Disease Company Coalition — a new lobbying group focused on advocating on behalf of companies that develop and manufacture rare disease treatments.
This first-of-its-kind alignment of organizations will "inform and help educate policymakers on the unique circumstances facing life science companies when developing and bringing to market therapies for very small and differentiated patient populations." The new coalition will also advocate for policies and regulations that enable cost-effective and more timely delivery of treatments by recognizing these differences.
The Rare Disease Company Coalition’s 10 founding members have brought 22 treatments to market and currently have more than 160 rare disease programs in the works.
Founding members of the Coalition include:
Acceleron Pharma
Aeglea BioTherapeutics
Agios Pharmaceuticals
Alnylam Pharmaceuticals
Harmony Biosciences
Orchard Therapeutics
Orphazyme US, Inc.
Sarepta Therapeutics
Taysha Gene Therapies
Ultragenyx Pharmaceutical