PTC Therapeutics said today it will eliminate approximately 18% of its workforce, or about 74 jobs, primarily positions held by employees and contractors in the U.S.

PTC said the layoffs were part of a cost-cutting program that follows the FDA’s issuing the company a Refuse to File letter in response to its New Drug Application (NDA) for Translarna™ (ataluren), the company’s candidate for nonsense mutation Duchenne muscular dystrophy (nmDMD).

“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 CEO Stuart W. Peltz, Ph.D., said of the job cuts in a statement.

PTC plans to complete its layoffs by June 30, and said it expected to incur approximately $2.5 million in related employee severance and benefit costs.

PTC spokeswoman Justine O'Malley told GEN the company now has about 300 employees, down from the 320 employees reported as of December 31, 2015, on the company's Form 10-K annual report for last year —including 309 full-time staffers and 37 full-time consultants and contractors.

In the Refuse to File letter, received by the company on February 22 and disclosed a week later, the FDA stated that the NDA for Translarna was not sufficiently complete to permit a substantive review. PTC said the letter conveyed the FDA’s view that both the Phase III Altaluren Confirmatory Trial (ACT) in DMD and an earlier Phase IIb study were negative and did not provide substantial evidence of effectiveness.

In ACT DMD, PTC reported a statistically insignificant 15-meter benefit in the overall study population in the primary endpoint of change from baseline in the 6-min walk test (6MWT), but a significant 47-meter benefit within a subpopulation of patients who could walk between 300–400 meters at baseline.

The FDA also characterized certain of the company's adjustments to ACT DMD as post hoc and therefore not effective. In addition, according to PTC, the FDA noted that the NDA did not contain adequate information regarding the abuse potential of Translarna, a requirement for new molecules that cross the blood–brain barrier.

As it said last month, PTC restated today that it intended to work with the FDA to determine the best path forward for advancing Translarna to approval: “PTC remains focused on the global development of Translarna and commercialization outside of the U.S. for nmDMD.”

In Europe, Translarna was granted conditional approval in 2014, subject to annual review and a further review of results from ACT DMD.

[This report has been updated from an earlier version to include an updated figure from PTC on its current workforce size.]

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