Following a milestone approval by the European Commission in July 2023, Vertex Pharmaceuticals has been granted a label expansion for its cystic fibrosis medicine KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) with ivacaftor.
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For the first time, a medicine to treat the most common form of cystic fibrosis has been approved in paediatric patients aged one to under two years old.
Cystic fibrosis patients can safely take a dual combination of cystic fibrosis modulator drugs for four years, a study has shown.
Safety results from a first-in-human study showed that orally inhaled murepavadin, a novel macrocycle compound, was well tolerated at all dose levels.
The top 20 biopharma companies averaged a market capitalisation decline of $3.45 trillion in Q2 to $3.14 trillion in Q3 2022, says GlobalData.
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.
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.