The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to VX-548 for the treatment of moderate to severe acute pain.

VX-548 selectively inhibits NaV1.8, a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. The designation is based on data from two phase 2 proof-of-concept studies that evaluated the efficacy and safety of VX-548 for the treatment of acute pain following abdominoplasty (ClinicalTrials.gov Identifier: NCT05034952) and bunionectomy surgery (ClinicalTrials.gov Identifier: NCT04977336). To support evaluation of the treatment effect, the studies also included an active reference arm (hydrocodone bitartrate/acetaminophen).

Findings from both studies showed that treatment with VX-548 resulted in a rapid, statistically significant and clinically meaningful improvement in the primary endpoint of the time-weighted Sum of Pain Intensity Difference over 48 hours vs placebo. VX-548 was generally well tolerated with most adverse events being mild to moderate. The most common adverse events were headache and nausea.

The Company plans to advance VX-548 into phase 3 clinical trials in the fourth quarter of 2022, and intends to initiate a phase 2 dose-ranging study of VX-548 for the treatment of neuropathic pain at the end of 2022.

“We are very pleased to complete our discussions with the FDA and reach agreement on the design of the pivotal development program for acute pain,” said Carmen Bozic, MD, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “VX-548 has demonstrated a positive benefit-risk profile in phase 2 and we are working with urgency to bring forward a medicine that delivers effective pain control without addictive potential to patients who are waiting.”

References

  1. Vertex Advances VX-548 in Acute and Neuropathic Pain. News release. Vertex Pharmaceuticals Inc. July 22, 2022. Accessed July 25, 2022. https://news.vrtx.com/news-releases/news-release-details/vertex-advances-vx-548-acute-and-neuropathic-pain
  2. Vertex announces statistically significant and clinically meaningful results from two phase 2 proof-of-concept studies of VX-548 for the treatment of acute pain. News release. Vertex Pharmaceuticals Inc. March 31, 2022. Accessed July 25, 2022. https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-statistically-significant-and-clinically