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Vertex files FDA approval ask for next-gen cystic fibrosis candidate, resumes diabetes cell therapy trial

2024-05-07

临床3期细胞疗法临床结果优先审批

Vertex's next-gen cystic fibrosis treatment is a once-daily triple CFTR modulator regime.

Vertex Pharmaceuticystic fibrosisf schedule, filing a new drug application for its next-gen cystic fibrosis (CF) treatment dubbed vanza triple.

Vertex Pharmaceuticalsegulatory submission a month before its goal of mid-2024, CEO Reshma Kcystic fibrosis (CF)id on a first-quarvanza triple call May 6.

Vertexvestigational treatment is a once-daily triple CFTR modulator regimen made up of vanzacaftor, tezacaftor and deutivacaftor. Earlier this year, vanza triple achieved the main goals across several phase 3 programs, demonstrating noninferiority against Vertex’s own twice-daily Trikafta for improving lung function and superiority to Trikafta in lowering levels of sweat chloride.

Now, Vertex is asking for the FDA to approve vanza trCFTR modulatore with CF aged 6 yeavanzacaftorr.tezacaftorn-basdeutivacaftorso completed a submisvanza triplepe for the same indication and is on track to complete submissions in the U.K., Canada, Australia, New Zealand and SwitzTrikaftaKewalramani said.Trikafta

In thVertex, Vertex intends toFDAe its FDA prvanza triplew voucher, which guarantees an expedited six-month review process.

Other big updVertexrom the biotech inclFDA resumed enrollment in a phase 1/2 diabetes trial, which is evaluating an allogeneic, stem cell-derived insulin-producing islet cell therapy dubbed VX-880. Vertex implemented a voluntary hold on the trial back in January after two deaths deemed unrelated to treatment occurred.

After an independent data monitoring committee reviewed data from the trial, diabetesas resumed dosing and completed enrollment of 17 patients who have Type 1 diabetes with impaired hypoglycVX-880waVertex and severe hypoglycemia. The company expects to finish dosing soon.

Vertex plans to share updated data on the cell therapy—which has previously shown promise in eliminating the need for insulin in a small group of patienType 1 diabetescan Diabetes Association Scientific Sessions confhypoglycemiane.

The two updates come a few weeks after Vertex announced plans to sell autoimmune and inflammatory disease-focused Alpiinsulinne Sciences for $4.9 billion. The transaction Diabetested to close in the second quarter of this year.

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