Hunt­ing a full OK for Baven­cio in front­line blad­der can­cer, Pfiz­er, Mer­ck KGaA re­ceive VIP treat­ment at FDA

The JAVELIN Blad­der 100 study was one of the rare bright spots in Baven­cio’s check­ered de­vel­op­ment record. Now Pfiz­er and Mer­ck KGaA get to bur­nish that with a break­through ther­a­py des­ig­na­tion for first-line main­te­nance treat­ment of lo­cal­ly ad­vanced or metasta­t­ic urothe­lial car­ci­no­ma.

The part­ners have sub­mit­ted their sBLA, which is now be­ing re­viewed on a re­al-time ba­sis. Un­der that pi­lot pro­gram — which be­gan in late 2018 — reg­u­la­tors could ac­cess da­ta pri­or to the of­fi­cial ap­pli­ca­tion fil­ing.

Pfiz­er and Mer­ck KGaA would have ap­plied for the RTOR at the be­gin­ning of this year, when it re­leased topline re­sults of a Phase III study with Baven­cio. As a main­te­nance ther­a­py, the PD-L1 drug hit the mark on over­all sur­vival for both the to­tal pa­tient group and a sub­set with PD-L1 pos­i­tive tu­mors.

The drug was al­ready sanc­tioned, un­der ac­cel­er­at­ed ap­proval, for use in pa­tients fol­low­ing dis­ease pro­gres­sion — an OK ce­ment­ed by tu­mor re­sponse and du­ra­tion of re­sponse back in 2017.

“For the past 30 years, chemother­a­py has been the first-line stan­dard of care for pa­tients with ad­vanced urothe­lial car­ci­no­ma. While this is an ef­fec­tive short-term op­tion for many pa­tients, most will ul­ti­mate­ly ex­pe­ri­ence dis­ease pro­gres­sion, un­der­scor­ing a need for ad­di­tion­al treat­ment op­tions,” said Pet­ros Gri­vas, a prin­ci­pal in­ves­ti­ga­tor in the JAVELIN Blad­der 100 tri­al.

Chris Boshoff

In the tri­al, Baven­cio was added to the reg­i­men for pa­tients whose dis­ease had not pro­gressed dur­ing or fol­low­ing plat­inum-con­tain­ing chemo. The OS re­sults could be prac­tice-chang­ing, Gri­vas added.

Chris Boshoff, Pfiz­er’s chief de­vel­op­ment of­fi­cer in on­col­o­gy, high­light­ed that Baven­cio is the first im­munother­a­py to demon­strate an OS ben­e­fit in first-line, lo­cal­ly ad­vanced or metasta­t­ic urothe­lial car­ci­no­ma.

As­traZeneca pro­vid­ed the lat­est il­lus­tra­tion on just how tough it can be to beat this ad­vanced can­cer when Imfinzi flopped an open la­bel piv­otal study last month. The ther­a­py — which al­so blocks PD-L1 — nabbed ac­cel­er­at­ed ap­proval in 2017 but failed to help pa­tients live longer com­pared to stan­dard-of-care chemother­a­py. The drug­mak­er may shift their fo­cus to the sec­ond-line set­ting, it in­di­cat­ed to End­points News.

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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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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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.

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.

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.

FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.