Reversal in Gunvalson v PTC Therapeutics
The Drug and Device Law Blog posteth, and I posteth on the post.
The decision that a teenage boy with muscular dystrophy,* Jacob Gunvalson, should have access to a proprietary, experimental drug has been reversed by the US Appeals Court for the Third District in Philadelphia. The original decision, made by a NJ federal judge, had ordered PTC Therapeutics, the owner of the experimental agent PTC124, to submit a single-patient investigational new drug application (IND) to the FDA on behalf of Jacob, and, if the FDA denied the IND, to ask for a protocol exception so that he could enroll in a phase 2a extension trial of PTC124. The decision had rested on alleged promises made by PTC employees to Jacob's mother about drug access.
(For a lot of important blog-type background on this case, Gunvalson v PTC Therapeutics, go here, here, here, and here. Also browse through the DDL Blog, the WSJ Health Blog, and Pharmalot.)
The appeals court determined that the Gunvalsons had failed to demonstrate a reasonable probability of success of their claim on the merits of a something called a promissory estoppel. In this case, a promissory estoppel would have prevented PTC Therapeutics from withdrawing its alleged promise to the Gunvalsons to access PTC124, if the Gunvalsons has reasonably relied on this promise and had acted on it to their detriment (meaning that Jacob had foregone enrollment in the phase 2a trial voluntarily).
The appeals court noted that the claim of a promissory estoppel requires a "clear and definite promise" and "a reasonable reliance on the promise." However, the appeals court found that the Gunvalsons failed to adequately plead these 2 requirements. With respect to the second requirement, the NJ court failed to recognize that Jacob did not enroll in the phase 2a trial (which was required for enrollment in the extension phase) because he was ineligible, not because he was promised access to PTC124 by some other means.
Gunvalson v PTC Therapeutics is now remanded to the NJ court for further claims. According to an AP story, the Gunvalsons and their attorney are assessing their legal options.
* Whether Jacob Gunvalson has Becker or Duchenne muscular dystrophy remains unclear.
