Accessibility Statement Skip Navigation
  • Resources
  • Blog
  • Journalists
  • +44 (0)20 7454 5110
  • Client Login
  • Send a Release
Return to PR Newswire homepage
  • News
  • Products
  • Contact
When typing in this field, a list of search results will appear and be automatically updated as you type.

Searching for your content...

No results found. Please change your search terms and try again.
  • News in Focus
      • Browse News Releases

      • All Public Company News
      • All Multimedia News
      • View All News Releases

      • Regulatory News

      • D/A/CH Regulatory News
      • UK Regulatory News
      • View All Regulatory News

  • Business & Money
      • Auto & Transportation

      • Aerospace & Defense
      • Air Freight
      • Airlines & Aviation
      • Automotive
      • Maritime & Shipbuilding
      • Railroads & Intermodal Transportation
      • Supply Chain/Logistics
      • Transportation, Trucking & Railroad
      • Travel
      • Trucking & Road Transportation
      • View All Auto & Transportation

      • Business Technology

      • Blockchain
      • Broadcast Tech
      • Computer & Electronics
      • Computer Hardware
      • Computer Software
      • Data Analytics
      • Electronic Commerce
      • Electronic Components
      • Electronic Design Automation
      • Financial Technology
      • High Tech Security
      • Internet Technology
      • Nanotechnology
      • Networks
      • Peripherals
      • Semiconductors
      • View All Business Technology

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film & Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Financial Services & Investing

      • Accounting News & Issues
      • Acquisitions, Mergers & Takeovers
      • Banking & Financial Services
      • Bankruptcy
      • Bond & Stock Ratings
      • Conference Call Announcements
      • Contracts
      • Cryptocurrency
      • Dividends
      • Earnings
      • Earnings Forecasts & Projections
      • Financing Agreements
      • Insurance
      • Investments Opinions
      • Joint Ventures
      • Mutual Funds
      • Private Placement
      • Real Estate
      • Restructuring & Recapitalisation
      • Sales Reports
      • Shareholder Activism
      • Shareholder Meetings
      • Stock Offering
      • Stock Split
      • Venture Capital
      • View All Financial Services & Investing

      • General Business

      • Awards
      • Commercial Real Estate
      • Corporate Expansion
      • Earnings
      • Environmental, Social and Governance (ESG)
      • Human Resource & Workforce Management
      • Licensing
      • New Products & Services
      • Obituaries
      • Outsourcing Businesses
      • Overseas Real Estate (non-US)
      • Personnel Announcements
      • Real Estate Transactions
      • Residential Real Estate
      • Small Business Services
      • Socially Responsible Investing
      • Surveys, Polls & Research
      • Trade Show News
      • View All General Business

  • Science & Tech
      • Consumer Technology

      • Artificial Intelligence
      • Blockchain
      • Cloud Computing/Internet of Things
      • Computer Electronics
      • Computer Hardware
      • Computer Software
      • Consumer Electronics
      • Cryptocurrency
      • Data Analytics
      • Electronic Commerce
      • Electronic Gaming
      • Financial Technology
      • Mobile Entertainment
      • Multimedia & Internet
      • Peripherals
      • Social Media
      • STEM (Science, Tech, Engineering, Math)
      • Supply Chain/Logistics
      • Wireless Communications
      • View All Consumer Technology

      • Energy & Natural Resources

      • Alternative Energies
      • Chemical
      • Electrical Utilities
      • Gas
      • General Manufacturing
      • Mining
      • Mining & Metals
      • Oil & Energy
      • Oil & Gas Discoveries
      • Utilities
      • Water Utilities
      • View All Energy & Natural Resources

      • Environ­ment

      • Conservation & Recycling
      • Environmental Issues
      • Environmental Policy
      • Environmental Products & Services
      • Green Technology
      • Natural Disasters
      • View All Environ­ment

      • Heavy Industry & Manufacturing

      • Aerospace & Defence
      • Agriculture
      • Chemical
      • Construction & Building
      • General Manufacturing
      • HVAC (Heating, Ventilation & Air-Conditioning)
      • Machinery
      • Machine Tools, Metalworking & Metallurgy
      • Mining
      • Mining & Metals
      • Paper, Forest Products & Containers
      • Precious Metals
      • Textiles
      • Tobacco
      • View All Heavy Industry & Manufacturing

      • Telecomm­unications

      • Carriers & Services
      • Mobile Entertainment
      • Networks
      • Peripherals
      • Telecommunications Equipment
      • Telecommunications Industry
      • VoIP (Voice over Internet Protocol)
      • Wireless Communications
      • View All Telecomm­unications

  • Lifestyle & Health
      • Consumer Products & Retail

      • Animals & Pets
      • Beers, Wines & Spirits
      • Beverages
      • Bridal Services
      • Cannabis
      • Cosmetics & Personal Care
      • Fashion
      • Food & Beverages
      • Furniture & Furnishings
      • Home Improvement
      • Household, Consumer & Cosmetics
      • Household Products
      • Jewellery
      • Non-Alcoholic Beverages
      • Office Products
      • Organic Food
      • Product Recalls
      • Restaurants
      • Retail
      • Supermarkets
      • Toys
      • View All Consumer Products & Retail

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film & Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Health

      • Biometrics
      • Biotechnology
      • Clinical Trials & Medical Discoveries
      • Dentistry
      • FDA Approval
      • Fitness/Wellness
      • Health Care & Hospitals
      • Health Insurance
      • Infection Control
      • International Medical Approval
      • Medical Equipment
      • Medical Pharmaceuticals
      • Mental Health
      • Pharmaceuticals
      • Supplementary Medicine
      • View All Health

      • Sports

      • General Sports
      • Outdoors, Camping & Hiking
      • Sporting Events
      • Sports Equipment & Accessories
      • View All Sports

      • Travel

      • Amusement Parks & Tourist Attractions
      • Gambling & Casinos
      • Hotels & Resorts
      • Leisure & Tourism
      • Outdoors, Camping & Hiking
      • Passenger Aviation
      • Travel Industry
      • View All Travel

  • Policy & Public Interest
      • Policy & Public Interest

      • Animal Welfare
      • Corporate Social Responsibility
      • Economic News, Trends & Analysis
      • Education
      • Environmental
      • European Government
      • Labour & Union
      • Natural Disasters
      • Not For Profit
      • Public Safety
      • View All Policy & Public Interest

  • People & Culture
      • People & Culture

      • Aboriginal, First Nations & Native American
      • African American
      • Asian American
      • Children
      • Diversity, Equity & Inclusion
      • Hispanic
      • Lesbian, Gay & Bisexual
      • Men's Interest
      • People with Disabilities
      • Religion
      • Senior Citizens
      • Veterans
      • Women
      • View All People & Culture

  • Overview
  • Distribution
  • Paid Placement
  • Multichannel Amplification
  • Disclosure Services
  • SocialBoost
  • Rooms
    • MediaRoom
    • ESG Rooms
  • AI Tools
  • General Enquiries
  • Media Enquiries
  • Partnerships
  • Hamburger menu
  • Cision PR Newswire UK provides press release distribution, targeting, monitoring, and marketing services
  • Send a Release
    • Phone

    • +44 (0)20 7454 5110 from 8 AM - 5:30 PM GMT

    • ALL CONTACT INFO
    • Contact Us

      +44 (0)20 7454 5110
      from 8 AM - 5:30 PM GMT

  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists
  • News in Focus
    • Browse News Releases
    • Regulatory News
  • Business & Money
    • Auto & Transportation
    • Business Technology
    • Entertain­ment & Media
    • Financial Services & Investing
    • General Business
  • Science & Tech
    • Consumer Technology
    • Energy & Natural Resources
    • Environ­ment
    • Heavy Industry & Manufacturing
    • Telecomm­unications
  • Lifestyle & Health
    • Consumer Products & Retail
    • Entertain­ment & Media
    • Health
    • Sports
    • Travel
  • Policy & Public Interest
    • Policy & Public Interest
  • People & Culture
    • People & Culture
  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists
  • Overview
  • Distribution
  • Paid Placement
  • Multichannel Amplification
  • Disclosure Services
  • Cision Communications Cloud®
  • AI Tools
  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists
  • General Enquiries
  • Media Enquiries
  • Partnerships
  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists

European Commission Grants Orphan Medicinal Product Designation for Emmaus Medical's Sickle Cell Treatment


News provided by

Emmaus Medical, Inc.

17 Jul, 2012, 13:45 GMT

Share this article

Share toX

Share this article

Share toX

-Company Provides Update on Phase III U.S. FDA Clinical Trials for the Treatment of Sickle Cell Disease-

TORRANCE, California, July 17, 2012 /PRNewswire/ -- Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine technology company, and subsidiary of Emmaus Life Sciences, Inc., today announced that the European Commission (EC) has granted Orphan Medicinal Product designation for the company's investigational drug Levoglutamide (L-glutamine) for the treatment of sickle cell disease. The EC designation follows the recommendation of the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products announced in May.

Sickle cell disease affects approximately 2.5 in 10,000 people in the European Union, according to studies filed with EMA. However, in some European countries, the concentration of those suffering from sickle cell disease is higher as a result of demographics, migration and geographic mobility, surpassing genetic disorders such as hemophilia and cystic fibrosis, according to a study in the Hematology Journal.

"The positive decision by the European Commission paves the way for us to provide accessible treatment to people throughout Europe who suffer from this debilitating disease," said Yutaka Niihara, M.D., MPH, founder and CEO of Emmaus. "Throughout the world a new treatment option for sickle cell disease is urgently needed, and we are committed to bringing a treatment to market for this severely underserved patient population."

Emmaus said it currently is conducting a clinical trial that is in Phase III with the U.S. Food and Drug Administration (FDA) to study L-glutamine as a treatment for sickle cell disease. Research is being conducted at over 30 clinical trial sites around the country with over 175 people presently enrolled. The company anticipates total enrollment of more than 200 and aims to complete the trial in 2013.

"We are pleased to have received this important designation, which provides for marketing exclusivity in the region and further enhances our efforts to team with a strategic partner, both in Europe and in other key regions," added Henry McKinnell, Ph.D., Chairman of the Board of Emmaus Life Sciences, Inc. and former CEO of Pfizer, Inc.

The patent protected treatment, whose research was led by Dr. Niihara and investigators at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, has both an orphan drug designation and fast track status in the United States.

For more information about the clinical trial, please visit the Emmaus Medical website at www.emmausmedical.com or www.clinicaltrials.gov (NCT01179217).

About Orphan Drug Status in the European Union
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 Sickle Cell Disease
Sickle cell disease is an inherited blood disorder causing red blood cells to become oxidized, forming rigid and sickle shaped cells that block small blood vessels. The condition causes debilitating pain crises and organ damages that can lead to death at an early age. An estimated 200,000 people in the United States and the Europe Union, and four to five million people worldwide, primarily in Latin America and Africa, are afflicted. Currently, there is no universal cure for sickle cell disease.

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.

Forward-Looking Statements
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 completion of the Phase III clinical trial in 2013, 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.

Contacts:

Media:
Lori Teranishi for Emmaus Medical, Inc.
+1-415-981-1964
lteranishi@iqprinc.com

Investors:
Matt Sheldon for Emmaus Medical, Inc.
+1-310-279-5975
msheldon@pondel.com

Clinical Trial Information:
Dr. Henry Macan or Dr. Rafael Razon
+1-310-214-0065
scdstudy@emmausmedical.com

Modal title

Contact PR Newswire

  • +44 (0)20 7454 5110
    from 8 AM - 5:30 PM GMT
  • General Enquiries
  • Media Enquiries
  • Partnerships

Products

  • Content Distribution
  • Multimedia Services
  • Disclosure Services
  • Cision Communications Cloud®

About

  • About PR Newswire
  • About Cision
  • Partnering Opportunities
  • Careers
  • Accessibility Statement
  • APAC
  • APAC - Simplified Chinese
  • APAC - Traditional Chinese
  • Brazil
  • Canada
  • Czech
  • Denmark
  • Finland
  • France
  • Germany
  • India
  • Indonesia
  • Israel
  • Japan
  • Korea
  • Mexico
  • Middle East
  • Middle East - Arabic
  • Netherlands
  • Norway
  • Poland
  • Portugal
  • Russia
  • Slovakia
  • Spain
  • Sweden
  • United States
  • Vietnam

My Services

  • All News Releases
  • Customer Portal
  • Resources
  • Blog
  • Journalists
  • Data Privacy

Do not sell or share my personal information:

  • Submit via Privacy@cision.com 
  • Call Privacy toll-free: 877-297-8921

Contact PR Newswire

Products

About

My Services
  • All News Releases
  • Customer Portal
  • Resources
  • Blog
  • Journalists
+44 (0)20 7454 5110
from 8 AM - 5:30 PM GMT
  • Terms of Use
  • Privacy Policy
  • Information Security Policy
  • Site Map
  • RSS
  • Cookie Settings
Copyright © 2025 PR Newswire Europe Limited. All Rights Reserved. A Cision company.