Vertex’s VX-548 Gets Breakthrough Therapy Designation for Acute Pain
Vertex Pharmaceuticals’ investigational oral drug VX-548 has received FDA’s Breakthrough Therapy designation for the treatment of patients with moderate-to-severe acute pain.
The investigational drug is an oral, selective inhibitor of the NaV1.8 sodium ion channel that plays a critical role in pain signaling in the peripheral nervous system.
Following positive phase 2 data, Vertex plans on advancing VX-548 into phase 3 clinical trials in the fourth quarter this year. The company also plans on launching a phase 2 dose-ranging study of VX-548 in neuropathic pain by the end of this year.
The FDA’s Breakthrough Therapy designation is meant to expedite the development and review of drugs that are intended to treat a serious condition. The designation requires preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapies.
About VX-548
VX-548 is an oral, selective NaV1.8 inhibitor that is highly selective for NaV1.8 relative to other NaV channels. NaV1.8 is a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. NaV1.8 is a genetically validated target for the treatment of pain, and Vertex has previously demonstrated clinical proof-of-concept with a small molecule investigational treatment targeting NaV1.8 in multiple pain indications including acute pain, neuropathic pain and musculoskeletal pain. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class of medicines that have the potential to provide superior relief of acute pain without the limitations of opioids, including their addictive potential. VX-548 is the most recent molecule to enter clinical development from Vertex’s portfolio of NaV1.8 inhibitors.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com.
August 2, 2022
