Snubbed at the FDA, PTC files their three-time los­er for Duchenne MD over protest

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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

David Baker working with a student on their protein design (Jason Mast)

Sci­en­tists are fi­nal­ly learn­ing how to de­sign pro­teins from scratch. Drug de­vel­op­ment may nev­er be the same

SEATTLE — It’s a cloudy Thursday afternoon in mid-July and David Baker is reclining into the futon in his corner office at the University of Washington, arms splayed out like a daytime talk show host as he coaches another one of his postdocs through the slings and arrows of scientific celebrity.

“Be jealous of your time,” he says, before plotting ways of sneaking her out of Zooms. “It’s this horrible cost to science that you’re tied up in some stupid meeting.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Patty Murray, D-WA (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.

Per­pet­u­al cri­sis? Phar­ma com­mu­ni­ca­tions and pub­lic re­la­tions pros just wait for the next shoe to drop

Welcome to pharma public relations where every day feels like it might be a crisis.

More than three-fourths (76%) of pharma communications leaders expect to face three or more crises this year — compared to less than half (49%) of their peers in other industries, according to new survey from risk intelligence company Crisp. The same 76% also expect that a brand new risk that is yet to be identified will crop up this year, compared to 66% of peers across other industries who worry about the same thing.

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As new C. diff treat­ments prep for mar­ket, spe­cial­ists di­vid­ed on 'poop in a pil­l' or small mol­e­cule, study finds

What do physicians think about the emerging market for recurrent C. diff? That depends on who you talk to, especially which specialists you talk to.

Fecal microbiota transplants, or FMT, are favored by gastroenterologists familiar with them, while infectious disease doctors prefer traditional small molecule therapies, according to a recent future market evaluation by Spherix Global Insights. While current FMT involves transplanting healthy stool into a C. diff patient through a colonoscopy, FMT encapsulated microbiome pills that are swallowed – referred to by doctors as “poop in a pill” in Spherix’s interviews – are likely on the way to market.

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