X
Pharma Advancement
  • Home
  • Drug Development
    • All
    • Clinical Trials
    • FDA Approvals
    • Research & Development

    New Delivery Path Brings To Light Efficacy of Immunotherapy

    Nasal Spray Shows Its Effect Against New COVID Variants

    Mid-Point Regulatory Science Strategy Study Published By EMA

    AADC Inadequacy To Be Handled By A Novel Gene Therapy

    New Drug Explored For Epilepsy Gets $3M Funding In Australia

    Care Access Research Launches Alliance to Provide Infrastructure for COVID-19 Trials

    Faster Clinical Trial Nods Planned By The UK To Curb Issues

    Pharma Drugs Gain From Tardigrade Proteins, Says Research

    Pharma Drugs Gain From Tardigrade Proteins, Says Research

    U.S. FDA Approves DARZALEX Split-Dosing Regimen

    EMA Keeping A Close Watch On Critical Medicine Shortages

    Pathways Laid Down By The CMS For Drug Price Negotiations

    Pathways Laid Down By The CMS For Drug Price Negotiations

  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us
No Result
View All Result
  • Home
  • Drug Development
    • All
    • Clinical Trials
    • FDA Approvals
    • Research & Development

    New Delivery Path Brings To Light Efficacy of Immunotherapy

    Nasal Spray Shows Its Effect Against New COVID Variants

    Mid-Point Regulatory Science Strategy Study Published By EMA

    AADC Inadequacy To Be Handled By A Novel Gene Therapy

    New Drug Explored For Epilepsy Gets $3M Funding In Australia

    Care Access Research Launches Alliance to Provide Infrastructure for COVID-19 Trials

    Faster Clinical Trial Nods Planned By The UK To Curb Issues

    Pharma Drugs Gain From Tardigrade Proteins, Says Research

    Pharma Drugs Gain From Tardigrade Proteins, Says Research

    U.S. FDA Approves DARZALEX Split-Dosing Regimen

    EMA Keeping A Close Watch On Critical Medicine Shortages

    Pathways Laid Down By The CMS For Drug Price Negotiations

    Pathways Laid Down By The CMS For Drug Price Negotiations

  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us
No Result
View All Result
Pharma Advancement
No Result
View All Result
Home Europe

Vertex obtains receives European approval for cystic fibrosis oral medicine

Yuvraj_pawp by Yuvraj_pawp
4th August 2014
in Europe, News

The European Commission has approved Vertex Pharmaceuticals’ oral medicine Kalydeco (ivacaftor) for people with cystic fibrosis (CF).

Kalydeco is for CF patients who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

In July 2012, the medicine first received European approval for CF patients of ages six and older with the G551D mutation, considered to be the most common gating mutation.

The Committee for Medicinal Products for Human Use (CHMP) expressed a positive opinion on the medicine in June 2014.
The eight additional gating mutations in the latest approval are G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

Cystic fibrosis is a result of either a defective or missing CFTR protein caused by mutations in the CFTR gene. CFTR proteins serve as channels at the cell surface that control the flow of salt and water into and out of the cell.

“We are committed to working closely with the appropriate national authorities to make Kalydeco available for [CF] patients as soon as possible.”

However, in people with CF problem, the CFTR protein at the cell surface becomes defective and causes abnormally thick, sticky mucus in the lungs. The digestive tract and several other organs are also affected.

Kalydeco regulates the CFTR protein to function normally once it reaches the cell surface.

Targeting the abnormal CFTR protein channels, the oral medicine helps open the channels to enable chloride ions to move into and out of the cell, which in turn helps thin the mucus and hydrate and protect the airways.

The latest approval is based on the data collected from the first part of a Phase III, two-part, randomised, double-blind, placebo-controlled, cross-over study of 39 CF patients ages six and older who have a non-G551D gating mutation.

In the first part of the study, patients treated with Kalydeco showed improvements in lung function (FEV1), sweat chloride, body mass index and CFQ-R scores, and in the second part these improvements were maintained throughout all 24 weeks of treatment.

Vertex senior vice president and international general manager Simon Bedson said: “We are committed to working closely with the appropriate national authorities to make Kalydeco available for these patients as soon as possible.”

The drug is also approved in Australia, New Zealand and Switzerland for people with CF who have at least one copy of the G551D mutation in the CFTR gene.

Tags: Europe
Previous Post

Rosetta Genomics Announces Agreement With Global Pharmaceutical Company to Advance Efforts in Alzheimer's Disease Diagnostics

Next Post

Pharma firms earn high margins; govt asked to cut MRP of drugs

Related Posts

Drug Development

New Delivery Path Brings To Light Efficacy of Immunotherapy

1st April 2023
Quotient Sciences completes integration of drug substance into Translational Pharmaceutics Platform
Facilities & Operation

£277m Funding Set To Benefit UK Life Sciences Manufacturing

31st March 2023
Drug Development

Nasal Spray Shows Its Effect Against New COVID Variants

27th March 2023
FDA Approvals

Mid-Point Regulatory Science Strategy Study Published By EMA

27th March 2023
Drug Development

AADC Inadequacy To Be Handled By A Novel Gene Therapy

24th March 2023
Drug Development

New Drug Explored For Epilepsy Gets $3M Funding In Australia

23rd March 2023
Next Post

Pharma firms earn high margins; govt asked to cut MRP of drugs

Latest News

Drug Development

New Delivery Path Brings To Light Efficacy of Immunotherapy

1st April 2023
Cathay Cargo signs global airline partnership with Tower Cold Chain
Packaging & Logistic

Cathay Cargo signs global airline partnership with Tower Cold Chain

31st March 2023
Quotient Sciences completes integration of drug substance into Translational Pharmaceutics Platform
Facilities & Operation

£277m Funding Set To Benefit UK Life Sciences Manufacturing

31st March 2023
Drug Development

Nasal Spray Shows Its Effect Against New COVID Variants

27th March 2023
FDA Approvals

Mid-Point Regulatory Science Strategy Study Published By EMA

27th March 2023
Pharma Advancement

About Us

Pharma Advancement is a leading Pharma information centric website. On one side Pharmaadvancement.com has established itself as one of the most efficient and comprehensive source of Pharma information online, dedicated to providing decision makers in all the Pharma industry sectors with reliable, accurate and useful insights into happenings in the Pharma sector.

Qucik Links

  • Drug Development
  • Manufacturing
  • News
  • Events & Conferences
  • Newsletter Archive

Resources

  • Advertise with us
  • Contact Us
  • Download Mediapack

System

  • Search
  • Sitemap
  • RSS Feed

© 2017 Copyright © Valuemediaservices 2017 All rights reserved.

No Result
View All Result
  • Home
  • Drug Development
  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us

© 2017 Copyright © Valuemediaservices 2017 All rights reserved.

Login to your account below

Forgotten Password?

Fill the forms bellow to register

All fields are required. Log In

Retrieve your password

Please enter your username or email address to reset your password.

Log In