FDA Approves A Drug For A Rare Disease That Causes Children To Age Quickly : Shots

Sam Berns and Audrey Gordon, executive director of The Progeria Research Foundation and Berns’s aunt, attend The New York Premiere Of HBO’s “Life According To Sam” on October 8, 2013 in New York City.

Thos Robinson/Getty Images for HBO


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Sam Berns and Audrey Gordon, executive director of The Progeria Research Foundation and Berns’s aunt, attend The New York Premiere Of HBO’s “Life According To Sam” on October 8, 2013 in New York City.

Thos Robinson/Getty Images for HBO

The Food and Drug Administration has approved a drug that extends the lives of children with an extremely rare genetic disorder that causes them to grow old before they grow up.

The disorder, progeria, ages cells rapidly and prematurely. As a result, affected children remain small and begin to look frail and old by the time they reach school age. Most die of heart disease in their early teens.

But the drug, Zokinvy, slows down the decline.

“Zokinvy is a treatment. It’s not a cure,” says Dr. Leslie Gordon, an assistant professor of pediatrics at Brown University and medical director of The Progeria Research Foundation. “But what we can say is that, so far, we know that it increases the average lifespan by about two-and-a-half years.”

Gordon and her family played a crucial role in making Zokinvy possible.

Their goal was to find a treatment for Gordon’s son, Sam Berns, who was diagnosed with progeria in 1998 and died in 2014.

“We started this for Sam, and Sam is always here, always,” Gordon says.

Sam Berns gave a TEDx talk about living with progeria.

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As a teenager, Sam became the public face of progeria. He spoke to NPR and other media about his condition, was the subject of an HBO documentary, and gave a TEDx talk about how to live a happy life that has been viewed more than 40 million times.

Sam was one of the first children with progeria to receive Zokinvy and Gordon says the drug seemed to extend her son’s life.

“I think Sam felt that way,” she says. “And looking at the data, and as parents, we felt that way too.”

Since Sam’s death, his family has continued to operate The Progeria Research Foundation and to search for a cure.

“We make a really great team,” says Audrey Gordon, who is Sam’s aunt and the foundation’s president. “Leslie [is] in charge of the science aspect, I’m in charge of the fundraising and her husband, Scott, is the chairman of the board.”

The approach reflects a philosophy embraced by Sam, who was a big sports fan, Audrey Gordon says.

“Whenever someone asked, who’s your favorite player on your beloved Patriots or your Boston Bruins, he always refused because he felt like it wasn’t any one person who made the team,” she says “It was the team as a whole.”

Team Sam published the first evidence that Zokinvy worked in 2012. But getting FDA approval required a whole new

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AstraZeneca to seek FDA authorization for vaccine based on foreign trial data

The company also said it would work with the agency to adjust the design of its late-stage U.S. trial to test the half-dose regimen, rather than the higher dose that proved less effective in the U.S and Brazil studies. That U.S. trial has enrolled about 10,000 of a planned 40,000 participants, but the company has not released any data from that study.

The U.K. and Brazil studies have enrolled about 24,000 participants — fewer than the 30,000 participants that the FDA is requiring for late-stage coronavirus vaccine trials.

The initial findings were based on 131 infections among trial participants. The company did not break down how many cases were reported among those who got a placebo versus those who got the vaccine, and within that vaccine group, how cases split among the two doses tested.

AstraZeneca also said that none of the infected people had severe Covid-19 or were hospitalized, but offered no further safety information.

Promising logistics: The AstraZeneca vaccine is cheaper and easier to distribute than the other two shots that have proven effective. It can be transported and stored under refrigerated conditions for at least six months, and the company says it can make up to 3 billion doses next year.

Vaccines from Pfizer and Moderna, which have each proven about 95 percent effective, must be frozen during distribution and kept at very cold temperatures.

“AstraZeneca and Oxford have developed an affordable, scalable vaccine that crucially can be stored and shipped in a regular refrigerator,” said Richard Hatchett, CEO of the Coalition for Epidemic Preparedness Innovations, said in a statement. “This makes it appropriate for use and easy to deliver almost anywhere in the world, including in low-resource settings.”

But questions remain: Others doubt whether the FDA will authorize the vaccine.

“We believe that this product will never be licensed in the US,” investment bank SVB Leerink analyst Geoffrey Porges said in a note on Monday. “This belief is based on the design of the company’s pivotal trials (which does not appear to match the FDA’s requirements for representation of minorities, severe cases, previously infected individuals and elderly and other increase risk populations), and based on the occurrence of severe safety events (why take the risk) that resulted in the extended clinical hold on enrollment into the trials in the US.”

The company halted its U.S. trial in early September over safety concerns, after a trial participant reported neurological problems. The study resumed earlier this month after FDA concluded that no evidence linked the volunteer’s symptoms to the shot.

Background: The AstraZeneca vaccine was developed by scientists at Oxford University, and uses a different technology than the Moderna and Pfizer vaccines. It uses a weakened version of the common cold that contains some genetic material from the coronavirus.

U.S. vaccine and therapeutics accelerator Operation Warp Speed paid for some of the clinical development of the AstraZeneca vaccine and purchased 300 million doses for $1.2 billion.

Other clinical trials are ongoing in Japan, Russia, South Africa, Kenya

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FDA bars critic from review panel of Biogen’s controversial Alzheimer’s drug

By Deena Beasley

(Reuters) – The U.S. Food and Drug Administration, citing conflict of interest, has recused a member of an 11-person advisory committee set to review Biogen Inc’s experimental Alzheimer’s drug aducanumab on Friday.

Mayo Clinic neurologist Dr. David Knopman confirmed on Monday that he was recused from the scheduled meeting of outside advisors to the FDA. Expert advisory panels often play an important role ahead of FDA approval decisions, although the agency is not required to take their advice.

Knopman, who said he was recused because of his involvement in conducting clinical trials of aducanumab, has been a vocal critic of the drug, including in a paper published on Sunday in the journal Alzheimer’s & Dementia.

“Aducanumab’s efficacy as a treatment for the cognitive dysfunction in Alzheimer’s disease cannot be proven by clinical trials with divergent outcomes,” he wrote.

The FDA, which is slated to decide by early March whether to approve aducanumab, did not immediately respond to a request for comment.

If approved, aducanumab would be the first drug to treat an underlying cause of the fatal, mind-wasting disease, a field that has for decades produced nothing but failure. By 2025, the number of people age 65 and older with Alzheimer’s dementia is projected to reach 7.1 million in the United States alone, according to the Alzheimer’s Association.

Biogen has said aducanumab, an antibody designed to remove harmful plaques from the brain, was shown in one of two major studies to significantly slow cognitive and functional decline. A second trial, however, showed a benefit only for a subset of patients who were given a high dose for at least 10 months.

Biogen abruptly ended a pivotal trial of aducanumab last year after an early look at results suggested it did not provide a desired benefit. Months later, the biotech company shocked many by reversing course, saying it had decided to seek FDA approval after all.

Shares of Biogen were down about 0.5% at $250.78.

(Reporting By Deena Beasley; Editing by Bill Berkrot)

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Here’s how the FDA is advising hospitals to prepare for future public-health emergencies

While the COVID-19 pandemic is far from over — and not even close to slowing down — the Food and Drug Administration is already looking toward the next public health emergency.

The FDA has released a list of essential medications that hospitals should stock up on in the event of a public-health emergency. (Getty Images)
The FDA has released a list of essential medications that hospitals should stock up on in the event of a public-health emergency. (Getty Images)

On Friday, the FDA released a list of essential medications and “medical countermeasures” — which include drugs and devices for “a potential public health emergency stemming from a terrorist attack with a biological, chemical, or radiological/nuclear material, or a naturally occurring emerging disease” — that are needed to help combat future threats to public health.

The list, which contains 223 drug and biological product essential medicines and medical countermeasures, along with 96 medical devices, features items that the FDA has deemed “medically necessary to have available at all times in an amount adequate to serve patient needs and in the appropriate dosage forms,” according to a press release issued by the agency.

“Basically, it’s a list of meds and devices to cover many conditions so that hospitals will be fully prepared in the event that there are mass shortages,” Jamie Alan, an assistant professor of pharmacology and toxicology at Michigan State University, explains to Yahoo Life. “This would be something the pharmacy could cross-check to make sure they were stocked up on everything they need.”

The list was created on the prompting of an executive order issued in early August.

“The goal of this work is to ensure the American public is protected against outbreaks of emerging infectious diseases, such as COVID-19, as well as chemical, biological, radiological and nuclear threats,” the release says. “To accomplish this goal, the executive order seeks to ensure sufficient and reliable, long-term domestic production of these products, and to minimize potential shortages by reducing our dependence on foreign manufacturers of these products.”

Ventilators were among the nearly 100 essential medical devices included on the FDA's list, which largely covered medications. (Getty Images)
Ventilators were among the nearly 100 essential medical devices included on the FDA’s list, which largely covered medications. (Getty Images)

The medications identified are ones that are “most needed for patients in U.S. acute care medical facilities, which specialize in short-term treatment for severe injuries or illnesses, and urgent medical conditions,” the release says. The devices include diagnostic testing kits, supplies for rapid test development and processing, personal protective equipment, active vital-sign monitoring devices, devices for vaccine delivery and medical devices like ventilators.

According to the FDA, the items that made the list are what the agency anticipates “will be needed to respond to future pandemics, epidemics, and chemical, biological and radiological/nuclear threats.”

“When identifying essential medicines and medical countermeasures, we focused on including those that are medically necessary to have available in adequate supply which can be used for the widest populations to have the greatest potential impact on public health,” the release says.

The list will be used by federal partners to “carry out directives in the executive order, such as facilitating domestic production and increasing the domestic procurement of items on the

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Nexus Pharmaceuticals Receives FDA Approval for Succinylcholine Chloride Injection, USP

Nexus Pharmaceuticals announced it has received U.S. Food and Drug Administration (FDA) approval for Succinylcholine Chloride Injection, USP in 200mg/10mL Multiple-Dose Vials. It is an AP-rated generic to QUELICIN™¥.

“Nexus Pharmaceuticals is very pleased to announce the approval of Succinylcholine Chloride. This critical need drug has seen an increased demand due to the COVID-19 pandemic and Nexus will be ready to meet the challenge. This approval further cements Nexus’ commitment to alleviating the drug shortage crisis with sterile injectable products produced exclusively in the US and Europe,” said Mariam S. Darsot, CEO & President of Nexus Pharmaceuticals.

Nexus Pharmaceuticals’ Succinylcholine Chloride Injection will be available in cartons of 25 vials.

About Succinylcholine Chloride Injection, USP

Succinylcholine Chloride Injection, USP is a sterile, nonpyrogenic solution to be used as an ultra-short-acting, depolarizing, skeletal muscle relaxant. Succinylcholine is indicated as an adjunct to general anesthesia, to facilitate tracheal intubation, and to provide skeletal muscle relaxation during surgery or mechanical ventilation.

For prescribing information, please see the following link.

About Nexus Pharmaceuticals Inc.

Nexus Pharmaceuticals Inc., a US-based healthcare company, specializes in innovative processes to make difficult-to-manufacture specialty and generic drugs that are easier to use, less labor intensive, and more streamlined in practice. Nexus ensures that its high-quality FDA-approved drugs fulfill a critical unmet medical need and delivers dependable life-saving treatment options when and where they’re needed most.

¥QUELICIN is a registered trademark of Hospira, Inc., a wholly-owned subsidiary of Pfizer Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201029006301/en/

Contacts

Diana Lebiecki
847-996-3790
[email protected]

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How will a COVID vaccine be distributed once it’s approved by FDA?

Tens of millions of COVID-19 vaccine doses are reportedly being manufactured and stored in several facilities around the country. Pfizer and Moderna will allegedly have somewhere between 20 million and 40 million doses of their vaccine stockpiled by the end of December, Operation Warp Speed’s chief adviser, Dr. Moncef Slaoui, told ABC News last week.

Their vaccine candidates could also be two of the first to be granted emergency use authorization from the Food and Drug Administration, which they claim may come as soon as mid-December as both companies near completion of their late-stage clinical trials.

Distributing the first wave of doses will be a massive undertaking by both private and government entities alike, including the military. The current plan only lays out the first few months of distribution, with nursing homes and long-term care facilities slated to receive the first doses.

Private companies will deliver the vaccine, not the military

The military will likely handle logistics and not directly deliver vaccine vials, despite President Donald Trump’s recent claims. Instead, FedEx and other private delivery companies will take on the task.

“There will not be this vision that some people have that there’ll be Army trucks driving through the streets delivering vaccines,” Army Gen. Gustave Perna, chief operating officer of Operation Warp Speed, said at a press conference Tuesday. “That’s not feasible or the right way to do it.”

Operation Warp Speed is a program created to accelerate a vaccine rollout without sacrificing safety, the Trump administration said. It has pumped billions of dollars into numerous pharmaceutical companies in hopes of developing one or more safe and effective COVID-19 vaccines. The funding was initially intended to help ramp up development, but a recent deal with Walgreens and CVS aims to hasten and coordinate distribution as well.

‘We’re likely to get to every nursing home that signs up within 3 weeks’

Roughly a third of COVID-19 deaths in the U.S. have occurred among residents of long-term care facilities. CVS claims it’s prepared to begin administering COVID-19 vaccines in those facilities before the end of the year if one is approved.

As part of the recent deal between Operation Warp Speed, CVS and Walgreens, facilities that opt into the program will have pharmacists and technicians come and administer vaccine doses on-site — free of charge to residents and staff.

“Ultimately,

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First-Ever Treatment Shown to Reverse Alzheimer’s Earns NeuroEM Therapeutics FDA Breakthrough Device Status

The FDA is expediting a revolutionary clinical-stage head device to beat back Alzheimer’s disease at home

NeuroEM Therapeutics is poised to deliver a knockout blow to Alzheimer’s disease. The clinical-stage medical device company is pleased to announce FDA Breakthrough Device designation for its bioengineered head device shown to reverse Alzheimer’s memory loss in its pilot study, published last fall in the Journal of Alzheimer’s Disease.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201028005376/en/

Woman pictured wearing NeuroEM Therapeutics’ bioengineered MemorEM™ cap and arm control box. This revolutionary head device has earned FDA Breakthrough Device designation, having demonstrated in a pilot study that it can reverse Alzheimer’s disease. This woman’s participation in the study resulted in her regaining the equivalent of 12 months of memory. (Photo: Business Wire)

“No other drug or device has received this highest FDA designation to accelerate a treatment for Alzheimer’s disease,” said Dr. Robert Matthews, PhD, CEO of NeuroEM Therapeutics. “No other treatment has demonstrated the clinical results we have thus far, to not only slow but in fact reverse the progressive, debilitating loss of memory caused by this devastating brain disease.”

The FDA has given Breakthrough status to over 300 devices and over 100 drugs, but none to a therapeutic for Alzheimer’s disease – until now. NeuroEM’s head device (MemorEMTM) is the first-ever Alzheimer’s treatment (device or drug) to receive FDA Breakthrough status. The FDA program is designed to expedite the development and review process of new technology for patients with life-threatening or irreversibly debilitating conditions, such as Alzheimer’s Disease.

  • NeuroEM’s novel, proprietary technology, called Transcranial Electromagnetic Treatment (TEMT), achieves what other treatments for AD have not. In fact, no other therapy comes close to MemorEM’s remarkable results, made possible by its unique modes of action.

  • In just two months of daily use, the MemorEM restored on average the equivalent of 12 months of memory for 7 of 8 participants in its pilot study, none of whom experienced adverse side effects.

  • Treatment is easy, comfortable and safe. A patient wears what appears to be a simple cap that belies the complex biomedical engineering within it. The device is worn twice a day in home for an hour at a time, and its design allows the patient to move around home as usual.

NeuroEM has pioneered TEMT to successfully penetrate the skull to reach vital nerve cells in the brain called neurons. NeuroEM’s revolutionary TEMT technology works by breaking up small aggregates (oligomers) of two toxic proteins, called β-amyloid and tau, that are made inside the brain’s neurons during the Alzheimer’s disease process. These two toxic oligomers appear to be the root cause of Alzheimer’s disease by wreaking havoc on vital functions within neurons – especially in brain areas involved in memory. The electromagnetic waves emanating from multiple emitters within NeuroEM’s MemorEM head device easily penetrate the human brain and gently disassociate the toxic oligomers inside neurons resulting in a reversal of Alzheimer’s memory impairment in key

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Close to ‘exponential spread’ in some parts of the US, former FDA official says

The country is facing another cycle of the Covid-19 pandemic, and it may be the hardest yet, former Food and Drug Administration Commissioner Dr. Scott Gottlieb said Monday.



a car parked on a city street filled with lots of traffic: People wait in their cars at a newly opened mega drive-thru site at SISD Student Activities Complex on July 21, 2020 in El Paso, Texas. As coronavirus deaths surge past 4000 in Texas, overwhelmed hospitals are being forced to plan for extra refrigerated storage to hold deceased patients. (Photo by Cengiz Yar/Getty Images)


© Cengiz Yar/Getty Images
People wait in their cars at a newly opened mega drive-thru site at SISD Student Activities Complex on July 21, 2020 in El Paso, Texas. As coronavirus deaths surge past 4000 in Texas, overwhelmed hospitals are being forced to plan for extra refrigerated storage to hold deceased patients. (Photo by Cengiz Yar/Getty Images)

“I think we’re right now at the cusp of what’s going to be exponential spread in parts of the country,” Gottlieb said on CNBC’s “Squawk Box.”

“If we took aggressive steps right now, we could potentially forestall the worst of it, but we’re not going to do that,” because there’s a lot of fatigue and “policy resistance to taking strong action,” he said.

“We really have two or three months of the acute phase of this pandemic to get through,” he said. “This is going to be the hardest phase, probably.”

Worst number of cases yet

That’s as the country continues to report the most number of cases we’ve seen to date. The seven-day average of daily new cases reached an all-time high of 68,767 on Sunday, according to data from Johns Hopkins University. The previous record of 67,293 was set July 22.

“Unfortunately, I think the statement about ‘new record’ is going to be repeated over and over again in the days and weeks to come,” said Dr. Ashish Jha, dean of the Brown University School of Public Health.

“I expect that those numbers will continue to climb. Hospitalizations are going to continue to climb.”

The abysmal week was marked by the two worst days of daily new cases reported since the pandemic began. More than 83,000 new cases were reported both Friday and Saturday, according to Johns Hopkins.

To be clear: This surge reflects an onslaught of new infections — not just increased testing, contrary to what skeptics claim.

“You know why we have cases? Because we test so much,” President Donald Trump claimed at a rally Saturday in North Carolina. “And in many ways, it’s good. And in many ways, it’s foolish.”

But the seven-day average of new Covid-19 cases has soared 23% in the past week, according to Johns Hopkins data. The seven-day average of new tests performed has risen only 2.87% over the past week, according to the Covid Tracking Project.

And we are long past the point of just urban, heavily populated areas being the only places hit hard. South Dakota’s test positivity rate is 23%, the state’s health department said Monday. That means of every 100 people tested, 23 have been infected. The World Health Organization in May advised governments not to reopen until test positivity rates were 5% or lower for at least 14 days.

States to receive 36.7 million rapid tests

The federal government is shipping 36.7 million rapid Covid-19 tests, and states should be

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What patients want the FDA to consider about the role of AI in medicine

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US should consider national mask mandate for the winter, former FDA commissioner writes in op-ed

As the US reports its second-highest day of new Covid-19 cases amid the continuing fall surge, a former commissioner of the US Food and Drug Administration says it may be time for a national mask mandate.



Alice Arnold wearing a hat: Salt Lake County Health Department public health nurses look on during coronavirus testing outside the Salt Lake County Health Department Friday, Oct. 23, 2020, in Salt Lake City, Utah. (Rick Bowmer/AP)


© Rick Bowmer/AP
Salt Lake County Health Department public health nurses look on during coronavirus testing outside the Salt Lake County Health Department Friday, Oct. 23, 2020, in Salt Lake City, Utah. (Rick Bowmer/AP)

In an op-ed for The Wall Street Journal, Dr. Scott Gottlieb wrote the mandate could be “limited and temporary.”

“A mandate can be expressly limited to the next two months,” Gottlieb wrote, adding that it’s easier to wear a mask in the winter than the summer. “The inconvenience would allow the country to preserve health-care capacity and keep more schools and businesses open.”

With deaths expected to rise this winter, policymakers will have to make moves to slow the spread, Gottlieb wrote. There already is no support for reinstating the stay-at-home orders from the spring.

If 95% of Americans wore masks in public, more than 100,000 lives could be saved in the United States through February, according to data released Friday by the Institute for Health Metrics and Evaluation at the University of Washington.

“If people are not wearing masks, then maybe we should be mandating it,” Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, told CNN’s Erin Burnett on Friday.

Gottlieb wrote the concern about needing fines to enforce the mandate leading to confrontations with police isn’t necessarily true.

“States should be able to choose how to enforce a mandate, but the goal should be to make masks a social and cultural norm, not a political statement,” he wrote. “Mandating masks has become divisive only because it was framed that way by some politicians and commentators.”

Gottlieb was appointed FDA commissioner by President Trump and served from May 2017 to May 2019. He is now a resident fellow at the American Enterprise Institute, a think tank in Washington, DC.

Saturday saw 83,718 new Covid-19 cases, just 39 cases shy of the all-time record that was reported Friday. Already, national cases total more than 8.6 million and 225,212 people have died, according to Johns Hopkins.

“We’re at a dangerous tipping point right now,” Gottlieb told Margaret Brennan Sunday on CBS’s “Face the Nation.” “We’re entering what’s going to be the steep slope of the curve, of the epidemic curve.”

Social gatherings and family events moving indoors to avoid the colder weather is largely to blame for the high rates of spread, officials said over the weekend.

In Maryland, the governor said this week family gatherings were the No. 1 source of transmission in the state, followed by house parties. In North Carolina, health officials reported its highest daily case count Friday and said they continue to see clusters “from social and religious gatherings.”

At least 35 states report rise in cases

The Florida Department of Health on Sunday reported 2,385 additional coronavirus cases and

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