This is why it is a hard stock to trade...
from yahoo...
15 Reasons to Celebrate Orphan Drug Designation
Imetelstat is now approved for orphan drug designation (ODD) vesting it with opportunity for significant development and revenue advantages that provide competitive edge: increased market share, lower development costs, higher pricing, faster returns, and focus on niche markets.
1. ODD now allows use of Imetelstat in primary orphan drug designation (MF) and in any other indication; and is gateway to MF (and other) registry studies.
2. ODD allows for a Fast Track procedure to evaluate registration files.
3. ODD reduces waiting period and provides priority FDA review of new drug applications (6 months instead of 10 months).
4. ODD takes, on average, 30% less time to market (3.9 years vs 5.4 years).
5. ODD allows smaller studies with no placebo required.
6. ODD provides FDA technical assistance during the elaboration of application file and in design of protocols including written recommendations in clinical and preclinical studies
7. ODD has higher rates of approval 82% compared to non-orphan at 35%. Of the 36 new drugs launched in 2013, 17 were ODD.
8. ODD provides availability to patients before market approval when disease is life-threatening, no equivalent treatment is available, and drug in clinical trials and active phase of marketing approval.
9. ODD provides 7 year exclusivity (monopoly) after market approval.
10. ODD provides up to a 50 percent research-and-development tax credit on US trials.
11. ODD waives drug approval application fees and annual FDA fees.
12. ODD application required disclosure of risk/benefit data and
13. ODD allows for development of personalized and predictive cancer treatment that target patient populations with a specific cancer causing mutation.
14. Almost 1/3 of orphan drugs earn more than 1 billion in annual sales. Category has more than 50 billion annually in world wide sales and rising 20% a year past several years.
15. ODD has produced "rolling blockbusters" - drugs initially may be approved for one indication in a small population, but later acquire additional uses after longitudinal studies and real world observations (registry studies).
All investors, traders, and players should do OWN due diligence.
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Also great SA article on subject by (Napodano, 2013). Some highlights:
1. ODD drug in phase I has same liklihood of approval as phase III drug (63%).
2. Stock prices post ODD can go as high as 30% after announcement and 4-5% next week.
3. Applications for ODD are not always made public at time of application.
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Arrowhead Research just got Orphan drug status recently and on their PR . it was mentioned about what orphan drug status can mean for a company .......The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan drug designation provides incentives for sponsors to develop products for rare diseases. These incentives include increased engagement with FDA on drug development activities, exemption from all future product-specific regulatory fees, the opportunity to apply for R&D funding, tax credits, an increased chance of priority review, and 7 years of orphan exclusivity at time of New Drug Application (NDA) approval.
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