{"disease":{"id":"cystic-fibrosis","name":"Cystic Fibrosis","therapeutic_area":"Respiratory","data":{"aiSummary":"The treatment landscape for cystic fibrosis has dramatically improved with the advent of CFTR modulators, which target the underlying cause of the disease. Trikafta (elexacaftor/tezacaftor/ivacaftor) is a highly effective modulator for many patients, regardless of mutation. Other modulators like Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor) are also available. The pipeline includes new modulator combinations and other therapies, such as inhaled antibiotics and gene therapy, to further improve outcomes for individuals with CF.","drug_count":9,"description":"Cystic fibrosis (CF) is a genetic disorder characterized by the buildup of thick mucus in the lungs, digestive tract, and other organs. This leads to chronic lung infections, digestive problems, and other complications. The disease is caused by mutations in the CFTR gene, which encodes a protein that regulates chloride transport across cell membranes. Current treatments focus on managing symptoms and slowing disease progression, including airway clearance, antibiotics, and CFTR modulators.","subtype_count":13},"enrichment_level":2,"last_enriched_at":null,"visit_count":0,"created_at":"2026-03-25T12:16:34.923Z","updated_at":"2026-04-19T18:39:44.960Z","meddra_pt":null,"meddra_code":null,"icd10_codes":null,"synonyms":[],"prevalence_global":null,"prevalence_us":null,"marketed_drug_count":5,"pipeline_drug_count":0,"trial_count":0},"drugs":{"marketed":[{"drug_id":"elexacaftor","indication_name":"Cystic fibrosis","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Trikafta (Copackaged)","generic_name":"ELEXACAFTOR","company_name":"Vertex Pharms Inc","drug_phase":"marketed","molecular_target":"Cystic fibrosis transmembrane conductance regulator","drug_class":"","quality_score":65,"revenue":"11100","mechanism":"Trikafta works by correcting the faulty protein that causes cystic fibrosis, allowing the body to produce normal amounts of mucus."},{"drug_id":"fusidic-acid","indication_name":"Synergy for P. 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