Ivacaftor deuterated

Vertex's next-gen cystic fibrosis treatment is a once-daily triple CFTR modulator regime. Vertex Pharmaceuticals is ahead of schedule, filing a new drug application for its next-gen cystic fibrosis (CF) treatment dubbed vanza triple. Vertex completed the regulatory submission a month before its goal of mid-2024, CEO Reshma Kewalramani, M.D., said on a first-quarter earnings call May 6. The investigational 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 triple for people with CF aged 6 years and older. The Boston-based company also completed a submission in Europe for the same indication and is on track to complete submissions in the U.K., Canada, Australia, New Zealand and Switzerland, Kewalramani said. In the U.S., Vertex intends to use its FDA priority review voucher, which guarantees an expedited six-month review process. Other big updates from the biotech include 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, Vertex has resumed dosing and completed enrollment of 17 patients who have Type 1 diabetes with impaired hypoglycemic awareness 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 patients—at the American Diabetes Association Scientific Sessions conference in June. The two updates come a few weeks after Vertex announced plans to sell autoimmune and inflammatory disease-focused Alpine Immune Sciences for $4.9 billion. The transaction is expected to close in the second quarter of this year.

Vertex Pharmaceuticals has kicked off 2024 by staying busy, submitting a new drug application to the FDA for its cystic fibrosis (CF) therapy, vanzacaftor triple combination, and a corresponding marketing authorization application to the European Medicines Agency. The vanzacaftor/tezacaftor/deutivacaftor combination has received Fast Track and Orphan Drug designations from the FDA for CF treatment. Vertex submitted its NDA using a priority review voucher. In CF, mutations reduce the amount and function of the CFTR protein on cell surfaces. The medications vanzacaftor and tezacaftor help increase CFTR protein levels by improving its processing and transport to the cell surface. Deutivacaftor, meanwhile, enhances the protein's function, boosting salt and water flow across cell membranes. In addition to its cystic fibrosis advancements, Vertex also recently reported a strong financial performance for the first quarter with product revenues rising to $2.69 billion, a 13% increase from the first quarter of 2023. This growth is attributed to the continued success of blockbuster Trikafta in both U.S. and international markets, especially following its approval for younger cystic fibrosis patients. The drugmaker has also initiated a rolling NDA submission for suzetrigine (VX-548) for the treatment of moderate-to-severe acute pain, expected to be completed within the quarter. Suzetrigine represents a new class of pain management solutions that could potentially offer an alternative to traditional opioids. Recently, Vertex also announced the acquisition of Alpine Immune Sciences. The deal includes povetacicept, a phase 3-ready asset for IgA nephropathy (IgAN), poised to become a leading treatment option for this chronic kidney disease.