Adolore BioTherapeutics has received Orphan Drug Designation (ODD) from the FDA for its Kv7-activating rdHSV-CA8 gene therapy.
The designation covers treatment of primary and secondary erythromelalgia (EM), a rare and debilitating neuropathic pain disorder.
The FDA’s Office of Orphan Products Development grants ODD to therapies addressing serious rare diseases.
For Adolore, this could unlock:
According to CEO Roelof Rongen, the designation validates both the disease focus and Adolore’s scientific approach.
Erythromelalgia is a rare, lifelong neuropathic pain condition.
Patients experience:
Triggers include:
There is no FDA-approved therapy for EM today.
Primary EM is often caused by gain-of-function mutations in the SCN9A gene.
This gene encodes the Nav1.7 sodium channel, critical in pain signaling neurons.
These mutations cause:
Adolore’s approach addresses this upstream dysfunction.
Adolore’s therapy uses a replication-defective HSV vector for localized delivery.
Its mechanism:
This targets pain without systemic drug exposure.
Small-molecule Kv7 activators have failed before. Despite strong efficacy, they caused:
Adolore avoids these issues through local gene delivery, not oral drugs.
According to founder Dr. Roy Clifford Levitt, rdHSV-CA8 has shown:
Critically, pain relief was equipotent to opioids, without opioid-related side effects.
Adolore is advancing two key programs:
The EM program adds strategic optionality and accelerates clinical validation.
Safe, effective non-opioid chronic pain treatments remain scarce.
This gap is acute amid:
Adolore positions rdHSV-CA8 as a Disease Modifying Anti-Pain Therapy (DMAP).
FDA Orphan Drug Designation strengthens Adolore’s path toward:
For EM patients with no approved options, this is a meaningful step forward.