Emmaus Medical, Inc.'s Sickle Cell Treatment Receives Positive Opinion for Orphan Drug Status from European Medicines Agency
Published: May 29, 2012
TORRANCE, Calif., May 29, 2012 /PRNewswire/ -- Emmaus Medical, Inc., a pharmaceutical company developing a new treatment for sickle cell disease, and subsidiary of Emmaus Life Sciences, Inc., announced today that its investigational L-glutamine treatment for sickle cell disease has received a positive opinion recommending orphan medicinal product designation by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP). The designation allows Emmaus ten years of marketing exclusivity in EU member countries after the product obtains marketing authorization, as well as streamlined regulatory review processes and registration and other fee reductions.
"We are very pleased with the EMA's recognition of our L-glutamine therapy as an orphan medicinal product for the treatment of sickle cell disease in Europe," said Yutaka Niihara, M.D., MPH, founder and CEO of Emmaus. "This designation is an important milestone in our effort to make this treatment available to millions of patients around the world who currently have very few therapies available to them."
Emmaus Medical has already received orphan drug and fast track designations from the U.S. Food and Drug Administration (FDA) for its patented L-glutamine drug, and is seeking FDA approval for L-glutamine as a treatment of sickle cell disease. The drug was originally developed by investigators led by Dr. Niihara at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center and is now being tested in a Phase III clinical trial at sickle cell research centers in the United States.
"Receiving orphan drug status in two major global regions will allow Emmaus Medical to continue its effort to provide L-glutamine treatment to sickle cell patients who currently have few options for treating this devastating disease," said Dr. Henry McKinnell, Chairman of the Board of Emmaus Life Sciences, Inc. and former CEO of Pfizer, Inc.
About Orphan Drug Status in the EU
The EMA grants orphan drug status for products that can be used in the diagnosis, treatment, or prevention of life-threatening or chronically debilitating conditions with an incidence rate of no greater than 5 in 10,000 individuals in the European Union. Orphan status is conferred when either no satisfactory method of diagnosis, prevention or treatment of the authorized condition exists, or, if such a method does exist, the treatment must be of significant benefit to patients affected by the condition. Orphan status allows the company 10 years of marketing exclusivity in all EU member countries after approval, and other benefits during the development and regulatory review process including scientific assistance for study protocols, access to a centralized review process covering all EU member countries, and reduced or waived registration and marketing authorization application fees.
About Emmaus Medical, Inc.
Founded in 2000, Emmaus Medical, Inc. is a specialty pharmaceutical company, and subsidiary of Emmaus Life Sciences, Inc., dedicated to the discovery, development and commercialization of innovative and cost-effective treatments and therapies for rare diseases. The company is completing its Phase III clinical trial for a treatment for sickle cell disease and has entered into a collaborative agreement for the research, development and commercialization of regenerative medicine technology products. For more information, please visit www.emmausmedical.com and www.nutrestore.com.
This press release contains forward-looking statements that involve substantial risks and uncertainties. These statements are often, but not always, made through the use of words or phrases such as "anticipates," "expects," "plans," "believes," "intends," and similar words or phrases. These forward-looking statements include, without limitation, statements regarding the potential for the L-glutamine treatment for sickle cell disease, the timing, progress and anticipated results of the clinical development of the L-glutamine treatment for sickle cell disease, Emmaus' ability to fund the development of the L-glutamine treatment to completion, as well as Emmaus' plans and objectives. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. Various important factors could cause actual results or events to differ materially from the forward-looking statements that we make. Such factors include, among others, risks that the results of clinical trials will not support our claims or beliefs concerning the effectiveness of the L-glutamine treatment or any of our other product candidates, our ability to finance the development of our product candidates, regulatory risks, including our ability to obtain FDA, European Commission and other regulatory approval for L-glutamine treatment for sickle-cell disease, our ability to commercialize our L-glutamine treatment for sickle-cell disease, and our reliance on third party researchers and other collaborators. Additional risks and uncertainties are described in reports filed by Emmaus Life Sciences, Inc. with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2011. Emmaus is providing this information as of the date of this press release and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or otherwise.
Lori Teranishi for Emmaus Medical, Inc.
Matt Sheldon for Emmaus Medical, Inc.
SOURCE Emmaus Medical, Inc.