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Expert Interview

Slingshot members are talking to an expert! The topic is:

Advancing Cystic Fibrosis Therapy: A Comprehensive Evaluation of Vertex Pharmaceuticals' Kaftrio (VX-121 + TEZACAFTOR + VX-561) in the Vanza Triple Program

Ticker(s): VRTX

Who's the expert?

Institution: Studienzentrum

  • Specialist for internal medicine in private practice, with a subspecialty in lung and bronchial medicine, specialist for internal medicine in private practice, with a subspecialty in lung and bronchial medicine
  • Previously worked as an internist and lung specialist, most recently as a senior physician at the Innenstadt Munich university hospital. 
  • Founding member and president of the German Society for Alpine and Expedition Medicine, and a member of the cystic fibrosis physicians working group.

Interview Questions
Q1.

In light of the SKYLINE trials, how do you evaluate the comparative efficacy of Kaftrio against TRIKAFTA in improving lung function and reducing sweat chloride levels in patients aged 12 and older?

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Q2.

Considering the significant reduction in sweat chloride levels achieved by Kaftrio, what is the clinical importance of this outcome for patients with CF?

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Q3.

The RIDGELINE 105 study showed remarkable results in children aged 6 to 11. How does early intervention with Kaftrio potentially alter the disease course in pediatric patients?

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Q4.

Can you discuss the safety profile of Kaftrio observed in the Vanza Triple Program, particularly noting any differences between adults and children?

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Q5.

Based on the one-year data from SKYLINE 102 and 103, what are the potential long-term benefits and challenges of using Kaftrio for CF treatment?

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Q6.

How does Kaftrio perform in patients with different CFTR mutations, and what does this mean for the broader CF patient population?

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Q7.

How do you see Kaftrio fitting into the current multidisciplinary approach to managing CF, including considerations for nutrition, physiotherapy, and other treatments?

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Q8.

Given the promising results of Kaftrio, what are the anticipated next steps for its clinical development, and are there any plans to explore its use in younger age groups or other CFTR mutations?

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