Marathon, PTC deal

PTC will acquire Duchenne muscular dystrophy drug Emflaza deflazacort from Marathon. Marathon had proposed pricing the drug at a wholesale acquisition cost (WAC) of $89,000 per year, but suspended its launch plans after facing criticism. Deflazacort has been available in the U.S. via importation from Canada or the U.K. for $1,200-$1,500 a year. The company said it believed 7-9% of U.S. DMD patients were receiving imported Deflazacort. PTC said it will "reexamine" Emflaza's price but will not disclose details until after the deal closes next quarter. PTC will not acquire the rare pediatric disease Priority Review voucher FDA granted Marathon in conjunction with Emflaza's Feb. 9 approval (see BioCentury, Feb. 10 & Feb. 16).

Emflaza is an anti-inflammatory, immunosuppressant glucocorticoid and is approved for DMD patients ages five and older. PTC markets another therapy, Translarna ataluren, in the EU to treat nonsense mutation DMD. Translarna is under FDA review in the U.S. in the same indication, with an Oct. 24 PDUFA date. PTC submitted Translarna's NDA over protest after FDA issued a refusal to file letter last year. PTC said the Emflaza deal will allow it to establish a U.S. commercial footprint ahead of a potential Translarna launch. Translarna is a small molecule that facilitates complete translation of proteins containing nonsense mutations (see BioCentury, March 9)...