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Research | CFTR Modulators FDA Approves Expansion of Modulators for People With Certain Rare Mutations

The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.

| 3 min read
CFTR Modulators | About the CF Foundation A Message From Our CEO

The CF Foundation has successfully completed a sale of its remaining stake in royalties related to Vertex's CFTR modulators for an upfront payment of $575 million and a potential future payment of $75 million, bringing additional resources to the fight against CF.

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Research | CFTR Modulators Roche Acquires Potential CF Therapies; Plans to Develop Alternatives to CFTR Modulators

Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.

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CFTR Modulators | About the CF Foundation | Clinician Resources CF Foundation Leaders Provide Expertise During ICER Public Meeting

On Aug. 27, leaders from the Cystic Fibrosis Foundation, along with several people from the CF community, participated in a virtual public meeting hosted by the Institute for Clinical and Economic Review to provide insights and expertise on the value of CFTR modulators.

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CFTR Modulators ICER Issues Draft Evidence Report for Assessment of Trikafta

The Institute for Clinical and Economic Review (ICER) recently posted a draft evidence report to outline their initial assessment of the clinical effectiveness and value of CFTR modulators, including elexacaftor/tezacaftor/ivacaftor (TrikaftaTM). The document is open for public comment through March 18.

 

| 2 min read