PTC Ther­a­peu­tics $PTCT isn’t go­ing to let a slate of three con­sec­u­tive, failed stud­ies for ataluren block its re­view at the FDA. Us­ing the agency’s rules for ap­pli­ca­tions on drugs reg­u­la­tors have re­fused to file, the biotech says it filed over protest, us­ing reg­u­la­to­ry pro­ce­dures to force a PDU­FA date of Oc­to­ber 24.

PTC CEO Stu­art Peltz was frank about why the FDA wouldn’t ac­cept their ap­pli­ca­tion ear­ly last year. Reg­u­la­tors felt that the com­pa­ny had flat failed to make a case for the drug that was wor­thy of a re­view. And he’s been beat­ing on the FDA’s door ever since, de­spite the agency’s de­ci­sion to re­ject its first ap­peal, claim­ing once again that the “to­tal­i­ty” of the da­ta war­rant its ap­proval.

In a state­ment, the CEO said he would be “work­ing close­ly with the FDA and the DMD com­mu­ni­ty to bring this much-need­ed ther­a­py to pa­tients.”

Cur­rent­ly, the EU al­lows the drug to be sold af­ter grant­i­ng a con­di­tion­al ap­proval that now re­quires PTC to con­duct a new clin­i­cal tri­al over the next 5 years. PTC’s move to­day un­der­scores a dif­fer­ent set of stan­dards that reg­u­la­tors have ap­plied to drugs in the field.

While the FDA spurned PTC and re­ject­ed Bio­Marin’s dris­apersen, the FDA’s Janet Wood­cock over­rode the stren­u­ous ob­jec­tions of nu­mer­ous sub­or­di­nates who felt that Sarep­ta had nev­er made its case for eteplirsen. Pa­tients and fam­i­lies or­ga­nized a co­or­di­nat­ed cam­paign to win an ap­proval, though, and the drug is now sold as Ex­ondys 51 with a la­bel that says ef­fi­ca­cy of the drug has nev­er been es­tab­lished.

Jef­feries’ Gena Wang doesn’t give PTC very good odds.

We not­ed that the ‘fil­ing over protest’ has been rarely pur­sued and would be con­sid­ered as a new ap­pli­ca­tion pro­ce­du­ral­ly. We found two prece­dents – Phar­ma­cyclics’ Xcytrin in lung can­cer brain metas­tases (fil­ing over protest in Apr ’07, PDU­FA date of Dec 31 ’07 and non-ap­prov­able let­ter on Dec 21 ’07), and GSK’s Bexxar in Non-Hodgkin’s Lym­phoma (ini­tial fil­ing in 1999, and ac­cel­er­at­ed ap­proval af­ter fil­ing over protest in June ’03; with­drawn by GSK in 2013 due to in­com­plete post-mar­ket­ing tri­als).

“The bull ar­gu­ment for PTCT has been that eteplirsen’s ap­proval “opens the door” for ataluren,” not­ed RBC’s Simos Sime­oni­dis. “We def­i­nite­ly agree that both ap­pli­ca­tions are “less-than-per­fect” and, in our view, nei­ther one should have been ap­proved. But even though we dis­agreed with FDA on eteplirsen, we can un­der­stand their ra­tio­nale for ac­cel­er­at­ed ap­proval. The key and very sig­nif­i­cant dif­fer­ence be­tween the two ap­pli­ca­tions is that we would char­ac­ter­ize the eteplirsen dataset as in­com­plete and the ques­tion of whether eteplirsen “works” in DMD as still unan­swered. This can­not be said about ataluren, where mul­ti­ple large, well-con­trolled, ran­dom­ized tri­als have been con­duct­ed. ” They all failed.

PTC ac­knowl­edged just days ago that its drug failed a late-stage pro­gram for cys­tic fi­bro­sis, adding to the ev­i­dence that the drug doesn’t work. Ear­li­er it failed a Phase IIb for Duchenne MD fol­lowed by an­oth­er flop in Phase III, de­signed to over­come the prob­lems the biotech saw ear­li­er.

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.