Pilavapadin Improves Diabetic Peripheral Neuropathic Pain, Application of 2025 McDonald Criteria, FDA Expands Eculizumab Indication to Pediatrics

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Welcome to this special edition of Neurology News Network. I'm Marco Meglio.

Newly announced data from the phase 2b PROGRESS study (NCT06203002) showed that treatment with investigational pila-vapadin (Lexicon Pharmaceuticals) resulted in noticeable reductions in pain relative to placebo in adults with moderate to severe diabetic peripheral neuropathic pain (DPNP). These findings, coupled with data from the previously conducted RELIEF-DPN-1 study (NCT04455633), identified 10 mg once daily as an appropriate dose to advance into phase 3 development for DPNP. PROGRESS included 496 patients with a diagnosis of diabetes and moderate to severe DPNP who received once daily pilavapadin doses of 10 mg, 20 mg, or 20 mg for 7 days followed by 10 mg thereafter. On the primary end point of change in average daily pain score (ADPS), patients in the 10 mg, 20mg/10 mg, and 20 mg dose arms achieved reductions of 1.74, 1.70, and 1.38, respectively, over the 8-week treatment period. In comparison, those on placebo demonstrated reductions of 1.31 in ADPS.

Late-breaking findings using data from the CAVS-MS study showed that nearly one-fifth of patients with atypical clinical symptoms or radiological-only presentation met the latest criteria for multiple sclerosis (MS) using the updated 2024 McDonald Criteria revisions. The study suggests that the central vein sign (CVS) is a valuable para-clinical tool for confirming MS diagnosis in individuals with atypical symptoms or radiological-only presentation, thus opening the door for initiating treatment earlier. Presented at the 2025 Americas Committee for Treatment & Research in Multiple Sclerosis (ACTRIMS) Forum, held February 27-March 1, in West Palm Beach, Florida, the analysis included 420 participants who were undergoing evaluation for MS. Of these, 166 were adjudicated as having atypical onset and an additional 25 were adjudicated as having radiological-only presentation. Of this group (n = 191), 19% (n = 36) met diagnostic criteria when applying the 2024 McDonald Criteria, which ultimately will allow for earlier treatment and potentially altering of patients’ disease course.

The FDA has granted approval for an expanded indication of eculizumab (Soliris; Alexion/AstraZeneca) to include both adult and pediatric patients who are 6 years of age or older with generalized myasthenia gravis (gMG) who are antiacetylcholine receptor (AChR) antibody positive. With the approval, it becomes the first and only treatment for pediatric patients living with the disease. The use of eculizumab in pediatric patients for this indication was supported by evidence from a well-controlled trial in adults with additional pharmacokinetic and safety data in pediatric patients with gMG who are 12 years of age and older, as well as pharmacokinetic and safety data in other pediatric populations in the range of at least 6 years to younger than 12 years. In a 26-week, single-arm study of 11 pediatric patients with gMG 12 to 17 years of age, adverse reactions were consistent with those observed in adults living with the disease.

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