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Top FDA News This Week


High hopes for COVID-19 vaccine for children before the end of the year, Breakthrough Designation for Jardiance, thumbs down on an EUA for cytokine storm therapy and no decision yet on Juul's vaping products.

Top vaccine official hopeful about OK for COVID-19 shots for kids this year

Peter Marks, M.D., Ph.D., said in an interview with the Associated Press that was published on Friday (Sep. 10) that he was “very, very hopeful” about 5- to 11-year-olds getting vaccinated against COVID-19 this year.

Peter Marks

Peter Marks

Marks is director of the Center for Biologics Evaluation and Research, the part of the FDA that oversees biological products such as vaccines, blood and blood products, and gene therapies.

In response to a question about whether 5- to 11-year-olds could be vaccinated this year, AP quoted Marks as saying, “I am very hopeful in that regard. Very, very hopeful in that regard.”

Marks referenced a statement from Pfizer that the company plans to give the drug regulator data from its clinical trials in children by the end of September. “We’re going to do a thorough job on that as quickly as we can so that at the end of the day, hopefully within a matter of weeks rather than a matter of months we’ll be able to come to some conclusion,” Marks said.

Jardiance gets Breakthrough designation

Eli Lilly and Boehringer Ingelheim announced on Thursday (Sept. 9) that the FDA had given Jardiance (empagliflozin) the Breakthrough Therapy designation as a treatment for adults with heart failure with preserved ejection fraction.

Jardiance, a drug in the SGLT2 inhibitor class that was originally approved as a treatment for type 2 diabetes, was approved as a treatment for heart failure with reduced ejection fraction in August. About half of the six million heart failure patients in the United States have reduced ejection fraction — the amount of blood pumped by each contraction of the left ventricle — and half have preserved ejection fraction.

Breakthrough designation means drug developers get guidance from the FDA that is supposed to hasten its development and eventual approval.

A press release from the two companies said the decision is based on results from the EMPEROR-Preserved phase 3 trial, in which Jardiance demonstrated a 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction compared with placebo. The benefit was independent of ejection fraction or diabetes status, said the press release. Results from trial were presented at the European Society of Cardiology Congress 2021 on August 27 and published in The New England Journal of Medicine.

Electric field cancer therapy for advanced liver cancer gets Breakthrough nod

Novocure announced on Thursday (Sept.9) that its device for treating cancer with electric fields had been granted a Breakthrough Designation by the FDA. For advanced liver cancer, the company’s system is designed to be used with Tecentriq (atezolizumab) and Avastin (bevacizumab).

The company announcement said the designation was based, in part, on results from its phase 2 pilot HEPANOVA trial, which included patients treated with Nexavar (sorafenib). In July, Novocure announced that in 21 patients in the trial, the disease control rate was 76% in a patient population with poor prognosis and limited exposure to study treatments. According to the company’s announcement, the objective response rate for the intent-to-treat population was 9.5%. In patients who completed at least 12 weeks with its electric treatment, the disease control rate was 91% with an objective response rate of 18%.

Novocure’s technology, which it calls tumor treating fields, uses alternating electric fields to disrupt the cell division of cancer cells. The
FDA has approved the technology as a treatment for glioblastoma and mesothelioma, and Novocure says it is being tested in phase 3 trials for brain, nonsmall lung, pancreatic and ovarian cancer.

No EUA for cytokine storm therapy

Serious cases of COVID-19 become even more so if the viral infection stirs up “cytokine storms,” an aspect of an exaggerated immune response that leads to overproduction of cytokines, which can cause bleeding, organ failure, severe respiratory distress and premature death. Several companies are developing therapies aim at quelling cytokine storm.

One of them, Humanigen, a biotech company in Burlingame, California, that bills itself as a “the cytokine storm company” announced on Thursday (Sept. 9) that announced on Thursday (Sept. 9) that the FDA had declined its request for an emergency use authorization for its main cytokine storm drug, lenzilumab, to treat newly hospitalized COVID-19 patients. “In its letter, FDA stated that it was unable to conclude that the known and potential benefits of lenzilumab outweigh the known and potential risks of its use as a treatment for COVID-19,” said company statement said.

“FDA slams the door in Humanigen’s face,” said the Fierce Biotech headline. The company’s stock price fell from $15.11 on Wednesday to $6.88 on Friday.

“We remain committed to bringing lenzilumab to patients hospitalized with COVID-19,” said Cameron Durrant, M.D., the company’s CEO said in the statement issued on Thursday. “We believe the ongoing ACTIV-5/BET-B trial, which has been advanced to enroll up to 500 patients, may provide additional safety and efficacy data sufficient to support our efforts to obtain an EUA to treat hospitalized COVID-19 patients.”

FDA taking a look at secondary end point claims in DTC ads

Endpoints News reported Friday (Sept. 10) that the FDA announced that it is studying the use of claims about secondary end points in direct-to-consumer advertising.

“In some cases, truthful and nonmisleading presentations about secondary endpoints in well-designed clinical studies can provide reliable information about treatment effects that may be distinct from the treatment effects described in the product’s indication statement,” FDA said in the Federal Register, according to the pharma news website.

The agency is focusing on diabetes drugs and the secondary claim disclosure that explains “even though the drug is not indicated for weight loss, that it can help some people lose weight,” according to Endpoints News.

Fate of Juul vaping products still uncertain as agency announces it has banned others

The FDA announced on Thursday (Sept. 9) that it had issued 132 market denial orders to keep 946,000 vaping products — “electronic nicotine delivery systems”, or ENDS products, in official FDA parlance — off the market. But as numerous news accounts pointed out, the FDA has not yet decided the fate of the Juul’s vaping products, which account for a large share of the vaping product.

The FDA announcement said that it had issued the marketing denial orders because the applications “lacked sufficient evidence that they have a benefit to adult smokers sufficient to overcome the public health threat posed by the well-documented, alarming levels of youth use of such products.”

The Wall Street Journal had reported earlier that the FDA won’t make a Sept. 9 deadline to review the vaping products of Juul and other major players in the vaping market, such as Reynolds American, which makes Vuse e-cigarettes.

The announcement on Thursday was noncommittal about when the review would be complete.

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