At Seeking Alpha, contributor Chemistfrog wrote an article explaining the background of the "Orphan Drug" designation, and listing some "some biotechs for investor consideration that have or are candidates for Orphan Drug designation therapies."
About Medgenics, he writes,
"Medgenics, Inc. (MDGN) chart has been on a tear since December 29th's low of $2.49 and is now trading above $6.70 per share, up over 170%. The chart remains bullish and shows no signs of abatement except for some possible resistance at $7.00. The company filed for Orphan Drug designation on April 23rd this year and expects to hear back from the FDA by the end of June for its INFRADURE treatment for Hepatitis D, a serious liver disease caused by the hepatitis D RNA virus. Although rare in the United States, the disease affects about 15 million people worldwide. INFRADURE is based on the company's proprietary Biopump platform which utilizes the patients' own tissue samples to manufacture and then re-implant back into their bodies the modified cells (biological pump) to produce their own human protein therapy for a sustained period of time."
"[This] technology has a broad range of possibilities with treatments possible for anemia, hepatitis C &D, multiple sclerosis, arthritis, obesity, chronic pain, cancer recovery, and more. INFRADURE is the company's lead product and is in Phase I/II for Hepatitis C utilizing the same technology and protein therapy, interferon-alpha. Proof-of-concept trials in mice and humans created therapeutic levels of interferon-alpha and other protein therapies in blood serum. If safety and efficacy data are promising early, the chart growth for this emerging biotech could continue on its uptrend; the current market capitalization of $66 million would be dramatically undervalued if interim data proves to be promising."
Read more at Seeking Alpha.