Top FDA News This Week

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

In response to a question about the possibility of immunizing children aged 5 to 11 this year, AP quoted Marks: “I have a lot of hope in this regard. Very, very optimistic in this regard.

Marks referred to a statement from Pfizer that the company plans to provide the drug regulator with data from its clinical trials in children by the end of September. “We’re going to do a thorough job on this as quickly as possible so that at the end of the day, hopefully a few weeks rather than a few months, we can come to a conclusion,” says Marques.

Jardiance obtains the Breakthrough designation

Eli Lilly and Boehringer Ingelheim announced Thursday, September 9 that the FDA has awarded 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 initially 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 a reduced ejection fraction – the amount of blood pumped by each contraction of the left ventricle – and half have a preserved ejection fraction.

The breakthrough designation means drug developers receive advice from the FDA that is supposed to accelerate its development and eventual approval.

The press release from both companies says the FDA’s decision was based on the results of the Phase 3 EMPEROR-Preserved trial, in which Jardiance showed a 21% relative risk reduction for the primary endpoint. composite of cardiovascular death or hospitalization for heart failure in adults with heart disease. failure with ejection fraction conserved relative to location (FDA) has

Electric field cancer treatment for advanced liver cancer gets revolutionary nod

Novocure announced Thursday, September 9, that its cancer treatment device with electric fields had received a breakthrough designation from the FDA. For advanced liver cancer, the company’s system is designed to be used with Tecentriq (atezolizumab) and Avastin (bevacizumab).

The company’s announcement said the designation was based, in part, on the results of its Phase 2 HEPANOVA pilot 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 an unfavorable prognosis and limited exposure to the study treatments. According to the company’s announcement, the objective response rate of the intention-to-treat population was 9.5%. In patients who completed at least 12 weeks with her electrical treatment, the disease control rate was 91% with an objective response rate of 18%.

Novocure’s technology, which it calls tumor treatment fields, uses alternating electric fields to disrupt the cell division of cancer cells. The
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, non-small lung, pancreatic and ovarian cancer. .

No EUA for cytokine storm therapy

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

One of them, Humanigen, a Burlingame, Calif., Biotech company that touts itself as a “cytokine storm company” announced Thursday, September 9 that the FDA had declined its request for authorization for use. emergency drug for its leading cytokine storm drug, lenzilumab, to treat newly hospitalized COVID-19 patients. “In its letter, the FDA said 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 the company press release.

“The FDA is slamming the door on Humanigen,” said the title of Fierce Biotech. The company’s stock price fell from $ 15.11 Wednesday to $ 6.88 Friday.

“We remain committed to bringing lenzilumab to patients hospitalized with COVID-19,” said Cameron Durrant, MD, the company’s CEO said in a statement released Thursday. “We believe that 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 achieve EUA to treat COVID-19 hospital patients. “

FDA Examines Secondary Endpoint Claims in DTC Ads

Terminal news reported on Friday (September 10) that the FDA announced it was investigating the use of secondary endpoint claims in direct-to-consumer advertising.

“In some cases, truthful and not misleading presentations of secondary endpoints in well-designed clinical studies may provide reliable information on the effects of treatment which may be distinct from the effects of treatment described in the statement of indication for the drug. product, ”the FDA said in the Federal Register, according to the Pharmaceuticals News site.

Agency Focuses On Diabetes Drugs And Disclosure Of Secondary Claim That “Even though the drug is not indicated for weight loss, it may help some people lose weight,” Endpoints says News.

Fate of Juul vaping products remains uncertain as agency announces it has banned more

The FDA announced Thursday (September 9) that it had issued 132 denial-of-market orders to maintain 946,000 vaping products – “electronic nicotine delivery systems,” or ENDS products, in the official parlance of the FDA – off the market. But as numerous press reports have pointed out, the FDA has yet to decide the fate of Juul’s vaping products, which make up a significant portion of the vaping product.

The FDA announcement said it issued the denial-of-market orders because the applications “lacked sufficient evidence that they provided sufficient benefit to adult smokers to overcome the public health threat posed by the drugs. alarming and well-documented levels of use of these products by young people. “

The the Wall Street newspaper reported earlier that the FDA would not set a September 9 deadline to review vaping products from Juul and other major players in the vaping market, such as Reynolds American, which makes Vuse e-cigarettes.

Thursday’s announcement was non-binding as to when the review would be completed.