Ear­ly last month, Proven­tion Bio’s shares cratered af­ter dis­clos­ing that the FDA found the phar­ma­co­ki­net­ic pro­files of its po­ten­tial type 1 di­a­betes drug, ac­quired from Eli Lil­ly, dif­fered when man­u­fac­tured by Lil­ly ver­sus Proven­tion.

At the time, the FDA said its con­cerns meant that it was not ready to start post-mar­ket­ing and la­bel dis­cus­sions with the com­pa­ny.

But now, two days ahead of the FDA’s En­docrino­log­ic and Meta­bol­ic Drugs Ad­vi­so­ry Com­mit­tee meet­ing to dis­cuss the drug, known as teplizum­ab, the FDA sound­ed a rel­a­tive­ly pos­i­tive tone on safe­ty and ef­fi­ca­cy. The com­pa­ny’s stock was up about 30% ear­ly Tues­day.

While not­ing some miss­ing da­ta from the com­pa­ny’s tri­als, the FDA said Tues­day that “be­cause the amount of miss­ing da­ta was small, sen­si­tiv­i­ty analy­ses per­formed us­ing dif­fer­ent miss­ing da­ta han­dling ap­proach­es show that the ob­served ef­fi­ca­cy by the pri­ma­ry analy­sis method is suf­fi­cient­ly ro­bust to with­stand con­ser­v­a­tive ap­proach­es in han­dling miss­ing da­ta.”

The ques­tion of what con­sti­tutes sub­stan­tial ev­i­dence of ef­fec­tive­ness will be top of mind for the com­mit­tee to ad­dress, FDA notes, ex­plain­ing how Proven­tion sub­mit­ted a rel­a­tive­ly small, sin­gle place­bo-con­trolled tri­al with the pri­ma­ry end­point of de­lay of clin­i­cal type 1 di­a­betes, as well as ad­di­tion­al da­ta, which FDA said can be used as “con­fir­ma­to­ry ev­i­dence.”

On safe­ty, the brief­ing doc­u­ments did not re­veal any glar­ing red flags, al­though the agency not­ed that “slight­ly more than 10% of pa­tients” in the tri­al “were not able to re­ceive the full course of teplizum­ab sec­ondary to meet­ing pro­to­col-spec­i­fied with­draw­al cri­te­ria.”

The com­mit­tee on Thurs­day will be tasked with not on­ly dis­cussing the clin­i­cal mean­ing­ful­ness of the ob­served me­di­an 2-year de­lay of on­set of type 1 di­a­betes demon­strat­ed in the com­pa­ny’s main study, but vot­ing on an up or down ap­proval ques­tion ahead of the FDA’s de­ci­sion, which is due by Ju­ly 2.

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.