June 7: The FDA granted another indication to Alexion Pharmaceuticals, Inc.’s Ultomiris (ravulizumab-cwvz) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in children at least 1 month old and adolescents. It is the only PNH therapy approved for use in these age groups. The agency initially approved the C5 inhibitor on Dec. 21, 2019, for PNH in adults. Dosing is weight-based; administration intervals also are weight-based and are either every four weeks or every eight weeks. The estimated average cost per year in adults is $458,000.

June 9: The FDA gave another indication to Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis who are 6 to 11 years old and have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data. The agency also approved a new dosage strength of the tablet: elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg. The FDA first approved the drug on Oct. 21, 2019. Dosing in this new age group for people less than 30 kg is two elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg tablets in the morning and one ivacaftor 75 mg tablet in the evening. For those weighing at least 30 kg, dosing is two elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg tablets in the morning and one ivacaftor 150 mg tablet in the evening. Website Drugs.com lists the price of 84 tablets of the latter dose as more than $24,900.

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