Cystic Fibrosis Weekly Update

Contents:

European Commission Approves Kalydeco for Children as Young as Six Months

Dec 11, 2019 07:00 am | Marisa Wexler MS

The European Commission has approved a label extension for Kalydeco (ivacaftor), allowing the therapy to be used in infants with cystic fibrosis (CF) who are between the ages of six months and one year. The decision follows a positive opinion on Kalydeco, marketed by Vertex Pharmaceuticals, that was issued by the Committee for Medicinal Products for […]
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Confessions from the 10 Percent: Life Without Trikafta

Dec 10, 2019 03:00 pm | Bailey Vincent

I am one of the 10 percent. The 10 percent cannot take the new, groundbreaking medication that will hopefully help cystic fibrosis (CF) patients for years to come. Some people cannot benefit from Trikafta (elexacaftor/tezacaftor/ivacaftor) because they recently had a transplant. For others, like me and anomalous genotypes, Trikafta isn’t an option because we are […]
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Gene Therapy Takes Center Stage at 2019 NORD Summit

Dec 10, 2019 10:00 am | Larry Luxner

Former FDA Commissioner Scott Gottlieb, ALS

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high costs and limitless potential for curing […]
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Kalydeco Improves Lung Function in Patient Carrying Responsive and Non-responsive Mutations, Case Study Reports

Dec 10, 2019 07:00 am | Patricia Inacio, PhD

Treatment with Kalydeco (ivacaftor) alone improved lung function and quality of life in a cystic fibrosis patient carrying both a therapy-responsive and a non-responsive mutation to treatment, a case study reports. The case study, titled “Positive clinical outcomes following ivacaftor treatment in a cystic fibrosis patient with the genotype 3272–26A > G/Q493X,” was published in the […]
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