This Week at FDA: FDA’s autism moves, Novartis gets untitled letter over TV ad, and more

Welcome to another installment of This Week at FDA, your weekly source for updates—big and small—on FDA, drug, and medical device regulation and what we’re reading from around the web. This week, FDA kicked off the process to change the label for acetaminophen, following claims by President Donald Trump that the drug can cause autism when used during pregnancy. The agency also took the unusual step of approving a drug to treat autism symptoms without a submission from a drugmaker, and it sent another enforcement letter to a drugmaker over a TV ad.

On Monday, FDA announced that acetaminophen use during pregnancy “may be associated with an increased risk of neurological conditions such as autism and ADHD in children,” following an announcement by Trump and federal health leaders citing the drug as a cause of the disorder. Acetaminophen is widely considered to be the only safe over-the-counter pain and fever reducer for pregnant women, as other commonly available pain relievers, such as ibuprofen and aspirin have known risks to the fetus.

During a press conference with Health and Human Services Secretary Robert Kennedy Jr. and FDA Commissioner Marty Makary, Trump told pregnant women not to take acetaminophen. “There’s no downside. Don’t take it … if you’re pregnant, don’t take Tylenol.”

The announcement drew criticism and pushback from numerous medical groups. The American College of Obstetricians and Gynecologists (ACOG) issued a statement declaring that the announcement “is not backed by the full body of scientific evidence and dangerously simplifies the many and complex causes of neurologic challenges in children.”

Other international health bodies also pushed back on the Trump Administration’s claims regarding acetaminophen. Both the European Medicines Agency (EMA) and the World Health Organization (WHO) issued statements supporting the drug’s use in pregnancy and refuting the claim that there is conclusive evidence of a link to neurodevelopmental disorders.

However, FDA announced that it has started the process of asking drugmakers to update their labels to highlight the possible association. As reported by several news outlets, including Forbes, Makary told Fox News' Laura Ingraham that there was overwhelming evidence of a causal relationship between the neurological disorders and acetaminophen use. However, FDA's own press release notes that a causal association has not been established and points out that acetaminophen is the only over-the-counter drug approved for use to treat fevers during pregnancy.

Following pushback on the decision, the White House also shared a 2017 Tweet from the makers of Tylenol stating that they do not recommend any of their products for pregnant women. However, experts pointed out to MedPage Today that the tweet was taken out of context and that all OTC drugs are required to include pregnancy warnings.

FDA also announced it has approved GSK’s Wellcovorin (leucovorin calcium), also known as folinic acid, to treat patients with cerebral folate deficiency (CFD). The agency said that people with CFD have been observed to have autistic features, seizures, and problems with movement and coordination. Wellcovorin was initially approved in 1983 to counteract the effects of certain chemotherapies and was withdrawn by GSK in 1999, though generic versions of the drug remain on the market.

While Makary said during the press conference that the move was intended to make the drug available to children with autism, the agency said in its press release that "there are limitations on the available data for the use of leucovorin in this population and additional studies are needed to assess safety and efficacy."

As the New York Times pointed out, the approval is unusual in that FDA granted it without a submission from the manufacturer supplying data to support the drug’s use in a new indication, and instead the agency relied on existing medical literature. GSK has said it will submit a supplemental new drug application (sNDA) to the agency to update the drug’s label with the new indication

Experts told MedPage Today that FDA's decision to make the drug available to patients with autism is based on limited data, and they could be setting up the autism community for disappointment. They also noted that Mehmet Oz, director of the Center for Medicare and Medicaid Services (CMS), who was also present at the White House press conference, is an investor in iHerb, a company that sells folinic acid as a supplement.

George Tidmarsh, director of the Center for Drug Evaluation and Research (CDER), published a blog post noting that despite legislation that took effect in 2017 requiring specific clinical trial research to be published on ClinicalTrials.gov, many studies have not been published by researchers. He pointed to a recent article in the Journal of the Academy of Public Health, supporting his argument, and encouraged researchers to comply.

Drugs & Biologics

Continuing its recent efforts to address false and misleading direct-to-consumer (DTC) advertising, FDA issued an untitled letter to Novartis regarding a TV ad for its drug Fabhalta (iptacopan). The agency said the ad had too much happening during its major statement, making it hard for consumers to comprehend the potential risks of the drug.

President Trump announced that, starting 1 October, any branded or patented drugs imported to the US would be subject to 100% tariffs. However, drug manufacturers with a manufacturing facility or plan to build one will be exempt from the tariffs. NBC reported that the move could significantly raise prices for American consumers starting next week.

FDA has lifted the Risk Evaluation and Mitigation Strategies (REMS) program for Sanofi's thyroid cancer drug Caprelsa (vandetanib). The program was put in place in 2011 to ensure heart rhythm monitoring and safe use, but the agency says it has decided to remove it after it found no reports of cases of Torsades de pointes or unexplained sudden deaths in US patients using the drug.

Eli Lilly's estrogen receptor antagonist drug Inluriyo (imlunestrant) was approved by FDA to treat adults with estrogen receptor (ER)-positive, human epidermal growth factor 2 (HER2)-negative, estrogen receptor-1 (ESR1)-mutated advanced or metastatic breast cancer with disease progression who have undergone at least one line of endocrine therapy. The agency announced it had also Guardant360 CDx assay as a companion diagnostic for the drug to diagnose patients with breast cancer with ESR1 mutations.

Crinetics' Palsonify (paltusotine) has been approved to treat acromegaly, a rare endocrine disorder, in adults who did not respond well to surgery and/or for whom surgery is not an option. FDA noted, however, that the drug increases the risk of cholelithiasis (gallstones); hyperglycemia (high blood sugar); hypoglycemia (low blood sugar); bradycardia (low heart rate); thyroid function abnormalities; steatorrhea (excessive fat in the stool) and malabsorption of dietary fats; and changes in vitamin B12 levels.

FDA announced it is hosting a public workshop on 23 October to discuss regulatory requirements for approving new patch test allergens to diagnose allergic contact dermatitis. The agency plans to use feedback from the meeting to inform its review of the products.

FDA also reminded stakeholders that registration for its public workshop on modeling and using artificial intelligence (AI) in generic drug development and product lifecycle management was open. The event, which is co-hosted by the Center for Research on Complex Generics (CRCG), is on 15 October.

FDA published several guidances this week, including a draft guidance on developing malaria medication. The guidance broadly addresses drug development issues, but also looks at considerations depending on the severity of the disease and statistical considerations.

Medtech

The medtech lobby group AdvaMed responded to the Department of Commerce's (DOC) decision to initiate an investigation under Section 232 of the 1962 Trade Expansion Act to review the impact of medical device imports on national security. The group highlighted the positive impact medical imports have on the US economy and said it will work with the administration to strengthen the medtech industry.

Essilor's Stellest eyeglass lenses have been authorized by FDA as the first device to correct and slow down myopia (nearsightedness), with or without astigmatism, in children between six and 12 years of age. CDRH director said the lenses could "meaningfully reduce the likelihood of severe eyesight issues later in adult life."

This Week at FDA: FDA’s autism moves, Novartis gets untitled letter over TV ad, and more

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