FDA prepares for controversial ALS drug review, denies approval for EpiPen alternative

The ALS drug NurOwn is part of a broader debate over how to balance the needs of patients against the rigors of scientific review.

September 21, 2023 Check out STAT+

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By Usha Lee McFarling National Science Correspondent

Good morning. Usha Lee McFarling here, filling in for Liz Cooney with armfuls of news: The story of why a nasal spray for allergic reaction won't be in pharmacies just yet, the medical limbo faced by people who aren't yet sick but carry genes for severe disease, more free Covid tests, and one reason I'm glad I gave up drinking Diet Coke.

nHealth No new nasal spray for people with anaphylaxis Courtesy ARS Pharma In what came as a surprise to drugmaker ARS Pharma, FDA officials Tuesday refused to approve neffy — a nasal spray alternative to injectable epinephrine products like EpiPen used for anaphylaxis. As STAT's Isa Cueto reported, many see neffy as a cheaper and easier to administer alternative — and one that would not be subject to shortages as injectable drugs have been.  But the FDA, wanting to see both more data on whether neffy works as well as EpiPens and more evidence of safety, asked for a study comparing repeat doses of neffy with repeat doses of injectable epinephrine. ARS plans to resubmit early next year with hopes of gaining approval by year's end. The company said their spray, which even children can self-administer, could be in pharmacies within eight weeks of approval. In a follow-up article, STAT's Annalisa Merelli looks at the role of "citizen's petitions" like the one filed by EpiPen maker Viatris in response to neffy's application for approval. Created in the '70s as a vehicle for Americans to share concerns with the agency, experts say the petitions appear to have evolved into a weapon pharmaceutical companies can wield against rivals with new products or those hoping to get cheaper generic formulations approved. HEALTH The surprising tie between gentrification and firearm injury Firearm injury, which claims 45,000 lives each year, is associated with lower-income neighborhoods — but not all lower-income neighborhoods. A study published Wednesday in JAMA Surgery found one risk factor that may surprise you: gentrification. The analysis of injuries that occurred between 2010 and 2019 showed that when wealthier people moved into a formerly low-income neighborhood, the risk of firearm injury was 26% greater than neighborhoods with similar economic status that were not undergoing gentrification.  The cause of the increased violence? The researchers suggest it could be social disruption and stress experienced by residents who'd long lived in neighborhoods that experienced disinvestment and system racial segregation — but were now being displaced to other low-income neighborhoods because of higher housing costs. While the violence eventually decreased once higher-income residents predominated, the researchers said the finding was evidence that violence prevention programs should be targeted to areas undergoing or projected to undergo gentrification. Closer LOOK  Hard questions about what to do for ALS patients — and those who carry its genes Constanza Hevia for STAT Despite facing a grim future, people carrying genes for ALS might not get sick for decades —  and there are few resources or hope for this in-limbo population. As STAT's Jason Mast writes, a group of such people, along with neurologists, advocates, and biotech leaders, are meeting this week in Philadelphia to "grapple with a central tension of modern medicine": What does medical science owe to carriers for grave diseases?  Options include monitoring carriers more closely to learn how the disease progresses and running clinical trials for symptomless people to find ways to prevent the disease. Much of the credit for sparking these important discussions goes to ALS carrier Jean Swidler (pictured above), a blunt and tireless advocate who Jason profiles in his story. In a related story, STAT's Damian Garde tells us an FDA panel will weigh in next week on NurOwn, a stem cell therapy with the potential to slow ALS progression. Some patients say the drug should be approved because people with ALS currently have little hope of stopping their incurable and fatal disease. Others say approval should be out of the question because the drug has not shown significant clinical efficacy in trials.

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IN THE LAB Artificial sweeteners and depression risk A handful of studies have hinted that people who eat diets heavy in ultra processed foods may be more likely to have depression, but there's been little research into which specific ingredients from those diets may increase risk. New research published Wednesday in JAMA Network Open shows that artificial sweeteners may be the cause.  Using data from the Nurses Health Study II, researchers looked at the health of nearly 32,000 middle-aged women, nearly all of them white, and their diets in the period between 2003-17. They found participants who ate more ultra processed foods were more likely to smoke, had greater BMI, were less likely to exercise regularly — and had higher rates of depression. The only specific component of the diet tied to higher depression risk were artificial sweeteners and artificially sweetened drinks. Other research, the authors note, ties such sweeteners to increased activity in a system of the brain (the purinergic) that's long been linked to depression and chronic pain. PUBLIC HEALTH Rare botulism case worries WHO officials With so many people traveling to France for the Rugby World Cup — Go American Samoa and Vanuatu! Aside from my Dodgers, I always root for underdogs — WHO officials are warning of a cluster of botulism cases that have included one death. The cases stem from a restaurant in Bordeaux where visitors earlier this month ate jarred sardines that were preserved in-house. Of the 15 known cases, 10 people were hospitalized, including eight in ICU units. Almost all cases occurred in foreign travelers, including people from Canada, Greece, Germany, Ireland, the U.K. and the U.S. WHO officials consider the outbreak a public health emergency and issued the warning because of botulism's eight-day incubation period and because quick diagnosis and treatment of the illness can prevent death.

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What we're reading More free Covid tests for U.S. households, STAT AlphaFold tool pinpoints protein mutations that cause disease, Nature A breakthrough cystic fibrosis drug gave them the gift of time. But miracles come with complications, STAT Only 2 percent of U.S. doctors are Latina, despite diversity leading to better care for patients, The 19th Researchers use AI to measure recovery from treatment-resistant depression, STAT

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Thanks for reading! More tomorrow — Usha

ushalee.mcfarling@statnews.com

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