Orphan Drug Designation in USA

Yesterday the FDA granted the request from BioArctic Neuroscience for orphan-drug designation of heparin activated recombinant human Fibroblast Growth Factor 1 (FGF1) in combination with a surgically implanted biodegradeble device for treatment of patients with confirmed traumatic complete spinal cord injury (SCI) where no motor or sensory function is preserved below the injury (American Spinal Injury Association [ASIA] Impairment Scale A.

Medicines for rare diseases so-called ´orphan´ medicinal products are intended for the treatment of life-threatening or chronically debilitating conditions that affect no more than 200 000 in the US.

BioArctic develops a drug to obtain a successful neurosurgical treatment of patients with spinal cord injury. This development is in collaboration with highly innovative Swedish research at the Karolinska Institut, the Karolinska University Hospital, Ångström laboratory and Elos Medtech.

There are no effective treatments for complete traumatic spinal cord injury, which is a chronic, devastating condition leading to markedly impaired life quality and very high economical costs to the society, says Dr. Pär Gellerfors, CEO of BioArctic Neuroscience.

The drug candidate is a growth factor in combination with a biodegradable device. It has demonstrated very promising effects in an animal model of complete spinal cord injury and no adverse effects in toxicology studies. The drug will be used during neurosurgical procedures to recreate a functional spinal cord.