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CHMP meeting highlights – April 2025
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of a variety of new medicines, including several treatments for hereditary and rare conditions.
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Cystic Fibrosis
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Next-in-class combination treatment shows potential in cystic fibrosis
Phase III trials have found CFTR modulator vanzacaftor/tezacaftor/deutivacaftor (vanza triple) to be non-inferior to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in improving cystic fibrosis lung function.
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Vertex scores European cystic fibrosis medicine approval
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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European Commission extends approval of cystic fibrosis medicine
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.
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Dual cystic fibrosis modulator therapy shows efficacy in four-year study
Cystic fibrosis patients can safely take a dual combination of cystic fibrosis modulator drugs for four years, a study has shown.
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Promising first-in-human study results for orally inhaled antibiotic
Safety results from a first-in-human study showed that orally inhaled murepavadin, a novel macrocycle compound, was well tolerated at all dose levels.
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Top biopharma firms report Q3 market capitalisation decline
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.
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Cystic fibrosis phage therapy trial commences
The National Institute of Allergy and Infectious Diseases (NIAID)-supported clinical trial will assess whether the bacteriophage therapy WRAIR-PAM-CF1 safely reduces the quantity of Pseudomonas aeruginos bacteria in fibrocystic lungs.
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Using RWE in rare disease drug development: effective innovations with historical controls
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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Kaftrio® with ivacaftor approved by EC to treat children with cystic fibrosis
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.
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Developing and delivering live biotherapeutic products
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…
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Global nebulisers market to decline after COVID-19
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.
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EMA’s transformative treatments of 2020
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
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DECODR app identifies unintentional DNA mutations from CRISPR gene therapies
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.
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What impact does disease rarity have on rare and orphan drug pricing?
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.
