An inexpensive "orphan drug" used to treat sleep disorders appears to be a potent inhibitor of cancer cells, according to a new study led by scientists at Fred Hutchinson Cancer Research Center. Their novel approach, using groundbreaking technology that allows rapid analysis of the genome, has broad implications for the ...

Medgenics, Inc., the developer of a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced that it has filed for Orphan Drug Designation with the U.S. Food and Drug Administration (FDA) for INFRADURE for the treatment of hepatitis D.

According to the Ministry of Health in France, there are almost 7,000 rare diseases, with 25 million sufferers in Europe alone. A rare disease is an illness that affects fewer than on in 2,000 people, and there is no cure for most, as the only treatments currently available only improves ...

Lexicon Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted its request for Orphan Drug designation for telotristat etiprate (LX1032) for the treatment of carcinoid syndrome.

Arno Therapeutics, Inc. announced today that its investigational compound, AR-42, has been granted orphan-drug designation by the U.S. Food and Drug Administration for the treatment of meningioma and schwannoma of the central nervous system.

Because rare diseases affect only a tiny group of people, pursuing the development of treatments has traditionally not been attractive for pharma companies. However, there are currently lucrative benefits in place set by the regulatory bodies that reward R&D efforts for orphan drugs by ensuring less intense competition, faster and ...

How can insurers justify spending hundreds of thousands of dollars per patient per year on "orphan drugs" - extremely expensive medications for rare conditions that are mostly chronic and life-threatening - when this money could provide greater overall health benefit if spread out among many other patients? Those spending decisions ...

How can insurers justify spending hundreds of thousands of dollars per patient per year on "orphan drugs" - extremely expensive medications for rare conditions that are mostly chronic and life-threatening -- when this money could provide greater overall health benefit if spread out among many other patients?

How can insurers justify spending hundreds of thousands of dollars per patient per year on "orphan drugs" – extremely expensive medications for rare conditions that are mostly chronic and life-threatening -- when this money could provide greater overall health benefit if spread out among many other patients? Those spending decisions ...

(The Hastings Center) Can insurers justify spending hundreds of thousands of dollars per patient per year on "orphan drugs" - extremely expensive medications for rare conditions that are mostly chronic and life-threatening -- when this money could provide greater overall health benefits to many other patients? Those spending decisions ...

Orphan drug development has become more feasible than in previous years but we've barely scratched the surface: only 367 approved orphan drugs out of 6,800 rare diseases. We need to keep the momentum going. Come April 10-13, over 300 key decision makers from orphan drug manufacturers, patient groups, payers ...

Ceptaris Therapeutics, Inc., a privately held specialty pharmaceutical company, announced today that the European Medicines Agency (EMA) has granted Orphan Drug Designation to Ceptaris' investigational drug, chlormethine gel, for the treatment of Cutaneous T-Cell Lymphoma.