PTC shares plunge anew af­ter FDA slaps down Duchenne drug for a sec­ond time

PTC Ther­a­peu­tics CEO Stu­art Peltz

There will be no Lazarus-like rise from the dead for PTC Ther­a­peu­tics at the FDA — at least for now. The agency has slapped down the biotech’s first ap­peal of the FDA’s re­fusal to even con­sid­er what it has al­ready con­clud­ed was an in­ad­e­quate ap­pli­ca­tion for its Duchenne mus­cu­lar dy­s­tro­phy drug Translar­na (ataluren).

The news drove PTC’s shares $PTCT down 40% by the end of the day, as in­vestors bet against any ap­proval in the near fu­ture.

The FDA’s con­tro­ver­sial de­ci­sion to ap­prove Sarep­ta’s Ex­ondys 51 (eteplirsen) for DMD has prompt­ed Bio­Marin $BM­RN to con­sid­er mak­ing an ap­peal on the FDA’s re­jec­tion of dris­apersen, as End­points News re­port­ed on Thurs­day. But PTC hadn’t wait­ed for that de­ci­sion be­fore start­ing its own cam­paign to get in front of reg­u­la­tors and ex­perts to make its pitch.

Back in Feb­ru­ary the FDA had is­sued a refuse-to-file no­tice on ataluren, re­ject­ing the ap­pli­ca­tion at the out­set. But even with this sec­ond re­jec­tion, the biotech isn’t back­ing off. In­stead, PTC says it is prep­ping for an­oth­er ap­peal to “the next su­per­vi­so­ry lev­el of the FDA.”

Like Sarep­ta $SRPT, PTC wants a chance to get pa­tient ad­vo­cates in front of reg­u­la­tors to help make their case. And some are ready to go.

“This in­con­sis­ten­cy is un­ac­cept­able and is con­cern­ing for the en­tire com­mu­ni­ty,” said Pat Fur­long, founder of Par­ent Pro­ject Mus­cu­lar Dy­s­tro­phy, in a state­ment. “This dev­as­tat­ing, mus­cle-wast­ing dis­ease cuts short the lives of boys and young men and every day that we wait for treat­ments, is a day in which mus­cle func­tion is lost and not re­gained. As a com­mu­ni­ty, we can­not rest un­til there are treat­ments for all the boys and young men.”

PTC has al­ready re­ceived con­di­tion­al ap­proval in Eu­rope, de­spite the fact that the drug has failed clin­i­cal stud­ies for DMD. But the biotech has nev­er stopped in­sist­ing that it has iden­ti­fied pos­i­tive da­ta that would sup­port an ap­proval.

Gena Wang at Jef­feries says she’s not sur­prised, and giv­en the re­cent ac­tiv­i­ty among Eu­ro­pean reg­u­la­tors, she thinks PTC has plen­ty to wor­ry about on that front as well.

“CHMP is­sued a re­quest for sup­ple­men­tal in­for­ma­tion (RSI) in­clud­ing a ma­jor ob­jec­tion for ataluren re­view, sug­gest­ing high risk for PTCT to re­new the EU mar­ket­ing au­tho­riza­tion,” she not­ed this morn­ing.  “Fol­low­ing an oral ex­pla­na­tion meet­ing re­gard­ing the re­new­al of ataluren EU mar­ket­ing au­tho­riza­tion (con­di­tion­al ap­proval in 2014), CHMP is­sued a RSI, in­clud­ing a ma­jor ob­jec­tion per­tain­ing to ataluren’s ef­fi­ca­cy, over­all risk-ben­e­fit pro­file and de­sign/con­duct of an ad­di­tion­al clin­i­cal tri­al to pro­vide com­pre­hen­sive clin­i­cal da­ta. Ad­di­tion­al con­cerns re­lat­ed to the pri­ma­ry phar­ma­col­o­gy of ataluren and la­bel mat­ters were al­so raised. It would be PTCT’s re­spon­si­bil­i­ty to ful­ly ad­dress con­cerns raised by CHMP and the com­pa­ny ex­pects an opin­ion from CHMP by YE16. We con­tin­ue to see high risk of re­new­ing ataluren’s EU au­tho­riza­tion.”

“We be­lieve that fair con­sid­er­a­tion of the to­tal­i­ty of Translar­na’s da­ta re­quires a full re­view of our ap­pli­ca­tion by the FDA,” said PTC CEO Stu­art W. Peltz. “In light of this, con­tin­u­ing the for­mal dis­pute res­o­lu­tion process re­flects our on­go­ing com­mit­ment to work with reg­u­la­tors and the Duchenne com­mu­ni­ty to make Translar­na avail­able to nmD­MD pa­tients in the Unit­ed States.”

John Hood [file photo]

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