FDA Accepts Vertex's Suzetrigine NDA with Priority Review for Acute Pain

Key Insights

• The FDA has accepted Vertex's NDA for suzetrigine, a novel, oral NaV1.8 inhibitor, for treating moderate-to-severe acute pain, potentially the first new class in over 20 years.
• Suzetrigine received priority review with a PDUFA target date of January 30, 2025, and has been granted Fast Track and Breakthrough Therapy designations by the FDA.
• Acute pain affects over 80 million people annually in the U.S., creating a significant unmet need for non-opioid treatments with improved efficacy and reduced addictive potential.

Vertex Pharmaceuticals announced that the FDA has accepted its New Drug Application (NDA) for suzetrigine, an investigational, oral, selective NaV1.8 pain signal inhibitor, for the treatment of moderate-to-severe acute pain. The FDA has granted suzetrigine priority review, setting a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2025. This drug has the potential to be the first new class of medicine to treat acute pain in over two decades. Suzetrigine has already been granted FDA Fast Track and Breakthrough Therapy designations.

Addressing Unmet Needs in Acute Pain Management

Acute pain, defined as pain lasting less than three months, affects over 80 million people in the U.S. annually. Current treatment options are limited, creating a significant unmet need for new analgesics. According to Scott Weiner, M.D., M.P.H., Vertex Acute Pain Steering Committee Chair, there is a "desperate need for new non-opioid therapies for treating pain," as many patients are either undertreated, experience negative side effects from existing therapies, or avoid pain medication altogether due to concerns about opioid dependence.

Suzetrigine: A Selective NaV1.8 Inhibitor

Suzetrigine (formerly VX-548) is designed to selectively inhibit the NaV1.8 voltage-gated sodium channel, which is primarily expressed in peripheral pain-sensing neurons (nociceptors). By selectively targeting NaV1.8, suzetrigine aims to block the transmission of pain signals without the limitations and risks associated with current therapies, including opioids. Clinical data from three Phase 3 studies and two Phase 2 studies have demonstrated a favorable benefit/risk profile in patients with moderate-to-severe acute pain. A Phase 2 study also showed positive results and a well-tolerated profile in patients with pain associated with diabetic peripheral neuropathy.

Clinical Development and Future Prospects

Beyond acute pain, suzetrigine is also being investigated for diabetic peripheral neuropathy and painful lumbosacral radiculopathy. A Phase III pivotal trial is ongoing for diabetic peripheral neuropathy, and a Phase III trial for painful lumbosacral radiculopathy is anticipated, pending discussions with regulators. If approved, GlobalData estimates that suzetrigine could reach peak sales of $1.4 billion by 2030. Furthermore, the NOPAIN Act, effective January 1, 2025, may provide additional payment for FDA-approved non-opioid pain treatments in hospital outpatient or surgical center settings, potentially benefiting suzetrigine upon approval.