With a growing number of therapies under development for rare diseases, ICON’s Dr William Maier discusses how real-world evidence (RWE) can be effectively used as a historical control (HC), overcoming challenges presented in clinical development.
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Children with cystic fibrosis aged six to11 years could benefit from Kaftrio® in combination with ivacaftor, as a treatment targeting the underlying cause of the disease.
The microbial content of the gut has a well established role in health and disease. In this article, European Pharmaceutical Review’s Hannah Balfour explores the development and formulation of live biotherapeutic products (LBPs), an emerging treatment modality that seeks to capitalise on the interaction between the microbiota and host in…
As the world recovers from COVID-19, research suggests the nebuliser devices market will initially shrink before recovering and reaching a value of $1.55 billion in 2025.
The DECODR app was developed to accelerate the development of CRISPR gene therapies, producing similar data as deep sequencing processes in a shorter timeframe and at less cost.
Bhavesh Patel, Cécile Matthews and Owen Male at CRA’s Life Sciences Practice assess the relationship between drug pricing for rare disease therapies and disease prevalence in Europe, taking a closer look at payer assessments of orphan drugs in Germany.
A clinical trial has shown that antibiotics administered orally are just as effective at killing Pseudomonas aeruginosa in cystic fibrosis patients as intravenous antibiotics.
The European Commission and European Medicines Agency have approved the triple combination Kaftrio for use in certain cystic fibrosis patients, triggering the drug to be accessible on the UK’s National Health Service.
Dapivirine Vaginal Ring (dapivirine) and Blenrep (belantamab mafodotin) have been recommended for approval by the EMA's CHMP, along with nine other medicines.
The European Medicines Agency's human medicines committee (CHMP) said the treatment would offer a therapeutic option for certain cystic fibrosis phenotypes that are currently untreatable.
AbbVie and Allergan have announced the Federal Trade Commission has accepted AbbVie's pending acquisition of Allergan for $63 billion.
New research from PhRMA found that in the US, there are almost 600 paediatric medicines in development in over 2,000 clinical trials.
Researchers at Aston University and Birmingham Children’s Hospital, UK, have discovered a highly effective three-drug approach to treat Mycobacterium abscessus (M. abscessus), a notoriously drug-resistant pathogen that affects up to 13 percent of cystic fibrosis (CF) patients in the UK.
The European Commission has approved the label extension for KALYDECO® (ivacaftor) to include the treatment of infants with cystic fibrosis between six and 12 months old.