South Plainfield, NJ – PTC Therapeutics, Inc. plans to cut its workforce by 18 percent in order to manage operating expenses following receipt from the FDA of a Refuse to File letter for its Translarna (ataluren) drug application.
In the letter received last month, the FDA stated that the application was not sufficiently complete to permit a substantive review. Despite the setback, company executives expressed the intention to “work with the FDA” in a subsequent attempt to bring Translarna to the U.S.
“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,” Stuart W. Peltz, Ph.D., CEO said in a statement.
Completion of the cutback, which primarily will target its workers in the U.S., is expected by the end of June at an estimated $2.5 million in employee severance and benefit costs.
Meanwhile, efforts toward development and commercialization of Translarna outside of the U.S. will continue, according to the company.
Translarna (ataluren) is an oral protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein.
The drug is conditionally approved in Europe for treatment in ambulatory patients who are five years of age and older. The European Medicines Agency had granted the conditional approval after a key committee decided that its “benefits are greater than its risks.”
Primarily affecting males, Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein, critical to the structural stability of skeletal, diaphragm, and heart muscles.
Patients lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and 20s, according to PTC, which estimated that nonsense mutations account for approximately 13 percent of DMD cases, or approximately 2,000 patients in the United States.
(Sources: PTC Therapeutics, Inc., European Medicines Agency)