Provides a 10-Year Regulatory Exclusivity for the European Community
Newton, Massachusetts – May 23, 2011.
NeuroHealing Pharmaceuticals today announced that the European Commission has granted Orphan Drug Designation EU/3/11/862 for NH001 for the treatment of patients following a moderate or severe traumatic brain injury (TBI). The designation follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) earlier this year. A characteristic feature of severe TBI is prolonged loss of consciousness; the aim of treatment with NH001 is to induce consciousness in these patients. Currently there are no approved pharmaceutical treatment options to induce consciousness for these patients. NH001 previously was granted orphan drug status by the Office of Orphan Products Development (OOPD) of the U.S. Food and Drug Administration (FDA) for the treatment of patients in a vegetative state or minimally conscious state following a brain injury, and the FDA OOPD has awarded NeuroHealing a three-year grant to assist in the clinical development of NH001. NH001 (continuous sc apomorphine infusion) 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 tested under a U.S. IND in a double-blind Phase II clinical study to evaluate the safety and efficacy of NH001 in improving the functional outcome of patients in a vegetative state or minimally conscious state following a severe TBI. Results of a pilot study, demonstrating an improvement in primary outcomes for all treated patients, have been published (Fridman, et al., Brain Injury 2010; 24:636–641). Awakening was seen as rapidly as within the first 24 hours of drug administration and as late as 4 weeks. Patients who completed treatment completely recovered consciousness and improvements were sustained for at least one year. “This orphan drug designation in Europe signifies another important step in the development of NH001, and it bolsters market protections in addition to our patents,” said Neal M. Farber, Ph.D., CEO. “NH001 is currently one of the most promising drugs for the improvement of patients in a coma.” The EMA Orphan Drug application dossier was submitted in collaboration with Britannia & Genus Pharmaceuticals Ltd (both wholly owned UK subsidiaries of STADA Arzneimittel AG).
About European Orphan Drug Designation
European Orphan Drug designation by the European Commission is granted to medicines intended for treatment of life-threatening or chronically debilitating pathologies that affect no more than 5 in 10,000 people in the European Community. Orphan designation by the EMA provides for scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized marketing authorization, and certain financial incentives. The designation also provides 10 years of marketing exclusivity subsequent to product approval. Orphan drugs are eligible for full reduction of fees associated with pre-authorization inspections, as well as full reduction of marketing application fees and annual fees for qualifying companies.
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. The leading causes of VS and MCS are traumatic brain injury (mostly due to motor vehicle accidents and falls) and stroke (ischemic and hemorrhagic). 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.
About NeuroHealing Pharmaceuticals
NeuroHealing Pharmaceuticals, Inc. is a clinical stage private company developing innovative treatments for specialty indications based on repositioning neurologically active compounds. Clinical stage programs include: NH001 (phase II), a dopaminergic agent to help post traumatic brain injury patients to emerge from a coma, vegetative or minimally consciousness state; NH004 (phase II), an anticholinergic agent in a convenient intra-orally muco-adhesive dissolving film to help treat motor neuron disease patients who suffer from sialorrhea (drooling); and NH02D, a neurologically active compound to treat premature ejaculation.