Vertex advances pipeline with NDA submission for cystic fibrosis

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.