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

SRShots: 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 ZealandVanzacaftor &…

ByRidhi RastogiMay 1, 2025

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…

BySenior EditorJanuary 8, 2025

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 …

ByDipanshu DixitFebruary 23, 2024

INSIGHTS+

Top Performing Drug – Trikafta/Kaftrio (May Edition)

Shots:In continuation of our previous series on the top-performing drug of the month, based on 2021 revenue, this month we have covered Trikafta/Kaftrio as the top-performing drug of the monthTrikafta is a combination of ivacaftor, tezacaftor, and elexacaftor indicated for the treatment of cystic fibrosis in pediatric patients who have at least one…

BySenior EditorMay 24, 2023

THOUGHTSPOT

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

Shots:With the healthcare sector moving ahead with its technological advances, there is still an unexplored section in therapeutics that requires much more attention. Through innovations, life science companies have evolved to make breakthrough discoveries in many focus areas, but when it comes to the rare disease segment, all our endeavors seem to be bleak…

BySaurabh ChaubeyMarch 14, 2023

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…

BySenior EditorJanuary 19, 2023

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

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

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

Top Performing Drug – Trikafta/Kaftrio (May Edition)

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

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

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

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

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

Top Performing Drug – Trikafta/Kaftrio (May Edition)

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

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

FOLLW US

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