Good Day BIO: New FDA commissioner puts animals on agenda

Beginning the short week with what the newly confirmed FDA Commissioner said about One Health, why CMS “ambushed” efforts to combat pediatric Alzheimer’s, and the start of Rare Disease Week. (886 words, 4 minutes, 25 seconds)

New FDA commissioner puts animals on his agenda

FDA Commissioner Dr. Robert Califf’s first public statements after his confirmation acknowledge the close relationship between animal and human health and the need for technology to address this challenge.

What he said: “One of the greatest areas of learning for me during my last time at FDA was the importance of veterinary products and the critical issues in One Health that are at the core of antimicrobial resistance and future pandemics,” Dr. Califf wrote Thursday in a series of tweets about his priorities, which also include the need to address the pandemic, opioids, and misinformation.

Confirmation of the FDA commissioner was essential to updating animal feed regulations, U.S. Secretary of Agriculture Tom Vilsack told a congressional hearing last month. Feed additives that reduce greenhouse gas emissions from livestock require costly, time-consuming approval because the FDA treats them as a drug. Vilsack said other countries have faster approval and gain “a market advantage.” 

“We do need to modernize our regulatory process as it relates to those kinds of feed additives,” Vilsack said during the hearing.

The FDA needs to join USDA’s effort to modernize its approach to animal biotech, BIO urged last week in a letter to the Biden administration. Gene editing animals can help address climate change, sustainable production of healthier food, and zoonotic disease, the letter said.

A One Health approach can address diseases that jump species, like COVID-19. One Health “recognizes that the health of people is closely connected to the health of animals and our shared environment,” according to the CDC.  

Read: Stopping Outbreaks Through One Health: Making Tomorrow's Breakthroughs Possible 

Birds with avian flu recently sparked concerns about a zoonotic risk, the CDC said. Gene editing is being used to make chickens resistant to bird flu, as we’ve reported.

More Agriculture and Environment News:

USDA: United States advances agricultural innovation at inaugural AIM for Climate Ministerial
“Agriculture Secretary Tom Vilsack underscored the importance of joint international action and investment in climate-smart agriculture and food systems innovation, calling on AIM for Climate partners to continue on their ambitious path towards addressing global climate change and hunger challenges.” (BIO is a non-governmental Knowledge Partner of AIM.)

CMS ‘ambushed’ effort to combat fatal pediatric disease

The head of a small biopharma developing a drug for a rare and fatal pediatric condition says his company has been “ambushed” by the Centers for Medicare Services (CMS) decision to “second-guess the FDA” and limit coverage for a new Alzheimer’s drug.

ICYMI: The FDA recently granted accelerated approval for the first disease-modifying treatment for Alzheimer’s—but in an unprecedented move, CMS proposed to restrict coverage for the drug to patients participating in clinical trials. 

“This overreaching by CMS has the potential to dramatically stifle innovation in new treatments,” writes Allievex CEO Thomas P. Mathers, in an op-ed in the Sunday Boston Herald. Allievex is developing the first treatment for Sanfilippo Syndrome, known as “pediatric Alzheimer’s.” 

Allievex is “working around-the-clock” to gain accelerated approval—but by rejecting the “gold standard” of the FDA, CMS “injected unnecessary bureaucratic uncertainty into an already arduous process,” Mathers writes. 

“If CMS can impugn the FDA, then the ability of small biopharma companies to raise capital is jeopardized,” he continues. “Companies will struggle to raise funding for their research projects. Patients will lose out on lifesaving treatments.” 

Read the whole thing.

“This is the first time this has ever happened for a drug for an on-label indication,” former CMS Administrator and FDA Commissioner Dr. Mark McClellan said during BIO’s CEO & Investor Conference—read more.

More Health Care News:

STAT News: STATUS List 2022
STAT News recognizes 46 leaders and influencers in the life sciences, including BIO President and CEO Dr. Michelle McMurry-Heath and several BIO member company CEOs.

Do you have questions about FDA regulatory programs and Emergency Use Authorizations? On Thursday, February 24, join BIO’s Leslie Harden and Alex May for a free webinar hosted by the Montana Bioscience Alliance, the ASCEND Hub, and the Montana Bioscience Cluster Initiative—get the details and register.

President Biden’s Tuesday: The White House has been preparing a response to an impending Russian invasion of Ukraine, The New York Times reports. A virtual event to announce critical mineral supply chains, powering clean energy manufacturing, and creating good-paying jobs—with several Cabinet members and California Gov. Gavin Newsom—is scheduled for 3:30 PM ET, though the situation in Ukraine could change Biden’s schedule. 

What’s Happening on Capitol Hill: Recess.