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Vertex Pharmaceuticals
NEWS

Vertex Pharmaceuticals’ Alyftrek Receives the EC’s Approval to Treat Cystic Fibrosis

Shots:    The EC has approved Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) to treat pts (≥6yrs.) with cystic fibrosis (CF), having ≥1 non-class I mutation in the CFTR gene; regulatory review is ongoing in Canada, Switzerland, Australia, & New Zealand In 2 H2H trials, Alyftrek + ivacaftor matched Kaftrio (ivacaftor/tezacaftor/elexacaftor) on ppFEV1 & showed superiority in reducing sweat chloride…
July 2, 2025

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Vertex Pharmaceuticals
NEWS

Vertex Pharmaceuticals’ Alyftrek Receives the CHMP’s Positive Opinion for the Treatment of Cystic Fibrosis

S R Shots:    The CHMP has recommended Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) to treat patients (≥6yrs.) with cystic fibrosis (CF), having at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Alyftrek is approved in the US and UK and is under review in Canada, Switzerland, Australia, and New Zealand Vanzacaftor &…
May 1, 2025

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NEWS

Orna Collaborates with Vertex to Develop Gene Therapies for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)

Shots: Orna & Vertex have entered into a 3yrs. strategic research collaboration to utilize the former’s LNP delivery solutions for supporting the development of next-gen. gene editing therapies for SCD & TDT, with Vertex funding the partnership As per the terms, Orna will get $65M upfront (incl. investment in the form of a convertible…
January 8, 2025

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Vertex Pharmaceuticals
NEWS

Vertex Receives CHMP’s Positive Opinion for Kalydeco as a Treatment of Cystic Fibrosis in Infants  

Shots:   The company received the CHMP’s positive opinion for the label expansion of Kalydeco (Ivacaftor) to include the treatment of infants (aged 1mos.-4mos.) with cystic fibrosis with specific mutations in the CFTR gene (R117H/G551D/G1244E/G1349D/G178R/G551S/S1251N/S1255P/S549N/S549R)  Kalydeco has been approved by the EU for the treatment of cystic fibrosis patients aged ≥4mos. with specific CFTR gene mutations …
February 23, 2024

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Insights+: Key Takeaways of J.P. Morgan Healthcare Conference 2023 Based on Therapeutic Areas
INSIGHTS+

Insights+: Key Takeaways of J.P. Morgan Healthcare Conference 2023 Based on Therapeutic Areas

Shots: The 41st Annual J.P. Morgan Healthcare Conference took place on 09 – 12 January 2023 in San Francisco, CA, USA. It is the largest and most informative healthcare investment symposium in the industry, bringing together a cohort of industry leaders, emerging fast-growing companies, innovative technology creators, and members of the investment community  The major announcement…
January 19, 2023

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