Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
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Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
CFF-Championed Bill Eliminates Barriers to Participation in Clinical Trials for People with CF and other Rare Diseases
Proposed legislation has the potential to revitalize the global marketplace for novel antibiotics, bringing urgently needed medicines for drug-resistant infections to patients.
Today, the Cystic Fibrosis Foundation announced it has awarded up to $3.3 million to Polyphor AG to develop an inhaled version of murepavadin, an antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in people with cystic fibrosis. About 17% of individuals with CF who had Pseudomonas infections last year had multi-drug resistant strains.
Legislation Includes Measures to Increase Patient and Expert Participation in FDA Review of Rare Disease Drugs