Newton, Mass., May 25, 2006
NeuroHealing Pharmaceuticals, Inc. today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted orphan drug status to NH001 for the treatment of patients in a vegetative state or minimally conscious state for up to twelve months following a brain injury (traumatic or spontaneous). Currently there are no FDA approved pharmaceutical treatment options for these patients.
NH001 is a potent dopaminergic agonist with well-established human safety data, appropriate phamacokinetic profile and convenient route of administration for this patient population.
NH001 is currently being evaluated in an open label Phase IIa clinical study to improve the functional outcome of patients in a vegetative state or minimally conscious state following a severe traumatic brain injury. Interim results of the trial were recently presented at the World Congress for Rehabilitation in Hong Kong in February, 2006 (Fridman, et al., Neurorehabilitation and Neural Repair 2006; 20(1):144).
“The orphan drug designation in the U.S. signifies another important step for NeuroHealing in the development of NH001, and it bolsters our market protections,” said Neal Farber, PhD, CEO. The FDA recently cleared NeuroHealing’s IND for a double-blind, placebo controlled trial to evaluate the safety and potential efficacy of NH001 in improving the functional outcome of patients in a vegetative state or minimally conscious state following a severe TBI.
About Orphan Drug Designation
The FDA Orphan Drug designation is reserved for promising new therapies being developed to treat life-threatening or very serious diseases that affect fewer than 200,000 people in the U.S. In the U.S., the Orphan Drug Act guarantees market exclusivity for seven years to the first sponsor that obtains market approval for an orphan-designated product. This designation provides companies with financial and regulatory benefits during the course of orphan drug development, including tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee.
About Vegetative State and Minimally Conscious State
Vegetative State (VS) and Minimally Conscious State (MCS) are diminished or altered state of consciousness, which result in an impairment of cognitive abilities and physical functioning. These impairments may be either temporary or permanent and cause partial or total functional disability or psychosocial maladjustment. In the United States, the leading causes of VS and MCS are traumatic brain injury (mostly due to motor vehicle accidents and falls) and stroke (ischemic and hemorrhagic). Each year in the United States, up to 120,000 patients will enter into a VS or MCS following a traumatic brain injury. Patients in VS and MCS create a large economic and social burden for the patient’s family and society. It is estimated that the average lifetime health care cost of a patient in a VS is over $1 million.
NeuroHealing Pharmaceuticals, Inc. (www.NeuroHealing.com) is a clinical stage company developing therapies to improve the neuro-rehabilitation process and functional outcome for patients who have suffered debilitating injuries to the central nervous system. Clinical stage programs include: NH001, a dopaminergic agent to help post traumatic brain injury patients to emerge from a coma, vegetative or minimally consciousness state; NH004, an anticholinergic agent in a convenient intra-orally dissolving film to help treat motor neuron patients who suffer from sialorrhea (drooling); and NH02D, a novel mechanism compound to accelerate the rehabilitation outcomes of stroke patients who remain with chronic motor and cognitive disabilities.