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Long-awaited cystic fibrosis drug could turn deadly disease into a manageable condition

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  1. skybrian
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    From the article: [...] [...]

    From the article:

    A new cystic fibrosis therapy dramatically improved patients’ lung function and showed clear signs of targeting the genetic root of the disease [...]

    The drug is the product of decades of steady, incremental scientific work that began with research in academic laboratories and was pushed forward and funded by patient advocates through an unusual “venture philanthropy” model now being emulated by other patient groups. The leap forward was preceded by many steps — Trikafta is the fourth therapy developed by Vertex Pharmaceuticals, a Boston-based company that has built a lucrative franchise around the disease.

    [...]

    Many physicians see the most transformative potential impact of the drug in the hope that it will be eventually approved for younger children, as Vertex’s other drugs have been over time. The drug can help older patients, but it can’t erase years of lung damage; if it works and is safe in younger children, it could prevent damage in the first place.

    [...]

    It also remains to be seen whether patients have an easy time gaining access to the drug, which will cost $311,000 a year.

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