FDA appears skeptical of ALS stem cell treatment

By David Lim, Katherine Ellen Foley and Lauren Gardner

Presented by 340B Health

With Chelsea Cirruzzo

FDA scientists have expressed concerns about authorizing a new ALS therapy that uses stem cells, like those depicted above. | Spencer Platt/Getty Images

‘MAJOR CONCERNS’ ABOUT ALS THERAPY — The FDA is weighing the first stem cell treatment to treat amyotrophic lateral sclerosis, and agency documents indicate regulators are leaning against clearing it for market.

FDA scientists said they have “major concerns” about the candidate, called NurOwn, ahead of a Wednesday meeting of the agency’s expert advisers. The drug, made by BrainStorm Cell Therapeutics, intends to treat ALS by using patients’ own stem cells to produce proteins thought to prevent neurons from dying.

In the past year, the FDA approved two ALS drugs that appear to slow the disease’s progression, marking a turning point in the industry. In 2022, the agency green-lighted Amylyx’s drug Relyvrio, and it granted accelerated approval to Biogen’s Qalsody earlier this year.

The two ALS treatments modestly prolonged patients’ ability to carry out key functions, and other treatments mitigate some of the disease’s side effects. About 31,000 individuals in the U.S. are living with ALS, and most patients die within five years of their diagnosis.

But the FDA is concerned about NurOwn. It noted that in a late-stage trial, the candidate failed to meet its endpoint and 10 out of 95 patients treated with the drug had died in the month after treatment, compared with three out of 94 who received the placebo.

Agency scientists also noted their misgivings about apparent missing data from the application package as well as concerns that “have yet to be resolved” about how BrainStorm Cell will manufacture the treatment.

The advisory meeting comes after the company asked the FDA for a second review. The company applied for NurOwn’s approval in January after the FDA had initially refused to review its application, citing data deficiencies.

In its new submission, the company said that further analyses of its late-stage trial show that NurOwn led to an increase in proteins that protect neurons and a decrease in those associated with neuron damage.

BrainStorm Cell Therapeutics did not respond to POLITICO’s request for comment.

What’s next: The FDA’s expert panel recommendations are nonbinding, and the agency is expected to decide on the submission by Dec. 8.

IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. You can once again order government-funded rapid Covid-19 tests from USPS.

Send news and tips to Lauren Gardner (lgardner@politico.com or @Gardner_LM), David Lim (dlim@politico.com or @davidalim) and Katherine Ellen Foley (kfoley@politico.com or @katherineefoley).

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TODAY ON OUR PULSE CHECK PODCAST, host Kelly Hooper talks with health care reporter Ben Leonard, who explains how hospitals are seeing a rise in uncompensated care as millions lose Medicaid coverage after redetermination resumed — and what that could mean for hospitals.

HHS Secretary Xavier Becerra is reminding insurers not to bill patients for Covid vaccines. | Evan Vucci/AP Photo

BECERRA TO INSURERS: COVER COVID SHOTS — The Department of Health and Human Services is warning pharmacies, providers and insurers to guarantee insured people get their updated Covid-19 vaccines for free amid reports that people have had trouble finding and accessing vaccines, Chelsea writes.

“Whether the problems are directly within your organization’s control or the control of your partners, your obligation as a plan or issuer to ensure that your members have coverage for COVID-19 vaccines without cost sharing is not conditional upon other parties’ compliance with applicable requirements,” HHS Secretary Xavier Becerra wrote in a letter to payers on Friday.

The government no longer purchases and supplies Covid shots for most of the public, and insurers must cover the vaccines for their members with no cost-sharing. But the first weeks of distribution have been bumpy, Chelsea and Lauren reported, with a mix of supply issues and insurance coverage hiccups that have left some people without their shots.

In the letter, Becerra pointed to a series of webinars the department has hosted over the past year to prepare for the rollout, as well as July and September letters from the CMS asking insurers to get their systems ready.

WHERE ARE THE KIDS’ SHOTS? Local health officials also report lags in pediatric Covid shots making their way to providers, Chelsea reports, backing up anecdotal reports that vaccines for young children are the hardest to find.

Adriane Casalotti, chief of government and public affairs of the National Association of County and City Health Officials, said many of her group’s members have yet to receive pediatric vaccines, and she expects supplies won’t arrive until the end of the month.

The youngest kids can be the toughest to vaccinate for Covid. Pharmacists and their technicians and interns can administer the shots for children as young as 3 until the end of 2024 under authority that was invoked during the public health emergency. But pediatric practices have to decide whether and how much vaccine to stock for a population that has seen low uptake — especially now that they’re on the hook for the shots’ upfront cost.

HAPPENING 9/28 — INSIDE THE CANCER MOONSHOT: Join POLITICO on Thursday, Sept. 28 for an in-depth discussion on the future of cancer treatment and innovation. Hear from experts including scientists, government officials and industry leaders as we explore the critical roles played by private industry, nonprofits, the National Cancer Institute and the new Advanced Research Projects Agency for Health in achieving the Biden administration's goal of cutting the cancer death rate in half over the next 25 years. Don't miss this opportunity to dive into the progress of cancer treatments and learn about the challenges patients encounter in accessing care. REGISTER HERE.

GOING TO BALTIMORE — Drugmaker AstraZeneca plans to negotiate a price with Medicare for its diabetes drug Farxiga, the company told POLITICO.

Farxiga — used to treat type 2 diabetes, heart failure and chronic kidney disease — was among the first 10 drugs selected by the Centers for Medicare and Medicaid Services last month for price negotiations Congress ordered in the 2022 Inflation Reduction Act.

“We remain committed to ensuring patients have access to Farxiga and plan to participate in the process outlined by CMS to communicate the value of Farxiga to people covered by Medicare,” AstraZeneca spokesperson Brendan McEvoy said in an email.

Litigation ongoing: Multiple drugmakers, including AstraZeneca, have sued the Biden administration over the Medicare drug price negotiation program.

SHUTDOWN WATCH — House Republicans are expected to try to tee up a rule to allow floor debate on a slate of fiscal 2024 bills, including the FDA’s funding bill, when they return this evening.

Among HHS agencies, the FDA is better positioned than most to weather a shutdown, but it would still face deleterious effects. Under the FDA’s fiscal 2024 contingency plan, 81 percent of agency employees would still be able to report to work in the event of a shutdown.

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FDA OPENS PUBLIC TOBACCO VIOLATIONS FORM — The FDA said Monday that it had established a portal for the public to flag potentially illegal tobacco products, including e-cigarettes, without marketing orders. Most e-cigarettes available for sale are unauthorized; the agency has granted marketing orders to only 23 products, all of which are tobacco-flavored.

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Ryan Long, a senior health policy staffer for Speaker Kevin McCarthy, is rejoining top lobbying firm BGR Group.

Bloomberg’s Ike Swetlitz profiles the impact dropping reimbursement rates for amoxicillin have had on manufacturer USAntibiotics.

The CDC on Friday recommended that people get vaccinated against respiratory syncytial virus when they’re 32 to 36 weeks pregnant.

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340B hospitals are an essential part of the nation’s health care safety net, providing crucial services to patients with low incomes and those who live in rural America. Hospitals use 340B savings – not taxpayer dollars – to provide most of the nation’s unpaid and Medicaid hospital care, and they deliver vital services where access is limited. They help keep rural hospitals open for the patients and communities they serve. 340B lowers health care costs; supports doctors, nurses, and pharmacists; and expands care for patients – all without using any taxpayer dollars. Protecting 340B is the best way forward for patients and communities. Learn more.

The FDA is holding a public workshop on the current landscape for drug dosing pediatric patients with renal impairment on Nov. 30 and Dec. 1.

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