№ files_lp_3_process_7_064923
File format: docx Character count: 41305 File size: 130 KB
Year: 2025
Region / City: Australia
Topic: Cystic fibrosis treatment
Document Type: Submission for listing in Section 100, Highly Specialised Drugs Program
Organization: Vertex Pharmaceuticals Pty Ltd
Author: Vertex Pharmaceuticals Pty Ltd
Target Audience: Healthcare professionals, Medical practitioners
Effective Date: February 6, 2025
Date of Last Consideration: November 2023
Treatment Phase: Initial treatment, Continuing treatment
Indication: Cystic fibrosis
Population: Patients aged 1 to 4 months with specific mutations in CFTR gene
Clinical Criteria: Must be assessed through a cystic fibrosis clinic/center, G551D mutation or other gating mutations in CFTR gene
Prescription Type: Authority Required
Date of Submission: 5 October 2025
Modification Date: Not specified
Status: Registered
Listing Request Basis: Clinical need, equity
Background: Ivacaftor approved for treatment of CF
Context Description: A submission for listing a new strength of ivacaftor (13.4 mg granules) for cystic fibrosis patients aged 1 to 4 months with specific CFTR gene mutations, requesting inclusion in the Highly Specialised Drugs Program.
Price: 8 / 10 USD
The file will be delivered to the email address provided at checkout within 12 hours.

Don’t have cryptocurrency yet?

You can still complete your purchase in a few minutes:
  1. Buy Crypto in a trusted app (Coinbase, Kraken, Cash App or any similar service).
  2. In the app, tap Send.
  3. Select network, paste our wallet address.
  4. Send the exact amount shown above.
After sending, paste your TXID (transaction ID) and your email to receive the download link. Need help? Contact support and we’ll guide you step by step.

The final amount may vary slightly depending on the payment method.
The file will be sent to the email address provided at checkout within 24 hours.
The product description is provided for reference. Actual content and formatting may differ slightly.
Supplementary material for CFTR activity enhancement by GLPG2222 with and without ivacaftor in FLAMINGO and ALBATROSS trials
Year: 2023
Study type: Randomized clinical trials
Drug investigated: GLPG2222
Combination therapy: With and without ivacaftor
Measured outcomes: Sweat chloride concentration, ppFEV1, FEV1 absolute changes
Study centers: Multiple international hospitals across Belgium, Netherlands, Serbia, Spain, United Kingdom, United States, Australia, Czech Republic, Germany, Ireland
Principal investigators: Scott C Bell, Peter J Barry, Kris De Boeck, Pavel Drevinek, J Stuart Elborn, Barry J Plant, Predag Minić, Eva Van Braeckel, Stijn Verhulst, Karine Muller, Desirée Kanters, Susan Bellaire, Herman de Kock, David E Geller, Katja Conrath, Olivier Van de Steen, Kors van der Ent
Participant groups: FLAMINGO (N=59), ALBATROSS (+ ivacaftor; N=37)
Safety population: Patients with cystic fibrosis and associated comorbidities
Data presentation: Supplementary figures and tables including waterfall plots and mean FEV1 changes
Topics: Sachet Kalydeco IVACAFTOR