Replies to post #48335 on Biotech Values

[DewDiligence]

FDA Revokes Orphan Status on ALTU-135

[Can anyone recall another case like this?]

http://biz.yahoo.com/e/070703/altu8-k.html

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3-Jul-2007

On June 28, 2007, Altus Pharmaceuticals Inc. (the "Company") was notified by the Office of Orphan Products Development of the Food and Drug Administration ("FDA") that the orphan drug designation granted in 2002 to ALTU-135 for the treatment of pancreatic insufficiency was being revoked. The FDA based its decision on a finding that if one includes all patients with HIV/AIDS who suffer from fat malabsorbtion in this indication, the patient population in the United States appears to exceed 200,000 persons and is thus ineligible for orphan drug designation.

We believe that only a subset of patients with HIV/AIDs have fat malabsorbtion due to pancreatic insufficiency and that our original filing was intended to be within the 200,000 person limit for this disease condition; however, the FDA concluded otherwise.

The principal anticipated advantage to the Company of an orphan drug designation is the availability of tax credits and the abatement of new drug application filing fees. In addition, the holder of the first new drug application approval also receives marketing exclusivity for a period of seven years over other products that contain the same drug or active ingredient. We are not aware of other products in development that are the same drug as ALTU-135 for orphan drug purposes. Given these facts and circumstances, the Company may consult with the Office of Orphan Products Development. If we conclude that re-filing with a more precisely defined indication has merit, we have the right to submit an application on or before the filing of a new drug application. We may also conclude that the advantages of continuing to seek orphan drug designation may not be warranted.
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[ghmm]

rfj,
Your prediction is looking pretty good, unfortunately I had purchased some before your post. I've added more. Judging by the timing of their call and when the IND was filed for 237 (hyperoxaluria / kidney stones) and the fact that we have not had a negative PR they should announce it being accepted and hopefully dosing patients soon!

I haven't been too thrilled with their lack of progress with 238 (growth hormone) and their business development (what good is it to tout crystallization of 70+ proteins and not have the ability to form strategic collaborations). I'd think Biomarin would love to get their hands on 236 (PKU). I keep hoping Sidney wants to retire (he sure is selling his shares as quick as he gets them) and they can convince Michael Astrue to give up the government job (Bush's term is ending soon anyway) and become CEO. Its interesting that David Pendergast is on the board.