PTC Therapeutics is reducing its workforce by approximately 18%, which will primarily affect employees and contractors in the U.S. This reduction is part of PTC's program intended to optimally manage operating expenses following its recent setback related to the Refuse to File letter received from the FDA for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). 

PTC remains focused on the global development of Translarna and commercialization outside of the U.S. for nmDMD. In parallel, PTC intends to work with the FDA to determine the best path forward to bring Translarna to patients in the U.S.

"I would like to express my sincere appreciation to those employees affected by today's announcement," said Stuart W. Peltz, Ph.D., chief executive officer, PTC Therapeutics. "While it is very difficult to part with this talented group of colleagues, it was a necessary step to better align our resources and enable us to continue our mission of bringing treatments to patients suffering from rare and neglected disorders."

PTC plans to complete this workforce reduction by June 30, 2016 and expects to incur related employee severance and benefit costs of approximately $2.5 million.    

Translarna, discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area (EEA) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.

Translarna is an investigational new drug in the U.S. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.