Suit challenges biotech firm over last-chance drug

In another case challenging the limits of "compassionate use" for drugs not approved by the FDA, the mother of a gravely-ill teenager has filed a lawsuit demanding access to an experimental drug. The mother, Cheri Gunvalson, argues that the company had promised her that her son would get the drug, but the company, PTC Therapeutics, denies that it made such promises and says the drug isn't ready. Gunvalson, whose 16-year-old son has Duchenne muscular dystrophy, had previously worked on behalf of PTC, which makes a drug she believes offers the best hope for treating her son's version of the condition. However, since the teen is no longer walking--he's confined to a wheelchair--the company says he's not eligible for the clinical trial underway of the experimental compound, PTC124. And they won't give the compound to boys not involved in trials. Gunvalson, for her part, says PTC124 is his best chance to slow or even reverse the effects of Duchenne's.

If PTC did reverse its decision, it wouldn't be a unique decision. Of late there's been a movement toward allowing seriously-ill patients to use experimental drugs, under a process known as compassionate use, which enrolls them in single-patient studies or clinical trials. In her suit, Gunvalson asks for just such an exception, arguing that there are no significant risks and that the compound could possibly save his life.

To learn more about this case:
- read this article in The New York Times

PLUS: The controversy over this case hasn't slowed PTC down. In fact, it just finalized a $437 million licensing deal with biotech Genzyme for its lead development program. FierceBiotech