DMD patient granted access to PTC124

A judge for the U.S. District Court for the District of New Jersey ruled that PTC Therapeutics (South Plainfield, N.J.) should give a 16-year-old Duchenne muscular dystrophy (DMD) patient access to the company's PTC124. Judge William Martini ruled that Jacob Gunvalson meets the criteria of the treatment use of an investigational new drug section of the Food, Drug, and Cosmetic Act, which outlines the criteria under which "desperately ill" patients can receive access to experimental drugs.

The judge ordered that Gunvalson receive PTC124 through a single-patient IND study conducted by his pediatrician or as a protocol exception to the ongoing extension of a Phase IIa trial. The small molecule that facilitates complete translation of proteins containing nonsense mutations also is in a Phase IIb trial for DMD and Phase IIa testing for cystic fibrosis (CF). ...