In an executive order signed Monday, President Joe Biden announced steps by his administration to bolster the “bioeconomy” in the United States, a classification that covers research and development across a broad swath of products, including medical supplies, sustainable new fuels and food, as well as technologies meant to help fight climate change.

The order comes barely a month after Biden signed a major piece of legislation, the CHIPS Act, meant to supercharge U.S. manufacturing of semiconductors, an area in which the U.S. has lost its once-dominant global position.

The effort to boost the U.S. biotech sector further underscores the administration’s apparent belief that deeper engagement by the federal government with domestic manufacturing operations is necessary to preserve U.S. competitiveness in the global economy.

In a briefing over the weekend, administration officials made it clear that the administration’s push to bring more U.S.-based biotechnology manufacturing online comes as a response to other countries — particularly China — investing heavily in the sector.

Administration officials stressed that biotechnology-based products and “biomanufacturing” present a promising alternative to many current products — fuel, plastics and other materials — that are currently made using the byproducts of carbon-laden petrochemicals.

Order text

The executive order reads, in part, “It is the policy of my Administration to coordinate a whole-of-government approach to advance biotechnology and biomanufacturing towards innovative solutions in health, climate change, energy, food security, agriculture, supply chain resilience, and national and economic security.”

It continues, “Central to this policy and its outcomes are principles of equity, ethics, safety, and security that enable access to technologies, processes, and products in a manner that benefits all Americans and the global community and that maintains United States technological leadership and economic competitiveness.”

Caution urged

Among the Biden administration’s promises in the executive order is a vow to “substitute fragile supply chains from abroad with strong chains at home.” But not everyone agrees that a government effort to manipulate the supply chain is the smartest strategy for the long run.

“Government can play a role in funding basic research, university labs and the rest, but when it starts micromanaging supply chains, you end up with more fragile supply chains than robust ones,” Scott Lincicome, a director for general economics and trade at the Libertarian-leaning Cato Institute, told VOA.

Similarly, he said, government decisions to privilege “onshore” production over foreign producers can be dangerous.

“There’s nothing wrong with domestic manufacturing, but as we’ve learned throughout the pandemic, there’s a big problem with putting all of your eggs in one basket, either all foreign or all domestic,” Lincicome said.

“While onshoring can insulate you from foreign shocks, it makes you far more vulnerable to domestic shocks,” he added. “And in the process, it makes you poorer and weaker overall. The best approach is to have a very open, diverse global supply chain with domestic networks, foreign suppliers, and a very light government touch on trade, investment, talent and the rest.”

Multiple aims

The executive order lays out a number of areas in which the Biden administration plans to flex the federal government’s muscle, including the domestic manufacturing of biotechnology products. The aim is to encourage both the creation of domestic manufacturing facilities, as well as the supply chains of fuel and raw materials needed to operate them.

The administration also promises to help create markets for biotechnology products by increasing mandatory purchasing requirements for federal agencies.

In addition, the executive order proposes to push more funding into research and development and to provide innovators support in the form of federal data that helps identify unmet needs. Other efforts will include job training programs, streamlined regulatory approval of new products, and cooperative programs with international partners.

Mending fences

The administration’s push to help U.S. biotechnology firms could go some way toward mending fences with the industry, which was angered by elements of the Inflation Reduction Act (IRA), which Biden recently signed into law.

The IRA, for the first time, allows Medicare, the government health insurance program for senior citizens, to bargain with pharmaceutical firms over the prices of some prescription drugs. Many in the industry strongly opposed the measure, claiming it would reduce incentives to innovate.

The executive order comes less than a week after the Biotechnology Innovation Organization sent a letter to the administration requesting it to “take additional steps to foster the development and deployment of pioneering technologies that will further reduce greenhouse gas emissions in manufacturing, transportation, and agricultural supply chains to build a stronger, more resilient, and environmentally sustainable economy.”

Biden signed the order before traveling to Boston, where he was scheduled to tout the results of the infrastructure bill he signed last year, which pumped federal money into a wide array of construction projects.

Also on Monday, Biden named Renee Wegrzyn, a biotechnology executive, to head the new Advanced Research Projects Agency for Health. The announcement came as part of a discussion of Biden’s “moonshot” initiative to drive new research on treatments for cancer.
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President Joe Biden on Monday urged Americans to come together for a new “national purpose” — his administration’s effort to end cancer “as we know it.”

At the John F. Kennedy Presidential Library and Museum, Biden channeled JFK’s famed moonshot speech 60 years ago, likening the space race to his own effort and hoping it, too, would galvanize Americans.

“He established a national purpose that could rally the American people and a common cause,” Biden said of Kennedy’s space effort, adding that “we can usher in the same unwillingness to postpone.”

Biden hopes to move the U.S. closer to the goal he set in February of cutting U.S. cancer fatalities by 50% over the next 25 years and to dramatically improve the lives of caregivers and those suffering from cancer. Experts say the objective is attainable — but with adequate investments.

The president called his goal of developing treatments and therapeutics for cancers “bold, ambitious, and I might add, completely doable.”

In his speech, Biden called on the private sector to make drugs more affordable and data more regularly available. He ticked off medical advancements possible with focused research, funding and data.

And he spoke of a new federally backed study that seeks evidence for using blood tests to screen against multiple cancers — a potential game-changer in diagnostic testing to dramatically improve early detection of cancers.

Danielle Carnival, the White House coordinator for the effort, told The Associated Press that the administration sees huge potential in the commencement of the blood diagnostic study on identifying cancers.

“One of the most promising technologies has been the development of blood tests that offer the promise of detecting multiple cancers in a single blood test and really imagining the impact that could have on our ability to detect cancer early and in a more equitable way,” Carnival said. “We think the best way to get us to the place where those are realized is to really test out the technologies we have today and see what works and what really has an impact on extending lives.”

In 2022, the American Cancer Society estimates, 1.9 million new cancer cases will be diagnosed and 609,360 people will die of cancer diseases. The Centers for Disease Control and Prevention rank cancer as the second-highest killer of people in the U.S. after heart disease.

The issue is personal to Biden, who lost his adult son Beau in 2015 to brain cancer. After Beau’s death, Congress passed the 21st Century Cures Act, which dedicated $1.8 billion over seven years for cancer research and was signed into law in 2016 by President Barack Obama.

Obama designated Biden, then vice president, to run “mission control” on directing the cancer funds as a recognition of Biden’s grief as a parent and desire to do something about it. Biden wrote in his memoir Promise Me, Dad that he chose not to run for president in 2016 primarily because of Beau’s death.

Despite Biden’s attempts to hark back to Kennedy and his space program, the current initiative lacks that same level of budgetary support. The Apollo program garnered massive public investment — more than $20 billion, or more than $220 billion in 2022 dollars adjusted for inflation. Biden’s effort is far more modest and reliant on private sector investment.

Still, he’s tried to maintain momentum for investments in public health research, including championing the Advanced Research Projects Agency for Health (ARPA-H), modeled after similar research and development initiatives benefiting the Pentagon and intelligence community.

On Monday, Biden announced Renee Wegrzyn as the inaugural director of ARPA-H, which has been given the task of studying treatments and potential cures for cancers, Alzheimer’s, diabetes and other diseases. He also announced a new National Cancer Institute scholars’ program to provide funding to early-career scientists studying treatments and cures for cancer, with a focus on underrepresented groups and those from diverse backgrounds.

The president was joined by Caroline Kennedy, the daughter of JFK who is now the U.S. ambassador to Australia. And he was expected to speak later in the day at a fundraiser for the Democratic National Committee.

Experts agree it’s far too early to say whether these new blood tests for finding cancer in healthy people will have any effect on cancer deaths. There have been no studies to show they reduce the risk of dying from cancer. Still, they say setting an ambitious goal is important.

Carnival said the National Cancer Institute study was designed so that any promising diagnostic results could be swiftly put into widespread practice while the longer-term study — expected to last up to a decade — progresses. She said the goal was to move closer to a future where cancers could be detected through routine bloodwork, potentially reducing the need for more invasive and burdensome procedures like colonoscopies, and therefore saving lives.

Scientists now understand that cancer is not a single disease, but hundreds of diseases that respond differently to different treatments. Some cancers have biomarkers that can be targeted by existing drugs that will slow a tumor’s growth. Many more targets await discovery.

“How do we learn what therapies are effective in which subtypes of disease? That to me is oceanic,” said Donald A. Berry, a biostatistician at the University of Texas M.D. Anderson Cancer Center. “The possibilities are enormous. The challenges are enormous.”

Despite the challenges, he’s optimistic about cutting the cancer death rate in half over the next 25 years.

“We can get to that 50% goal by slowing the disease sufficiently across the various cancers without curing anybody,” Berry said. “If I were to bet on whether we will achieve this 50% reduction, I would bet yes.”

Even without new breakthroughs, progress can be made by making care more equitable, said Dr. Crystal Denlinger, chief scientific officer for the National Comprehensive Cancer Network, a group of elite cancer centers.

And any effort to reduce the cancer death rate will need to focus on the biggest cancer killer, which is lung cancer. Mostly attributable to smoking, lung cancer now causes more cancer deaths than any other cancer. Of the 1,670 daily cancer deaths in the United States, more than 350 are from lung cancer.

Dr. Michael Hassett of the Dana-Farber Cancer Institute in Boston said Biden’s goal to reduce cancer deaths could be met by following two parallel paths: one of discovery and the other making sure as many people as possible are reaping the advantages of existing therapies and preventive approaches.

“If we can address both aspects, both challenges, major advances are possible,” Hassett said.
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The European Medicines Agency (EMA) on Monday recommended a COVID-19 booster designed to combat the currently circulating Omicron BA.4/5 subvariants, days after endorsing a pair of boosters tailored to target the older BA.1 Omicron variant.

The latest recommendation is for a so-called bivalent vaccine developed by Pfizer PFE.N and BioNTech 22UAy.DE, which targets BA.4/5 as well as the strain of the virus that originally emerged in China in December 2019 targeted by earlier COVID vaccines.

The EMA recommendation is to authorize the retooled booster shots for people aged 12 and above who have received at least primary vaccination against COVID. The final go-ahead will be subject to European Commission approval, which is expected to come shortly.

If authorized, the BA.4/5-tailored booster will be available in days to all 27 EU member states, Pfizer said in a statement on Monday.

While existing coronavirus vaccines provide good protection against hospitalization and death, their effectiveness, particularly against infection, was reduced as the virus evolved.

Earlier this month, the EMA endorsed both Pfizer-BioNTech and Moderna’s MRNA.O vaccines updated for BA.1.

EU officials signaled in recent months they were open to initially using boosters targeting the older BA.1 variant, given those specifically targeting the newer, now dominant Omicron BA.4/5 offshoots are further behind in development.

In contrast, the U.S. Food and Drug Administration insisted it was only interested in vaccines targeting BA.4/5. Last week, Pfizer-BioNTech and Moderna secured U.S. authorization for those despite limited available clinical data.

Given BA.1’s earlier emergence, data from human trials testing those redesigned vaccines has been submitted to EU regulators. For the BA.4/5 adapted vaccines, regulatory submissions are largely based on lab and animal studies.

Using animal and lab data to solicit regulatory approval is done regularly for flu vaccines that are revamped each year to combat the latest circulating strains.

On Monday, the EMA said its backing of the Pfizer-BioNTech updated BA.4/5 shot relied partly on data from human clinical trials available on the companies’ BA.1-tailored vaccine.

A clinical trial testing the Pfizer-BioNTech BA.4/5 vaccine in humans was initiated in early September, and data should be available later this autumn. Meanwhile, human trial data on Moderna’s BA.4/5 shot is expected by later this month or early October.

EU officials have encouraged member states to roll out boosters of the established original vaccines and the bivalent shots — whatever is readily available — for the vulnerable and elderly following a rise in summer infections, as protection waned due to the domination of BA.4 and especially BA.5.

Uptake could be limited, as people have become less worried about the disease, thanks in large part to the success of the first generation of shots. Experts also worry that the public may be suffering from vaccine fatigue and less likely to seek the boosters, which could be a fourth or fifth COVID shot for some.
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Jeff Bezos’ rocket company suffered its first launch failure Monday. No one was aboard, only science experiments. 

The Blue Origin rocket veered off course over West Texas about a minute after liftoff. The capsule’s launch abort system immediately kicked in, lifting the craft off the top. Several minutes later, the capsule parachuted onto the remote desert floor. 

Blue Origin’s launch commentary went silent when the capsule catapulted off the rocket, later announcing: “It appears we’ve experienced an anomaly with today’s flight. This wasn’t planned.” 

The mishap occurred as the rocket was traveling nearly 700 mph (1,126 kph) at an altitude of about 28,000 feet (8,500 meters). There was no video shown of the rocket — only the capsule — after the failure occurred. The rocket usually lands upright on the desert floor and then is recycled for future flights; clearly, that did not happen this time. 

Launch commentator Erika Wagner said the capsule managed to escape successfully, with the webcast showing it reaching a maximum altitude of more than 37,000 feet (11,300 meters). Thirty-six experiments were on board, half sponsored by NASA. 

“Booster failure on today’s uncrewed flight. Escape system performed as designed,” the company tweeted later. 

No further details were provided. 

It was the 23rd flight for the New Shepard program, named after the first American in space, Mercury astronaut Alan Shepard. The same kind of rocket and capsule have been used to carry paying passengers on 10-minute rides to the edge of space. It was the ninth flight for this rocket. 

Its most recent passenger flight was just last month. Bezos was on the first New Shepard crew last summer. Altogether, Blue Origin has carried 31 people to the edge of space. The company’s headquarters is in Kent, Washington. 

 
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When Emmanuel Obeng-Dankwa is worried about making rent on his New York City apartment, he sometimes holds off on filling his blood pressure medication. 

“If there’s no money, I prefer to skip the medication to being homeless,” said Obeng-Dankwa, a 58-year-old security guard. 

He is among a majority of adults in the U.S. who say that health care is not handled well in the country, according to a new poll from The Associated Press-NORC Center for Public Affairs Research.

The poll reveals that public satisfaction with the U.S. health care system is remarkably low, with fewer than half of Americans saying it is generally handled well. Only 12% say it is handled extremely or very well. Americans have similar views about health care for older adults. 

Overall, the public gives even lower marks for how prescription drug costs, the quality of care at nursing homes and mental health care are being handled, with just 6% or less saying those health services are done very well in the country. 

“Navigating the American health care system is exceedingly frustrating,” said A. Mark Fendrick, the director of the University of Michigan Center for Value-Based Insurance Design. “The COVID pandemic has only made it worse.” 

More than two years after the pandemic’s start, health care worker burnout and staffing shortages are plaguing hospitals around the country. And Americans are still having trouble getting in-person medical care after health centers introduced restrictions as COVID-19 killed and sickened millions of people around the country, Fendrick said. 

In fact, the poll shows an overwhelming majority of Americans, nearly 8 in 10, say they are at least moderately concerned about getting access to quality health care when they need it.

Black and Hispanic adults in particular are resoundingly worried about health care access, with nearly 6 in 10 saying they are very or extremely concerned about getting good care. Fewer than half of white adults, 44%, expressed the same level of worry. 

Racial disparities have long troubled America’s health care system. They have been abundantly clear during the COVID-19 pandemic, with Black and Hispanic people dying disproportionately from the virus. Black and Hispanic men also make up a disproportionately high rate of recent monkeypox infections.

Fifty-three percent of women said they are extremely or very concerned about obtaining quality care, compared to 42% of men. 

While Americans are united in their dissatisfaction with the health care system, that agreement dissolves when it comes to solutions to fix it. 

About two-thirds of adults think it is the federal government’s responsibility to make sure all Americans have health care coverage, with adults ages 18 to 49 more likely than those over 50 to hold that view. The percentage of people who believe health care coverage is a government responsibility has risen in recent years, ticking up from 57% in 2019 and 62% in 2017. 

Still, there’s not consensus on how that coverage might be delivered. 

About 4 in 10 Americans say they support a single-payer health care system that would require Americans to get their health insurance from a government plan. More, 58%, say they favor a government health insurance plan that anyone can purchase. 

There also is broad support for policies that would help Americans pay for the costs of long-term care, including a government-administered insurance plan similar to Medicare, the federal government’s health insurance for people 65 or older.

Retired nurse Pennie Wright, of Camden, Tennessee, doesn’t like the idea of a government-run health care system. 

After switching to Medicare this year, she was surprised to walk out of her annual well-woman visit, once fully covered by her private insurance plan, with a $200 bill. 

She prefers the flexibility she had on her private insurance plan. 

“I feel like we have the best health care system in the world, we have a choice of where we want to go,” Wright said. 

A majority of Americans, roughly two-thirds, were happy to see the government step in to provide free COVID-19 testing, vaccines and treatment. Roughly 2 in 10 were neutral about the government’s response. 

The government’s funding for free COVID-19 tests dried up at the beginning of the month. And while the White House says the latest batch of recommended COVID-19 boosters will be free to anyone who wants one, it doesn’t have money on hand to buy any future rounds of booster shots for every American. 

Eighty percent say they support the federal government negotiating for lower drug prices. President Joe Biden this summer signed a landmark bill into law allowing Medicare to negotiate the price of prescription drugs. The move is expected to save taxpayers as much as $100 billion over the next decade. 

“Medication costs should be low, to the minimum so that everyone can afford it,” said Obeng-Dankwa, the Bronx renter who has trouble paying for his medication. “Those who are poor should be able to get all the necessary health they need, in the same way someone who also has the money to pay for it.”
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New York Governor Kathy Hochul declared a disaster emergency Friday in a bid to accelerate efforts to vaccinate residents against polio after the virus was detected in wastewater samples taken in four counties. 

Hochul’s executive order followed the discovery of the virus last month in samples from Long Island’s Nassau County, bordering the New York City borough of Queens. Earlier this year the virus was found in samples from Rockland, Orange and Sullivan counties, all north of the city.  

In July, the first confirmed case of polio in the United States in nearly a decade turned up in an adult in Rockland County, according to the state health department. 

“On polio, we simply cannot roll the dice,” State Health Commissioner Mary Bassett said in a statement. “If you or your child are unvaccinated or not up to date with vaccinations, the risk of paralytic disease is real.” 

Polio can cause irreversible paralysis in some cases, but it can be prevented by a vaccine first made available in 1955. While there is no known cure, three injections of the vaccine provide nearly 100% immunity. 

People of all ages are under threat, though the virus primarily affects children age 3 and younger.  

Officials urged inoculations for unvaccinated adults and children as young as 2 months, and they advised that vaccinated people receive a lifetime booster dose. 

Hochul’s declaration authorizes paramedics, midwives and pharmacists to administer polio vaccinations, among other steps, to accelerate inoculation rates. The order also directs health care providers to update the state with data on immunizations. 

The state of emergency will stay in effect until October 9. Health officials set a goal of getting 90% of residents vaccinated. 

The state health department warned that people in New York City and Rockland, Orange, Sullivan and Nassau counties are at the highest risk.  

Orange County has the lowest vaccination rate of the counties of concern with less than 59% being immunized, according to the state health department. 
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NASA’s moon mission suffers another setback, this time delaying a test launch for at least a few weeks. Plus, a look back at the first color images of Mars, and we wish a very happy birthday to a TV space pioneer. VOA’s Arash Arabasadi brings us The Week in Space.  
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The introduction of a nationwide mental health hotline has increased the numbers of people calling for help in the United States and the need for more counselors to answer their calls. For VOA, Svitlana Prystynska has this story from the Western state of Colorado. Camera – Vladimir Petruniv.
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The World Health Organization is warning of a resurgence of deadly meningitis in Africa because COVID-19 has disrupted lifesaving vaccination campaigns.

The near elimination of the deadly form of meningitis type A in Africa is one of the continent’s biggest health success stories. Over the last 12 years, about 350 million Africans have received a single dose of MenAfriVac, a vaccine designed specifically for the African meningitis belt.  

The WHO regional director for Africa, Matshidiso Moeti, said not a single case of meningitis Group A has been reported on the continent in the past five years.

“Now, however, the COVID-19 pandemic has delayed vaccination campaigns targeting more than 50 million African children, raising the risk that these gains will be reversed,” she said. “In addition, major outbreaks caused by meningitis Group C have been recorded in seven of the African Sub-Saharan meningitis belt countries in the past nine years.”

Moeti noted a four-month outbreak last year in the Democratic Republic of Congo claimed more than 200 lives.  

Francois Marc Laforce, director of technical services for the Serum Institute of India, played a pivotal role in the development of the MenAfriVac vaccine at the Serum Institute nearly two decades ago. He said that besides meningitis Group C, Africa currently is contending with residual outbreaks of other forms of meningitis.

“A new vaccine again specifically designed for the African meningitis belt will, hopefully, be prequalified later this year or early next year,” he said. But this vaccine holds the promise of finishing what MenAfriVac began, such that Africa may be the first continent to be free of meningitis epidemics.”  

Meningitis is caused by inflammation of the membranes that surround the brain and spinal cord. Acute bacterial meningitis can cause death within 24 hours.  Young children are most at risk. About half the cases and deaths occur in children under age five.

The WHO launched a new strategy Thursday to defeat bacterial meningitis in the African region by 2030. The plan calls for shoring up diagnosis, surveillance, care, and vaccination. The WHO estimates $1.5 billion will be needed to implement the plan between now and 2030.
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A new malaria vaccine developed by Britain’s University of Oxford is 80% effective in preventing infection, according to trial results published Thursday in The Lancet medical journal. Scientists say it represents a huge breakthrough that has the potential to save millions of lives and eventually eradicate the disease.

The vaccine, named R21/Matrix-M, had already shown encouraging trial results after three primary doses. Maintaining that immunity has always been a big challenge and the latest trial shows that a booster dose is effective, explained Professor Adrian Hill, the director of the Jenner Institute at the University of Oxford and co-author of The Lancet paper. 

“The technology has been complex to develop because we need very strong antibody responses to get protection against malaria and those antibodies, like all responses, decline over time,” Hill told VOA. 

“One of the worries was that this would be short-term protection and only last for a few months. That’s definitely not the case with the data we’re releasing today,” Hill said. “And indeed, 80% efficacy in the second year of follow up after a booster dose is really very encouraging in that respect.” 

The latest phase II trial involved 450 children between 5 and 17 months old, recruited from the Nanoro region in Burkina Faso. The results show a higher strength booster dose was 80% effective in preventing malaria infection. No serious side effects were seen. 

“This is a parasite we’re trying to vaccinate against. It’s not a virus. It’s got thousands of genes. [So it’s] complex to design a vaccine,” Hill said. “Over 100 have been in clinical trials and this looks like the best data so far. So we’re excited.” 

The Serum Institute of India, which has produced billions of doses of the Oxford-AstraZeneca COVID-19 vaccine, is producing the R21/Matrix-M malaria vaccine. It has signed an agreement to rapidly scale up production if the vaccine receives World Health Organization (WHO) approval in coming months. 

“We’re trying to do something similar with malaria, to produce a low-cost vaccine — a few dollars a dose — and to manufacture that really upscale so we can get 100 million doses or more out there as soon as possible,” Hill told VOA. 

Malaria killed an estimated 627,000 people in 2020, the most recent available data. The majority are children under five years old in sub-Saharan Africa. 

The WHO approved the first-ever malaria vaccine in October of last year, called RTS,S. But R21 may offer even greater hope, said Hill. 

“A vaccine with high efficacy like this should be able to save hundreds of thousands of lives a year and ultimately millions of lives over the next decade or so,” he said. “After we get a vaccine rolled out for that very vulnerable population, we’ll be looking at a travelers’ vaccine and then we’ll be looking at a vaccine that could actually eliminate regionally and eradicate globally this terrible disease.” 

Results from the ongoing phase III trial involving 4,800 children are due later this year and it’s hoped that mass production and rollout can begin in 2023, following WHO approval. 
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A panel of federal health advisers voted Wednesday to recommend approval for an experimental drug to treat Lou Gehrig’s disease, a remarkable turnaround for the much-debated medication that was previously rejected by the same group earlier this year.

The Food and Drug Administration advisers voted 7-2 that data from Amylyx Pharma warranted approval, despite hours of debate about the strength and reliability of the company’s lone study. The FDA is not required to follow the group’s advice, but its positive recommendation suggests an approval is likely later this month.

The FDA has approved only two therapies for the disease, amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions like walking, talking and swallowing.

Patients support drug

ALS patients and their families have rallied behind Amylyx’s drug, launching an aggressive lobbying campaign and enlisting members of Congress to push the FDA to grant approval.

Despite a negative review published by FDA’s internal scientists ahead of the meeting, a majority of the outside panelists said Amylyx had presented enough evidence to suggest the drug is helping patients live longer. The same group of neurology experts narrowly voted against the drug in March, because of concerns about missing data and other issues in the company’s study.

“To deprive ALS patients of a drug that might work, it’s probably not something I would feel terribly comfortable with,” said Dr. Liana Apostolova of Indiana University’s School of Medicine, who voted for approval. “At the previous meeting it wasn’t that clear and it’s still questionable.”

Amylyx also appeared to benefit from an unusual exchange in which a company executive — at the FDA’s request — committed to pull the drug from the market if its benefits aren’t confirmed by a large, ongoing study.

The FDA has the power to force companies to pull drugs from the market, though it’s generally faster if drugmakers voluntarily take that step. In cases where companies resist removal the regulatory process can drag on for years.

New data prompts second look

Wednesday’s vote concluded a rare second meeting to review several new statistical analyses submitted by Amylyx in support of the treatment’s benefit in slowing disease and extending life.

The ALS drug review is being closely watched as an indicator of FDA’s flexibility in reviewing experimental medications for the terminally ill and its ability to withstand outside pressure.

Amylyx conducted one small, mid-stage trial of its drug that showed some benefit in slowing the disease, but it was plagued by missing data and other problems, according to FDA reviewers.

“The final result — for a single study — is borderline and not very statistically persuasive,” FDA statistician Tristan Massie told panelists.

Company says data shows drug extends life

The Cambridge, Massachusetts, company says follow-up data gathered after the study concluded showed the drug extended life. Patients who continued taking the drug survived about 10 months longer than patients who never took the drug, according to a new company analysis.

Panelists favoring the drug cited that data, along with the drug’s mild side effects, to suggest there would be little downside for patients even if it doesn’t ultimately slow ALS.

Hanging over the review is FDA’s controversial approval of the Alzheimer’s drug Aduhelm last year, which was reviewed by the same agency scientists and outside advisers.

In that case, the FDA disregarded the overwhelmingly negative vote by its outside advisers, three of whom resigned over the decision. The agency’s approval — which followed irregular meetings with drugmaker Biogen — is under investigation by Congress and federal inspectors.
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