FDA hands Bris­tol My­ers a land­mark ap­proval in ear­ly-stage lung can­cer af­ter just five days

On Feb. 28, the FDA ac­cept­ed Bris­tol My­ers Squibb’s ap­pli­ca­tion for ap­proval of a new use of block­buster check­point in­hibitor Op­di­vo and said it would make a de­ci­sion by Ju­ly 13.

That es­ti­mate turned out to be con­ser­v­a­tive, to say the least. On Fri­day, just five days af­ter ac­cept­ing the ap­pli­ca­tion, the FDA OK’d it, hand­ing the Big Phar­ma a land­mark ap­proval.

Op­di­vo can now be used to treat pa­tients with ear­ly-stage non-small cell lung can­cer pri­or to re­ceiv­ing surgery to re­move their tu­mor. Al­though Op­di­vo and oth­er PD-(L)1 in­hibitors have al­ready trans­formed treat­ment for more ad­vanced forms of the dis­ease, im­prov­ing sur­vival by re­plac­ing or sup­ple­ment­ing tox­ic chemother­a­py with more tol­er­a­ble and ef­fec­tive mol­e­cules that take the “brakes” off the im­mune sys­tem, the drugs have yet to reach many of the ear­li­est stage pa­tients.

Op­di­vo’s new ap­proval is part of a broad­er race by Big Phar­mas to move their PD-(L)1 drugs in­to ear­li­er lines of ther­a­py, a push that ex­perts say could con­tin­ue to re­make the stan­dard-of-care treat­ment (along with com­pa­nies’ pock­ets).

It’s al­so a key win for Bris­tol My­ers, the #2 play­er in the check­point world, in gain­ing a toe­hold on Mer­ck’s dom­i­nant po­si­tion on the mar­ket. Al­though Bris­tol put the first PD-(L)1 drug on the mar­ket, it lost its pre­ferred sta­tus among clin­i­cians af­ter fail­ures in ad­vanced non-small lung can­cer, where Mer­ck pro­duced prac­tice-chang­ing re­sults.

In the Phase III tri­al that led to ap­proval, 388 pa­tients with op­er­a­ble lung can­cer were ran­dom­ized to re­ceive chemother­a­py alone or chemother­a­py plus Op­di­vo pri­or to surgery. Pa­tients in the Op­di­vo arm went a me­di­an of 31.6 months with­out any type of re­cur­rence, com­pared to 20.8 months for pa­tients on chemo alone.

Mark Awad

Just un­der a quar­ter of pa­tients on Op­di­vo al­so saw patho­log­ic com­plete re­sponse — biop­sies with no signs of can­cer un­der close in­spec­tion — com­pared to 2.2% of pa­tients on chemo alone. Op­di­vo pa­tients al­so ap­peared to live longer, al­though that num­ber was not yet sta­tis­ti­cal­ly sig­nif­i­cant.

Mark Awad, clin­i­cal di­rec­tor of the Lowe Cen­ter for Tho­racic On­col­o­gy at the Dana-Far­ber Can­cer In­sti­tute and a study in­ves­ti­ga­tor, said in a state­ment that the ap­proval “marks a turn­ing point in how we treat re­sectable NSCLC.”

Mer­ck, of course, is now run­ning its own tri­als in the same set­ting, as are oth­er PD-(L)1 play­ers such as Roche. They are all al­so com­pet­ing to be first in ear­ly lines of ther­a­py for oth­er can­cers.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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FDA re­view­ers head back to White Oak in 2023, with lead­er­ship look­ing to ap­pease a new Con­gress

Republicans have taken a stand against the pandemic era habit of lax work-from-home schedules. Now that they’ve wrestled control of the House majority, the FDA’s leadership is playing ball, sending many of the agency’s more than 18,000 employees back to their desks early next year.

Whether this exodus back to White Oak in Silver Spring, MD (many staff will still be allowed to work from home for multiple days per week) will mean more defections to industry and elsewhere remains to be seen.

Bags of shred­ded docs: In­di­an drug­mak­er Lupin hand­ed a Form 483 by FDA in­spec­tors

The generics manufacturer Lupin has been given another Form 483 from the FDA this year.

US regulators inspected Lupin’s pharmaceutical manufacturing site in the town of Mandideep, India from Nov. 14 through Nov. 23, with the 14-page report marking 16 observations.

The inspection report stated that the site did not have the appropriate controls over its computer systems to ensure that changes in “master production” or records are only done by authorized personnel, along with written procedures not being established to conduct annual reviews of records associated with drug batches.

Bro­ken promis­es? FDA needs more pow­er to re­move drugs from mar­ket­place, JA­MA analy­sis finds

The FDA is struggling to remove drugs from the marketplace that don’t show effectiveness in late stage trials, new JAMA analyses found, thanks to the persistent tension between speed and confidence in early clinical data.

Congress, regulated industry and patients have urged the FDA to shorten the amount of time that the market has to wait for drugs to become available that may help severe and prevalent diseases – and the FDA has listened, offering up a quick accelerated approval pathway that’s frequently used by new cancer drugs.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.