Spinal injury drug granted orphan designation by FDA
Amphix Bio’s lead therapy for spinal cord injury has been granted orphan drug status by the US Food and Drug Administration, potentially speeding up access for patients.
The treatment, AMFX-200, uses engineered peptides—small protein fragments—that act as drugs to activate cell receptors and as scaffolds to support tissue regeneration.
The designation is reserved for conditions affecting fewer than 200,000 people in the US. Around 18,000 spinal cord injuries occur annually, often resulting in permanent paralysis.
AMFX-200 is based on supramolecular therapeutic peptides (STPs) developed by Professor Samuel Stupp’s research group at Northwestern University.
These molecules can control the collective motion of nanofibres, enhancing biological potency and promoting neural regeneration.
Dr James Guest, neurosurgeon at the University of Miami and the Miami Project to Cure Paralysis, said: “Experiencing a spinal cord injury is devastating for patients and their families, as it leads to a lifetime of lost mobility, high healthcare costs, and a heavy reliance on caregivers.
“Previous attempts at treatments have faced many limitations but Amphix Bio is taking a completely new approach to neural regeneration, with the potential for a breakthrough in the field.”
In preclinical models of acute injury, a single injection of AMFX-200 into the spinal cord allowed motor neurons to regrow past the injury site, re-establishing connections and restoring movement in treated animals.
The company reports this reversed paralysis in the injected area.
Amphix Bio plans to apply its STP platform to additional neurological and musculoskeletal conditions.
It aims to build on established biology and areas of high-impact cellular regeneration to create viable clinical therapies.
AMFX-200 follows the company’s earlier product, AMFX-100, a bone graft substitute for spinal fusion surgery.
It enables procedures without donor tissue or recombinant proteins and received FDA breakthrough device designation in October 2024.
Stupp said: “This designation from the FDA is a major milestone for supramolecular therapeutics and validates the high unmet need that our approach addresses.
“The expedited assessment and review are especially important given that we are aiming to advance an entirely new regenerative medicine platform to the clinic.”
