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.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.