Rare Disease Business Not Such An Easy Road - Just Ask Pfizer

ByPropThink:Thursday morning, DARA Biosciences (DARA) announced it has submitted an application to the U.S. FDA requesting Orphan Drug status for KRN5500 for the treatment of chronic chemotherapy-induced peripheral neuropathy (CCIPN).

ByBeacon VP Investments: By April 30, 2013, the United States Food and Drug Administration (FDA) will decide whether to approve Raptor Pharmaceutical's (RPTP) drug Procysbi for the treatment of nephropathic cystinosis.

By Brian L. Wilson:This year, one of many trends in the world of biotech investing is the increasing popularity of orphan drugs on Wall Street.An orphan drug is a special designation that is given to drugs that are intended to treat very rare diseases (in the United States this means less than 200,000). In the past we've seen drug companies avoid the targeting of certain diseases because of the very small number of patients that would be providing sales revenue, but things have changed.

NEW DELHI (AP) — India's Supreme Court on Monday rejected drug maker Novartis AG's attempt to patent an updated version of a cancer drug in a landmark decision that health activists say ensures poor patients around the world will get continued access to cheap versions of lifesaving medicines.

BySharon di Stefano:Another clinical score for Pluristem Therapeutics (NASDAQ:PSTI) with the FDA's recent grant of Orphan Drug status for PLX cells in aplastic anemia (AA), a rare and hard to treat blood disorder affecting under 200,000 patients in the US.

By Jason Boyce:NPS Pharmaceuticals (NPSP) is an FDA run-up candidate and possibly one to tuck away for a long-term hold. Quick facts

By Jessica King Holden:Despite rare diseases in some cases only affecting hundreds to thousands worldwide, the market for drug treatments has been speculated to hold a potential $1 trillion a year price tag. Orphan drug status benefits and unique intellectual property are certainly attractive draws to this market, in addition to direct revenue increase.

Jason Chew submits: Pegylated interferon plus ribavirin is the current standard of care for HCV treatment. However, it is a 48-week treatment and is only effective in about 50% of patients.