PTC Therapeutics has been on a roll over the last few months. But the biotech hit a loud bump when the New York Times featured the story of one mother's legal fight to gain access to PTC124 for her son, who suffers from Duchenne muscular dystrophy. PTC just inked a $437 million deal with Genzyme for PTC124, which has demonstrated promising results for treating a segment of the Duchenne MD population. Cheri Gunvalson says that the experimental therapy is her son's last and best hope. But PTC isn't providing the therapy to anyone who isn't in the clinical trial and the boy, who can no longer walk, isn't eligible. Gunvalson is asking for access as part of a compassionate use approach that a number of drug developers have in place, but PTC CEO Stuart Peltz has said that there's been too little experience with the mid-stage therapy to provide it outside of a trial.
The Times notes that for many people an experimental drug is their only hope. But the Abigail Alliance for Better Access to Developmental Drugs took their case for broader access to experimental cancer therapies to court last year--and lost.
- read the article in the New York Times