Press Releases

The Rare Disease Company Coalition Continues Growth and Announces New Members

Coalition Welcomes Amryt Pharma, Fulcrum Therapeutics and Signature Biologics, LLC

WASHINGTON – October 12, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced the addition of three new companies to the Coalition. Launching in May 2021, the Coalition now boasts a total membership roster of 18 companies and provides a unified voice for innovator companies dedicated to rare disease treatment development. 

New members that join the Rare Disease Company Coalition include:

  • Amryt Pharma
  • Fulcrum Therapeutics
  • Signature Biologics, LLC

As Congress considers policy reforms that could impact the ability for companies to develop treatments for the one in ten Americans living with rare disease and have extraordinarily high unmet needs, the Coalition continues to help inform policymakers on the unique circumstances impacting therapy development for the 93% of rare diseases that have no FDA-approved treatments. 

“We welcome the three new members to our Coalition, and applaud their commitment to developing rare disease treatments through ongoing research and development while often taking on significant risk and uncertainty,” said Betsy Ricketts, Chair of the Rare Disease Company Coalition. “The sustained growth of the Coalition is reflective of the need for ongoing education and engagement with policymakers on the importance of well-informed policy that can support continued progress for developing treatments for people with rare diseases.” 

Life science companies that develop treatments for rare disease patients face numerous challenges from clinical trial enrollment through proving meaningful endpoints given the smaller population sizes and the more limited knowledge known about the disease progression. As a result, a comparatively higher percentage of operating expenses is dedicated to research and development at these life science companies. In 2020, Rare Disease Company Coalition members invested more than $4.1 billion in research and development, representing on average more than half of their annual operating budgets. 

This necessary commitment to research and development underscores the importance of supportive policy and regulatory frameworks that can foster a greater understanding of rare diseases and recognizes the distinct considerations associated with the development of and access to rare disease treatments. 

To learn more about the Coalition and the need for informed policy and legislative discourse and actions related to rare disease treatment development and commercialization, please visit


The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit

Media Contact:

Anna Stallmann