Latest Orphan Drug Act Stories

-- Clinical Trial in Patients with Huntington's Disease Slated to Begin Next Quarter -- SEATTLE, May 23, 2013 /PRNewswire/ -- Omeros Corporation (NASDAQ: OMER) today announced that its Investigational New Drug Application (IND) to evaluate OMS824 in Huntington's disease has been cleared by the U.S. Food and Drug Administration (FDA). OMS824 selectively inhibits phosphodiesterase 10 (PDE10), an enzyme expressed in areas of the brain linked to a wide range of diseases that affect...

30th Anniversary Gala to honor rare disease heroes and support the continuation of NORD's critical work on behalf of the rare disease community WASHINGTON, May 10, 2013 /PRNewswire-USNewswire/ -- The 30(th) Anniversary Gala of the National Organization for Rare Disorders (NORD), to be held Tuesday, May 14, 2013, will honor rare disease heroes, celebrate 30 years of progress through the Orphan Drug Act (ODA) and NORD, and promote a promising future for the rare disease community....

Pulmonary hypertension, a rare and serious lung disease once considered rapidly fatal, is now treatable with nine drugs all developed in the past 20 years. The roles of policy, science, medicine, business and patient advocacy in producing this progress show the impact possible in the three decades since the Orphan Drug Act passed. Silver Spring, MD (PRWEB) May 08, 2013 Pulmonary hypertension (PH), a rare and life-threatening lung disease, has gone from rapidly fatal to treatable with nine...

Emmaus in Process of Completing Phase 3 Study For First Widely Available Treatment for Sickle Cell Disease TORRANCE, Calif., May 9, 2013 /PRNewswire/ -- Timothy Cote, M.D., MPH, former director of the Office of Orphan Product Development of the U.S. Food and Drug Administration, has joined Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine company, in an advisory capacity, the company announced today. Dr. Cote will advise Emmaus on the regulatory pathway for...

-- Orphan Drug Designation Could Provide Financial Incentives and Faster Regulatory Review -- SEATTLE, April 17, 2013 /PRNewswire/ -- Omeros Corporation (NASDAQ: OMER) today announced that it has filed an Application for Orphan Drug Designation with the U.S. Food and Drug Administration (FDA) for OMS721, the lead human monoclonal antibody in Omeros' mannan-binding lectin-associated serine protease-2 (MASP?2) program, for use in the treatment of atypical hemolytic uremic syndrome...

On 30(th) Anniversary of Orphan Drug Act and NORD, Rare Disease Patient Advocates Fear That Budget Cuts for NIH and FDA Will Slow Momentum WASHINGTON, Feb. 26, 2013 /PRNewswire-USNewswire/ -- On Thursday (Feb. 28), millions of people around the world will be observing Rare Disease Day and this annual observance has special significance for Americans this year for two reasons. The first is that 2013 is the 30(th) anniversary of the Orphan Drug Act - landmark legislation made...

WASHINGTON, Jan. 28, 2013 /PRNewswire/ -- Today ten families of patients with a rare disease called cystinosis and three foundations supporting cystinosis research tell their stories to policymakers on Capitol Hill in an effort to educate lawmakers about rare diseases like cystinosis and how upcoming federal spending decisions could impact the availability of medicines for rare diseases. Cystinosis is a metabolic lysosomal storage disease that is diagnosed in infancy and often...

PALO ALTO, Calif., Jan. 11, 2013 /PRNewswire/ -- Telik, Inc. (Nasdaq: TELK) was notified today that its product candidate, ezatiostat HCL (Telintra), has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of myelodysplastic syndrome (MDS). Orphan designation grants potential US market exclusivity to a drug for the treatment of a specified condition for a period of seven years following FDA marketing approval. Additional potential...

Rare Disease Patient Advocates Celebrate 30(th) Anniversary Today WASHINGTON, Jan. 4, 2013 /PRNewswire-USNewswire/ -- Thirty years ago today, President Ronald Reagan signed the Orphan Drug Act into law to encourage the development of treatments for the millions of Americans with rare diseases. (Logo: http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b) The new law represented a triumph of patient advocates working with government partners, the media, and other supporters to...

WASHINGTON, Dec. 25, 2012 /PRNewswire-USNewswire/ -- Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act. By portraying on his Quincy, M.E. television show the plight of people with rare diseases with no treatment, Mr. Klugman focused a spotlight on the need for incentives to encourage the development of therapies for small patient populations. He did this in partnership with patients and patient advocates who...