Vertex requests FDA approval for new cystic fibrosis treatment, resumes diabetes cell therapy trial

Vertex Pharmaceuticals has made significant strides with its next-generation cystic fibrosis (CF) treatment, known as the vanza triple regimen. This new therapy, a once-daily triple CFTR modulator regimen, includes the components vanzacaftor, tezacaftor, and deutivacaftor. Demonstrating notable progress, Vertex has managed to submit the new drug application (NDA) ahead of schedule, as revealed by CEO Reshma Kewalramani, M.D., during a first-quarter earnings call on May 6.

Initially, Vertex aimed to complete the regulatory submission by mid-2024, but the company achieved this milestone a month earlier. The vanza triple regimen has shown promising results in various phase 3 clinical programs, meeting primary goals and proving itself noninferior to Vertex’s existing twice-daily treatment, Trikafta, in terms of enhancing lung function. Moreover, the vanza triple has shown superiority over Trikafta in reducing sweat chloride levels, a key marker in cystic fibrosis management.

Vertex is now seeking approval from the U.S. Food and Drug Administration (FDA) for the use of vanza triple in individuals aged six and older who have cystic fibrosis. The company has also submitted applications in Europe and plans to complete submissions in the U.K., Canada, Australia, New Zealand, and Switzerland. To expedite the approval process in the U.S., Vertex plans to use its FDA priority review voucher, which guarantees a six-month review period.

In addition to advancements in cystic fibrosis treatment, Vertex has also resumed enrollment in its phase 1/2 diabetes trial for VX-880. This trial focuses on an allogeneic, stem cell-derived insulin-producing islet cell therapy. Earlier this year, Vertex voluntarily paused the trial due to two deaths that were deemed unrelated to the treatment. However, following a review by an independent data monitoring committee, Vertex has resumed dosing and completed the enrollment of 17 participants who suffer from Type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia. The company anticipates finishing dosing soon and plans to present updated data on this promising cell therapy at the American Diabetes Association Scientific Sessions conference in June.

These developments come shortly after Vertex announced its intention to acquire Alpine Immune Sciences, a company that focuses on autoimmune and inflammatory diseases, for $4.9 billion. This acquisition is expected to be finalized in the second quarter of this year.

Overall, these updates highlight Vertex Pharmaceuticals' commitment to advancing treatments for cystic fibrosis and diabetes, and expanding its portfolio through strategic acquisitions. The company's proactive approach and timely submissions reflect its dedication to delivering innovative therapies to patients in need.