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Latest Orphan Drug Act Stories

2014-09-02 08:29:52

SAN DIEGO, Sept. 2, 2014 /PRNewswire/ -- Arena Pharmaceuticals, Inc. (NASDAQ: ARNA) today announced that the US Food and Drug Administration (FDA) has granted orphan drug status to APD811 for the treatment of pulmonary arterial hypertension. "The FDA Office of Orphan Products Development (OOPD) evaluates scientific and clinical data submissions from sponsors to identify and designate drug candidates that could potentially treat rare diseases to help advance the evaluation and...

2014-06-09 00:21:32

EPI-743 in phase 2B development for Leigh syndrome MOUNTAIN VIEW, Calif., June 9, 2014 /PRNewswire/ -- Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome. Vatiquinone is the International Nonproprietary Name (INN) for Edison's EPI-743. The INN is a unique international name issued by the World Health Organization, which is used to identify the active pharmacological ingredient in a drug. This is...

2014-05-15 12:29:33

LONDON, May 15, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report:Global Markets for Orphan Drugshttp://www.reportbuyer.com/pharma_healthcare/drug_discovery/global_markets_orphan_drugs.htmlINTRODUCTIONSTUDY OBJECTIVESThe orphan drug market is one of the fastest-growing segments in the pharmaceutical and biotechnology industry. BCC's goal in conducting this study is to provide an overview of the current and future global market for orphan drugs. The key...

2014-05-14 23:09:18

New Kauffman Foundation paper highlights solutions to speed patients’ access to drugs with therapeutic value for more than one disease. Kansas City, MO (PRWEB) May 14, 2014 For patients with rare medical conditions, the typical 12 to 15 years it takes to bring a new drug to market may be too long to wait. Repurposed drugs – those already approved for a specific condition that can be used to treat another – offer a solution: Because their safety profile is already established, they...

2014-04-29 20:23:44

LONDON, April 29, 2014 /PRNewswire/ -- Reportbuyer.com has added a new market research report:The World Market for Orphan Drugshttp://www.reportbuyer.com/pharma_healthcare/therapeutic/world_market_orphan_drugs_1.htmlIn recent years, there has been a substantial increase in the number of drugs available to treat rare ("orphan") diseases. This comprehensive report, The World Market for Orphan Drugs, looks at this trend and provides estimates for the market opportunity in the U.S.,...

2014-02-27 23:03:20

Resolution Promotes Awareness of Rare Diseases Sacramento, CA (PRWEB) February 27, 2014 Today at the state Capitol, Assemblymember Kevin Mullin (D-South San Francisco), in partnership with the California Healthcare Institute (CHI), presented House Resolution 32 recognizing Feb. 28 as Rare Disease Day in California. CHI is a nonprofit, public policy research organization, representing leading California academic institutions, biotechnology, medical device, diagnostics and pharmaceutical...

2014-02-25 08:32:12

EASTBOURNE, England, February 25, 2014 /PRNewswire/ -- Daval International, the emerging life sciences company focused on the development of novel treatments for serious unmet medical needs, today announced receipt of Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), Office of Orphan Products Development, for AIMSPRO(R) for the treatment of systemic sclerosis (scleroderma). "The granting of Daval's request for Orphan Drug Designation represents a...

2014-02-14 23:21:13

MarketReportsOnline.com adds "US Orphan Drug Market Outlook 2018" report to its research store. Dallas, Texas (PRWEB) February 14, 2014 In the largest market for orphan drugs, USA, there was a shortage of adequate therapies for treating many rare diseases. These therapies were not developed as companies did not expect these drugs to be highly profitable. Hence there was a lack of interest and thus investment on the part of pharma companies in the USA. Therefore, the FDA...

2014-02-07 08:22:40

NEW DELHI, February 7, 2014 /PRNewswire/ -- KuicK Research releases "Global Orphan Drug Market Outlook 2018" research report offering comprehensive insight on recent trends, regulatory development related to US orphan drug market, Europe orphan drug market & Asia orphan drug market. Report also gives insight on more than 600 orphan drugs in clinical pipeline and 231 marketed orphan drugs. Global Orphan Drug Market Report Findings: - Orphan...

2014-02-04 23:00:35

EPI-743 phase 2b Friedreich's ataxia trial fully enrolled. Downingtown, PA (PRWEB) February 04, 2014 Edison Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to vatiquinone (EPI-743) for the treatment of Friedreich’s ataxia (http://www.prnewswire.com/news-releases/fda-grants-edison-pharmaceuticals-epi-743-orphan-status-for-friedreichs-ataxia-243439351.html). Orphan status brings additional momentum and acceleration to the...


Word of the Day
omphalos
  • The navel or umbilicus.
  • In Greek archaeology: A central boss, as on a shield, a bowl, etc.
  • A sacred stone in the temple of Apollo at Delphi, believed by the Greeks to mark the 'navel' or exact center-point of the earth.
'Omphalos' comes from the ancient Greek.
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