After talks with the FDA, Vertex Pharmaceuticals has been given the go-ahead to advance its non-opioid pain killer into phase 3 trials, which are set to begin in the last quarter of this year.
The news follows proof-of-concept phase 2 results released this March for Vertex’s drug, dubbed VX-548. Compared to placebo, VX-548 offered rapid relief of pain intensity over 48 hours after bunion-removal or tummy-tuck surgery.
The selective NaV1.8 inhibitor has received an FDA breakthrough tag for patients with moderate to severe pain, a designation intended to expedite development and review.
The Boston biotech has also received an FDA nod to advance VX-548 into a phase 2 trial for treating diabetic peripheral neuropathy pain by the end of the year.
With a high unmet global need for pain management meds and an addiction risk tied to opioid painkillers, there’s an urgent need for new non-opioid painkillers. When considering VX-548’s potential in both acute and neuropathic pain, SVB Securities analysts project the drug could rack up $510 million in 2030 estimated risk-adjusted sales.
The pivotal phase 3 trial will include two placebo-controlled studies evaluating VX-548’s efficacy and safety for patients with moderate to severe acute pain. Both studies will include a hydrocodone bitartrate/acetaminophen treatment arm. A third, single-arm study will assess the drug across other types of moderate to severe acute pain beyond bunion-removal or tummy-tuck surgery patients.
The good news comes on the heels of a Vertex collab with Verve Therapeutics, announced earlier this week. The $60 million partnership aims to use in vivo gene editing to combat a single undisclosed liver disease.
Beyond Vertex’s approved cystic fibrosis medicines, the biotech has a robust pipeline of investigational small molecule, cell, and genetic therapies in other diseases, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, diabetes, alpha-1 antitrypsin deficiency, and Duchenne muscular dystrophy.