Subject: Genentech's AIDS Drug Gets FDA's 'Orphan' Status 
Date: Published: 4/5/89 (62 lines)
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

    Technology:  
    Genentech's AIDS
    Drug Gets FDA's
    'Orphan' Status
    ----   
    By Marilyn Chase
    Staff Reporter of The Wall Street Journal

    SOUTH SAN FRANCISCO -- Genentech Inc. said its
 experimental AIDS drug CD4 was granted orphan drug status by
 the U. S. Food and Drug Administration.
    Such designation could yield marketing advantages if and
 when the drug proves useful in treating the disease.
    CD4 -- a synthetic protein that mimics the site used by
 the AIDS virus to penetrate and infect human cells -- hasn't
 yet been proven effective against the disease.  Genentech so
 far has tested it in 45 patients with only minimal side
 effects.  More than half a dozen other groups are trying to
 develop the agent, including companies such as Biogen Inc. of
 Cambridge, Mass., and SmithKline Beckman Corp. of
 Philadelphia -- which also have launched CD4 in human
 clinical trials.
    U. S. law accords orphan drug status to medicines that are
 aimed at a disease with a patient population of 200,000 or
 less.  Currently, the U. S. count of acquired immune deficiency
 syndrome cases exceeds 88,000, including more than 50,000
 deaths.
    Although Genentech is believed first to get orphan drug
 status for its CD4, more than one company can receive the
 orphan drug designation while the product is still under
 development.  However, once a company gets FDA approval on its
 product, the orphan drug tag carries benefits including seven
 years' marketing exclusivity and certain tax breaks.
    Genentech stock, battered by recent downbeat news on its
 heart drug TPA, closed yesterday at $18.50, up $1.375, in
 composite trading on the New York Stock Exchange.
    Genentech's recent laboratory coup, combining CD4 with
 Immunoglobulin-G to make a custom antibody, isn't included in
 the FDA's orphan drug tag.  That enhanced version will be the
 subject of a separate application, when the company launches
 clinical trials, perhaps later this year.
    Stephen Sherwin, Genentech's vice president, clinical
 research, said it is too soon to speculate about whether CD4
 in any form will be useful against AIDS. However, Dr. Sherwin
 said:  "We're pleased with the progress {of clinical trials.}
 We haven't seen any serious toxicity."  Nor have patients
 developed antibodies against the drug so far, as some
 scientists had feared, he added.
    In Cambridge, Biogen dismissed the importance of the FDA's
 move.  "It's a non-issue," said Jack Catterall, Biogen's vice
 president and treasurer.  "You don't get any points for this."
 He added that Biogen hasn't sought orphan drug status for its
 CD4 product, but hasn't ruled out doing so.

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