Rivulet Media Inc (fka RIVU)

[jjr04001]

Hi Agraff....Pharmacist here.

This treatment is for Aplastic anemia, a disease which can be described currently as lacking a profit motive because of how rare the disease and/or how expensive it is to treat. If you look at some of the antiviral treatments for HIV/AIDS (Glaxo's Lexiva - Fosemprenavir) or Genzyme's Fabrazyme (Agalsidase beta), they did not go through 8 years of clinical trials.

Orphan drugs have increased in popularity with the FDA. An orphan drug, historically, has an 83% chance of approval to phase II clinical trials, as compared to 33% for non-orphan drugs.

Aplastic anemia, if left untreated, WILL kill in 7 months or less. Deaths from this disease are generally quick. "Orphan drug status" is the key here. These drugs are an exception to normal regulatory pre-marketing practices. Treatments for "Orphan diseases" are a guaranteed profit as the patient is a chronic patient, always requiring therapy, payers usually had no problem covering these treatments because of how rare the diseases are, and there are incentives given federally to reduce sales cost and what have you...

If you go on the website for national institute of health (NIH) and type in hemaxellerate, you will find a mechanism of the treatment. You should compare this treatment to the current modes of treatment for Aplastic Anemia (Immunosuppression, bone marrow transplant, ATG/ALG, steroids) and compare the side effects/ efficacy / safety and survival rates. Using my professional judgement (PharmD.) this treatment has a lot to offer to those who suffer from this debilitating disease (often children)

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3543295/

Scroll down and you will find "Practical use of endothelial cells for stimulation of hematopoietic recovery: hemaxellerate autologous adipose derived cells as a source of endothelial cells"

There are a lot of sources to play with here. Orphan drugs are almost a foot in the market, so I don't think we'll have to wait 5 years to see a hike in pps. Hope this calms some nerves.