Orphan Drug Designations Lead to New Therapeutic Breakthroughs for Rare Disorders and Diseases

CORAL SPRINGS, Florida, April 29, 2015 /PRNewswire/ --

According to a recent article published on the Wall Street Journal, the debate over prescription drug pricing has grown increasingly contentious, particularly as drug makers issue more new medicines with high price tags. But despite the tumult, the cost for orphan drugs - medicines that are designated by the FDA & EU to treat a rare disease and may cost as much as $400,000 annually - is apparently not fazing some insurers. Biotech Companies in focus today are: Amarantus Bioscience Holdings, Inc. (OTCQB: AMBS), Catalyst Pharmaceutical Partners' (NASDAQ: CPRX), Biogen Inc. (NASDAQ: BIIB),  Hemispherx Biopharma, Inc. (NYSE: HEB) and Biocept, Inc. (NASDAQ: BIOC).

Amarantus BioScience Holdings, Inc. (OTCQB: AMBS), a biotechnology company focused on developing therapeutic and diagnostic products for neurological disorders and orphan indications, announced that the European Commission, acting on the positive recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP), has granted orphan drug status for MANF (mesencephalic-astrocyte-derived neurotrophic factor) for the treatment of retinitis pigmentosa (RP). RP refers to a group of inherited diseases causing retinal degeneration often leading to blindness. The Company previously announced in December of 2014 that it also received orphan drug designation for MANF for the treatment of RP from the U.S. Food and Drug Administration (FDA). "We are extremely pleased to have received European orphan drug status for our promising investigational product, MANF in RP," said Gerald E. Commissiong, President & CEO of Amarantus BioScience Holdings, Inc. "This EU orphan designation, combined with our recent U.S. orphan designation, positions MANF for an accelerated global regulatory product development pathway to address the significant unmet need in this important ophthalmologic indication.".  

To read the full AMBS press release, please click here:  http://www.financialnewsmedia.com/profiles/ambs.html 

Orphan drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.

In other Biotech News & Happenings: Catalyst Pharmaceutical Partners' (NASDAQ: CPRX) plan to seek approval and market a drug for a rare muscle-wasting disease is in jeopardy following the surprise disclosure Thursday of positive clinical trial results from a similar, competitive drug. Privately held Jacobus Pharmaceuticals presented a poster Thursday at the American Academy of Neurology annual meeting showing "unequivocal evidence" that 3,4 Dap, the company's experimental drug, is an effective treatment for patients with Lambert-Eaton Myasthenic Syndrome (LEMS). Read the full story at http://www.thestreet.com/story/13125612/1/catalyst-pharma-competitor-surprises-with-positive-orphan-disease-drug-study-results.html?puc=yahoo&cm_ven=YAHOO

George Scangos, Biogen CEO (NASDAQ: BIIB), discusses progress in clinical trials for Alzheimer's treatments, and the outlook for its multiple sclerosis Tecfidera drug. See the video discussion at http://finance.yahoo.com/video/biogen-takes-alzheimers-202900299.html

Hemispherx Biopharma, Inc. (NYSE: HEB) recently announced that the European subsidiary, "Hemispherx Biopharma Europe N.V./S.A" gained a positive opinion from the COMP (Committee on Medical Products) regarding its Orphan Medicinal Product Application for Ampligen(R), an experimental therapeutic, to treat Ebola Virus Disease (EVD). Orphan designation by the European Medicines Agency (EMA) is granted to promote the clinical development of drugs that target rare life-threatening conditions and which are expected to provide significant therapeutic advantage over any existing treatments and includes some tropical diseases primarily found in developing nations as is the case for EVD. Hemispherx submitted in vitro and in vivo data in appropriate preclinical models relevant to the EVD indication to the European Union (EU) Committee for Orphan Medicinal Products. In addition to the efficacy data, clinical safety information was also included in the comprehensive application.

Biocept, Inc. (NASDAQ: BIOC), a molecular oncology diagnostics company specializing in biomarker analysis of circulating tumor DNA (ctDNA) and circulating tumor cells (CTCs), announced recently that it has entered into a clinical collaboration with University of California, San Diego Moores Cancer Center to determine the clinical utility of detecting biomarkers present in CTCs and ctDNA in blood samples for non-small cell lung cancer patients using Biocept's OncoCEE(TM) LU platform and CEE-Selector(TM) technology.

FinancialNewsMedia.com  is leading provider of third party publishing &  news dissemination services. If you would like more information regarding our news coverage solutions, please visit  http://www.financialnewsmedia.com  for more details. Get an edge on the market with our  Premium News Alerts  that are FREE for a limited time at  http://www.financialnewsmedia.com/. Follow us on Facebook:  http://www.facebook.com/financialnewsmedia  and Twitter:  http://twitter.com/FNMgroup.

FN Media Group LLC (FNMG) is a third party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNMG is NOT affiliated in any manner with any company mentioned herein. FNMG and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNMG's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNMG is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNMG has been compensated one thousand dollars for Amarantus Bioscience Holdings, Inc news coverage by the Company.   FNMG HOLDS NO SHARES OF Amarantus Bioscience Holdings, Inc.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNMG undertakes no obligation to update such statements.

Contact Information:
Company: FN Media Group, LLC
Contact email: [email protected]
U.S. Phone: +1(954)345-0611
URL: http://www.financialnewsmedia.com

SOURCE FN Media Group LLC