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Biotech / Medical : Millennium Pharmaceuticals, Inc. (MLNM) -- Ignore unavailable to you. Want to Upgrade?

To: Biomaven who wrote (1974)	12/17/2003 9:33:42 AM
From: Icebrg	Read Replies (1) \| Respond to of 3044

That seems clear enough. I had my information from an analyst's comments on Trisenox, which is in a similar situation with many potential areas of use. And there it was stated three years. Unfortunately the file was lost in a disk crash, so I am not able to retrieve it. Possibly there was a mix-up with a "three-year New Use/New Clinical Studies Exclusivity" which also seems to exist, but which is not related to orphan drug status exclusivity. burnsdoane.com Erik

To: Biomaven who wrote (1974)	12/17/2003 2:25:16 PM
From: software salesperson	Read Replies (4) \| Respond to of 3044

peter, miljenko, peter, Intrigued by the orphan drug issue, I emailed someone at the fda late last night before your last post on the topic. Here is the result: sales: I reviewed the orphan drug law, yet cannot ascertain the answer to a question. If a drug is designated to be an orphan drug for one rare disease indication and thereby obtains 7 year marketing exclusivity, can it then receive an additional 7 year marketing exclusivity for a second rare disease indication? Or once it receives orphan drug status , does it receive a total of 7 years of marketing exclusivity for ANY and ALL rare disease indications within that 7 years and, therefore, cannot apply for subsequent orphan drug status designations for the same drug? Thanks for any light that you or your colleagues can shed on this issue. Any relevant links would be appreciated. Fda employee: Orphan Exclusivity is specific to a drug product (active moiety) and an indication. Therefore, a drug product could theoretically have Orphan Exclusivity for more than one orphan indication. A recent example of this would be for the product Gleevec. Gleevec was approved for chronic myeloid leukemia (CML) May 10, 2001, and its Orphan Exclusivity would protect this indication until May 10, 2008. On February 1, 2002, Gleevec was approved for gastrointestinal stromal tumors (GIST) and this indication is protected by Orphan Exclusivity until February 1, 2009. This would mean that after May 10, 2008, a generic product could be marketed only for the CML indication. After May 10, 2008, the GIST indication could be added to the label of the generic product. sales: Thank you very much for your response. A followup question please. Thalidomide has orphan drug exclusivity for the treatment of erythema nodosum leprosum (ENL) in leprosy. Velcade has been approved for the treatment of multiple myeloma patients who have received at least two prior therapies and have demonstrated disease progression on the last therapy Could Thalidomide apply for and potentially be approved for orphan drug exclusivity for MM with Velcade already on the market? If so, could Velcade still be marketed? Fda employee: Yes, because they are different active moieties. sales: (1) Was that "yes" to both those questions? And, hopefully, one final question: 2) Since we began emailing, I've learned that Thalidomide has an orphan drug designation for MM as well. As I only see one designation date on your website, I assume that all the designations for T began on the same date. Is there a time limit during which the designation has to be either (i)converted into an approval or (ii) else it will expire? Fda employee: (1) yes (2’) Your assumption is incorrect. Orphan status is NOT granted automatically. Each sponsor has to submit it own request and each request is reviewed based on its own merits. Thalidomide was designated as a treatment for multiple myeloma October 14, 1998. Velcade (borteozmib) was designated for multiple myeloma January 15, 2003. I apologize that the designation date for thalidomide does not appear on our website, apparently there is a glitch that needs to be addressed by the FDA webmaster. (2’’) There is no time limit. Please remember that the designation process and the approval process are separate and distinct. By no means does an orphan designation imply safety or efficacy for a product. A sponsor must conduct clinical trials to show safety and efficacy and then data is submitted to the FDA for review in the form of a New Drug Application (NDA). Clinical trials are conducted under an IND. This office is not involved with the approval process. The IND/NDA process is the responsibility of the Center for Drug Evaluation and Research (CDER) Orphan status basically provides financial incentives to encourage a sponsor to develop its product for a rare disease. End of dialogue with fda. One question that I thought I didn’t need to ask was whether 2 drugs could be granted orphan drug status for the same indication. I thought that the answer was given in the tktx/genz battle: Within weeks of each other in the summer of 2000, Genzyme Corp. and Transkaryotic Therapies Inc. submitted applications for nearly identical drugs to treat Fabry disease. The unprecedented situation raised a number of difficult scientific and policy questions. Under the so-called orphan drug act, designed to encourage the development of drugs for rare diseases, the first company with a breakthrough treatment wins seven years of market exclusivity - a provision that seems to turn the competition into a winner-takes-all contest and has sparked a firestorm of speculation about what FDA would do. Analysts have scrutinized every development for signs, dividing into three camps: those who believe only Genzyme's drug, Fabrazyme, will be approved; those who think TKT's Replagal will be the sole drug to reach the market; and those who predict regulators will find a loophole that allows them to approve both. ''Orphan drug laws dictate that only one product can get approved, and the one that will be approved is the one that is clinically superior,'' said Ron Renaud, a biotech analyst at Bear Stearns who falls in the Fabrazyme camp. ''Someone has to decide which drug is better, and I think that's what FDA is doing.'' Buoyed by this communication success with the fda, I have placed a call to sepr mgmt. to offer my services on estorra at the rate of $ 25,000/day. miljenko, (i) why do you believe that T will be a $ 400 m drug? Growth in mm? Solid tumors? (ii) How do you think Velcade will impact T revenues in mm? Thanks. sales