Breaking News
April 23, 2019 - Research shows mindful body awareness training helps women recover from drug addiction
April 23, 2019 - Researchers are developing brand-new method to cure brain tumors
April 23, 2019 - Torrent Pharmaceuticals Limited Expands Voluntary Nationwide Recall of Losartan Potassium Tablets, USP and Losartan Potassium/Hydrochlorothiazide Tablets, USP
April 23, 2019 - First case of person without the protein needed to transport vitamin D identified
April 23, 2019 - Precision-guided anticancer nanoparticles help enhance treatment of peritoneal tumors
April 23, 2019 - Smoking cessation during pregnancy associated with reduced risk of preterm birth
April 23, 2019 - Researchers discover why women get autoimmune diseases far more often than men
April 23, 2019 - New Medicare reimbursement rules provide some relief to safety-net hospitals
April 23, 2019 - Sensory Sensitivity Tied to Constipation in Young Children
April 23, 2019 - More than half of internal medicine graduates choosing primary care
April 22, 2019 - Researchers discover good news for fish populations living on bleached coral reefs
April 22, 2019 - Plant-based diets associated with lower risk of heart failure
April 22, 2019 - Food Allergies Can Strike at Any Age
April 22, 2019 - Cerebro-facio-thoracic dysplasia – Genetics Home Reference
April 22, 2019 - Poverty leaves a mark on our genes
April 22, 2019 - Countdown to Big Data in Precision Health: When industry and academia converge
April 22, 2019 - The U.S government may account for up to $37.8 billion due to opioid epidemic
April 22, 2019 - Improving ACA’s Insurance Coverage Provisions will lead to better care for patients
April 22, 2019 - Study identifies possible therapeutic effects of curcumin on stomach cancer
April 22, 2019 - Hyaline fibromatosis syndrome – Genetics Home Reference
April 22, 2019 - Scientists use CRISPR for possible ‘bubble boy’ therapy
April 22, 2019 - Hematologist (and a mom, singer, actress and much more) stands up for diversity
April 22, 2019 - Novel AI voice tool can help diagnose PTSD
April 22, 2019 - Overlooked part of cell’s internal machinery may hold key to treating acute myeloid leukemia
April 22, 2019 - Nursing, medical, and dental students train together to improve pediatric oral health
April 22, 2019 - Soft bedding responsible for majority of sleep-related infant deaths, study reveals
April 22, 2019 - Study finds worse health-related quality of life among transgender adults
April 22, 2019 - MIT scientists reverse some behavioral symptoms of rare neurodevelopmental disorder
April 22, 2019 - Scientists find new therapy target for drug-induced liver failure
April 22, 2019 - Opioid dose variability could lead to increased risk of overdose, study suggests
April 22, 2019 - Newly developed model predicts salmonella outbreaks several months in advance
April 22, 2019 - Deep-learning model better predicts survival outcomes for lung cancer
April 22, 2019 - One in Three U.S. Adults Aged 35 to 44 May Have Drinking Problem
April 22, 2019 - Why the measles virus is so contagious
April 22, 2019 - Magnet ‘Zap’ to the Brain Might Jumpstart Aging Memory
April 22, 2019 - Immune response to gut microbes may be early indicator of type 1 diabetes
April 22, 2019 - Destination Limbo: Health Suffers Among Asylum Seekers In Crowded Border Shelter
April 22, 2019 - Research shows how dopamine contributes to sex differences in worms
April 22, 2019 - Marijuana users weigh less compared to non-users
April 22, 2019 - Research uncovers critical RNA processing aberrations in ALS and FTD
April 22, 2019 - Many cancer patients use marijuana and prescription opioids, study reveals
April 22, 2019 - Frailty may up fracture risk in patients with type 2 diabetes
April 22, 2019 - Study provides new insight into how obesity, insulin resistance can affect cognition
April 22, 2019 - Study seeks to better understand the genetic causes for hypospadias
April 22, 2019 - FDA grants approval of first generic naloxone nasal spray to treat opioid overdose
April 22, 2019 - FDA authorizes marketing of first medical device to treat ADHD
April 22, 2019 - Vanderbilt researchers to develop and test ‘safe harbor’ standards of care
April 22, 2019 - You’re probably brushing your teeth wrong – here are four tips for better dental health
April 22, 2019 - Pharmacy closures contribute to medication non-adherence among heart patients
April 22, 2019 - Using Edge AI technology to observe behavior of cattle
April 22, 2019 - Bacteria play a role in the development of stomach ulcers in pigs
April 22, 2019 - Hand Hygiene Compliance Poor in Task Transitions
April 22, 2019 - smoking could harm your baby
April 22, 2019 - Scientists identify rare, paradoxical response to antiretroviral therapy
April 21, 2019 - More TV, Tablets, More Attention Issues at Age 5
April 21, 2019 - Drug reduces risk of kidney failure in people with diabetes, study finds
April 21, 2019 - New research identifies novel link between antibiotic resistance and climate change
April 21, 2019 - Simple intervention can provide lasting protection for teens against junk food marketing
April 21, 2019 - The protein p38-gamma identified as a new therapeutic target in liver cancer
April 21, 2019 - Novel system enables researchers to study bacteria within mini-tissues in a dish
April 21, 2019 - Discovery of oral cancer biomarkers could save thousands of lives
April 21, 2019 - Geneva Exhibition committee gives gold medals to two medications developed by Kazan
April 21, 2019 - Scientists aim to minimize or eliminate hair loss during cancer treatment
April 21, 2019 - WiFi interacts with signaling pathways in the human brain
April 21, 2019 - Stroke Hospitalizations Down in Black, White Medicare Enrollees
April 21, 2019 - First common risk genes discovered for autism
April 21, 2019 - Researchers map auditory sensory system of the mouse brain
April 21, 2019 - Scientists Bring Pig’s Brain, Dead 4 Hours, Back to ‘Cellular Activity’
April 21, 2019 - Virtual reality a promising tool for reducing fears and phobia in autism
April 21, 2019 - New analysis lists out opportunities for U.S. medical schools to advance population health
April 21, 2019 - More sleep may help teens with ADHD focus and organize
April 21, 2019 - Breakthrough antibody treatment suppresses HIV without antivirals
April 21, 2019 - AveXis Data Reinforce Effectiveness of Zolgensma in Treating Spinal Muscular Atrophy (SMA) Type 1
April 21, 2019 - Is your hand pain arthritis, carpal tunnel or something else?
April 21, 2019 - Measles outbreaks may become more frequent if vaccination rates continue to decline
April 21, 2019 - Researchers succeed in accelerating process of creating 3D images
April 21, 2019 - Tiny worm mimics key genetic risk for Alzheimer’s
April 21, 2019 - Angry dreams explained by brain waves
April 20, 2019 - Parenteral Antimicrobial Tx at Home Burdens Children’s Caregivers
April 20, 2019 - Diabetes treatment may keep dementia, Alzheimer’s at bay
Vertex gets European CHMP positive opinion for KALYDECO to treat patients with cystic fibrosis

Vertex gets European CHMP positive opinion for KALYDECO to treat patients with cystic fibrosis

image_pdfDownload PDFimage_print

Vertex Pharmaceuticals (Europe) Limited today announces that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KALYDECO® (ivacaftor) to include the treatment of people with cystic fibrosis (CF) aged 12 to <24 months who have at least one of the following nine mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

If the European Commission issues a favorable adoption of the EMA CHMP opinion for the extension of indication, ivacaftor will be the first and only medicine approved in Europe to treat the underlying cause of CF in patients aged 12 to <24 months, who have specific mutations in the CFTR gene.

“Cystic fibrosis is a chronic, progressive disease that is present at birth, with symptoms often occurring in infancy, so early treatment is crucial to deliver the best possible outcomes for patients,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “Today’s announcement marks an important step towards allowing young CF patients to benefit from treatment at an early stage of their disease, and brings us one step closer to our goal of treating all people living with CF.”

The submission was supported by data from the ongoing Phase 3 open-label safety study (ARRIVAL) of children with CF aged 12 to <24 months who have one of 10 mutations in the CFTR gene that demonstrated a safety profile consistent with that observed in previous Phase 3 studies of older children and adults, and improvements in sweat chloride, a key secondary efficacy endpoint.

Ivacaftor is already approved in Europe for the treatment of CF in patients aged two years and older who have one of the nine following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. It is also approved for the treatment of CF in patients aged 18 years and older who have an R117H mutation in the CFTR gene.

 

Posted in: Drug Trial News | Medical Condition News

Tags: Alanine, Biotechnology, Cell, Cell Membrane, Children, Cough, Cystic Fibrosis, Fibrosis, Gene, Genes, Genetic, Genotyping, Inherit, Liver, Lungs, Mutation, Pharmaceuticals, Protein, Research, Respiratory

Tagged with:

About author

Related Articles