FDA grants orphan drug designation to oral frontotemporal dementia treatment

The FDA has granted orphan drug designation to an oral, investigational, small-molecule therapeutic developed to treat frontotemporal dementia.

In a press release, drug manufacturer CervoMed said the ruling was based on results from the AscenD-LB phase 2a clinical trial. Researchers found that treatment with neflamapimod significantly improved dementia severity as measured by Clinical Dementia Rating Sum-of-boxes, as well as functional mobility assessed through the Timed Up and Go Test, compared with placebo.

Further, according to the release, treatment effects with neflamapimod in those with early-stage dementia with Lewy bodies (DLB) were significant when compared with the overall patient population.

The company also stated that neflamapimod demonstrated a favorable safety profile and was generally well-tolerated in a pair of early-stage clinical trials involving 300 participants with the rare, neurodegenerative condition.

Neflamapimod is also under investigation in the ongoing RewinD-LB phase 2b study, a randomized, double-blind, placebo-controlled clinical trial evaluating thrice-daily 40 mg oral neflamapimod in 159 patients with early-stage DLB over 16 weeks. Topline results are expected sometime before the end of the year, the company said.

“We are pleased to have received orphan drug designation as it implicitly recognizes the scientific rationale and potential for neflamapimod to treat this debilitating condition. Patients diagnosed with frontotemporal dementia have no available treatment options, and this rare condition is extremely burdensome to patients and caregivers alike,” CervoMed CEO John Alam, MD, said in the release. “We are in active discussions with clinical thought leaders regarding the design of a proof-of-principle study, while continuing to prepare to advance neflamapimod into a phase 3 trial in mid-2025.”