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 and joined the MJH Life Sciences team in December 2019. She graduated from Youngstown State University in 2019 with a bachelor's degree in journalism and a minor in political science. Prior to graduating, Morgan worked as the editor-in-chief of her college newspaper. She went on to work as an assignment editor at a broadcast news station in Youngstown, Ohio before joining 

 You can reach her at mpetronelli@mjhlifesciences.com.

Articles

. He joined the brand in March of 2023 as an editor before being promoted to senior editor in January 2024. Fitch graduated from Youngstown State University in Youngstown, Ohio in 2020 with a degree in telecommunications and journalism. He started his career as a news and sports videographer before becoming an on-air sports anchor at the NBC-affiliated news station in Youngstown. Fitch briefly worked as a national content writer for a Chicago-based national television station before joining the 

team. He can be reached at: jfitch@mjhlifesciences.com.

Higher omega-3 intake may protect children from myopia, while diets high in saturated fats may raise risk, study finds.

Omega-3 fatty acid intake linked to reduced risk of myopia in children

Immune surveillance study shows pandemic interventions suppressed viral exposure in children, driving post-COVID resurgence and future outbreak risk.

Immune surveillance study reveals drivers of post-pandemic viral resurgence in children

Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.

Weekly review: AAP immunization schedule, vatiquinone CRL, and more

In recent studies, donidalorsen (DAWNZERA) has demonstrated durable efficacy and long-term disease control.

FDA approves first RNA-targeted therapy for hereditary angioedema

Contemporary Pediatrics connects pediatricians with peer-reviewed articles, guideline updates, and practice strategies for real-world clinical application.

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High-dose centanafadine significantly improved ADHD symptoms in children aged 6–12 and was well tolerated, according to a phase 3 clinical trial.

Centanafadine shows efficacy and tolerability for pediatric ADHD in phase 3 trial

If approved, vatiquinone would have been the first therapy approved for pediatric patients with FA. 

FDA issues CRL for vatiquinone to treat Friedreich ataxia

Richard Auchus, MD, PhD, discusses how crinecerfont is improving CAH care and easing the transition from pediatric to adult management.

Richard Auchus, MD, on how crinecerfont is changing CAH treatment

FDA approves first generic iron sucrose injection for treating iron deficiency anemia in CKD patients 2 years and older.

FDA approves first generic iron sucrose injection for iron deficiency anemia in CKD

Manufacturer MannKind as submitted a supplemental Biologics Application to the FDA for its inhaled insulin Afrezza.

Michael Haller, MD discusses inhaled insulin and Afrezza data for type 1 diabetes

On August 6, 2025, MannKind Corporation submitted a supplemental Biologics License Application to the FDA for its inhaled insulin Afrezza for a pediatric population indication, with a decision expected early in the fourth quarter of 2025, according to a financial report from the company.

The filing was based on data from the INHALE-1 study, led by Michael Haller, MD, MS-CI, lead author of the study and professor and chief of Pediatric Endocrinology at the University of Florida. The study evaluated the safety and efficacy of the inhaled insulin regimen as a replacement for rapid-acting meal insulin in children with type 1 diabetes (T1D). It included 230 patients aged 4 to 17 years, who were randomized to either inhaled insulin or rapid-acting analogue for meals, with a primary outcome of change in A

According to a press release from the American Diabetes Association's 85th Scientific Session, where data were presented at the June meeting in Chicago, findings indicated that inhaled insulin is safe and effective in children with T1D. Full study results, including a safety extension, have been submitted for presentation at a medical meeting sometime in the second half of 2025, according to MannKind.

"The primary outcome essentially showed non-inferiority of inhaled insulin compared to rapid-acting injected insulin," stated Haller in the video above. "This finding is slightly qualified by the inclusion of 1 patient who was highly non-adherent to their insulin regimen and was assigned to the inhaled insulin group. Including this patient in the analysis resulted in a non-significant p-value for non-inferiority. However, in a sensitivity analysis excluding this non-adherent [patient], the inhaled and injected insulin groups showed nearly identical outcomes in terms of hemoglobin A

Currently, Afrezza is being prescribed off-label for the pediatric population, with the adult population being the only FDA-approved indication for the inhaled insulin. Now, with a filing submitted to the federal agency, Afrezza is one step closer to a proper label pediatrics. 

"The field has long been in search of more rapid-acting insulins—truly rapid insulins—that can more physiologically cover meal excursions and corrections and reduce risk for post-meal hypoglycemia," added Haller. "In terms of injectable insulins, we've made some progress by manipulating the insulin structure so it gets into the bloodstream faster, but again, it's just not fast enough."

"Essentially, this is a biomaterial technosphere that gets bound to regular human insulin," Haller explained of Afrezza. "So, it's your non-manipulated insulin, and then, when it's inhaled into the lung, these particles dissociate, and the insulin is absorbed very rapidly through the lung blood vasculature, allowing the insulin to reach peak levels within 15 or 20 minutes. So, much closer to the way a human pancreas that's working should work. Because of that, it really can provide more optimal meal coverage, safer control of hyperglycemic corrections, and it's much better for athletes in terms of not having hypoglycemia associated with insulin dosing." 

Haller added that until there is a wider shift in how T1D treatment is looked at, utilization rates, even among adults, could be lower compared to other T1D insulin options. 

"It's been successful in adults, although it remains fairly underutilized, I would say, just because the paradigm hasn't shifted so that people think of using inhaled insulin as a first option. Everybody still kind of assumes we have to use injected," he said. "Current pediatric use is off-label, and myself and many others are prescribing it that way. Lots of pediatric medicine is off-label, unfortunately."

MannKind Corporation reports second quarter 2025 financial results and provides business update. MannKind Corporation. Press release. August 6, 2025. Accessed August 7, 2025. https://investors.mannkindcorp.com/news-releases/news-release-details/mannkind-corporation-reports-second-quarter-2025-financial 

Inhaled insulin shown as a safe and effective replacement for standard-of-care in children with type 1 diabetes. American Diabetes Association. Press release. June 22, 2025. Accessed August 7, 2025. https://professional.diabetes.org/sites/dpro/files/2025-06/ADA_InhaledInsulinPressRelease_FINAL.pdf

Videos

Manufacturer MannKind has submitted a supplemental Biologics Application to the FDA for its inhaled insulin Afrezza. 

News

Michael Haller, MD, discusses inhaled insulin and Afrezza data for type 1 diabetes

Suzanne Hollander, MS, RD, LDN and Timothy Tramontana, MD offer thoughts on the newly FDA-approved sepiapterin for PKU.

WATCH: Experts react to the FDA approval of sepiapterin to treat PKU

FDA approved PTC Therapeutics' sepiapterin (Sephience) 

to treat adult and pediatric patients with phenylketonuria (PKU). Sepiapterin is now the third FDA-approved medication for lowering blood phenylalanine (Phe) levels and can be used alongside a Phe-restricted diet to expand protein tolerance in individuals with sepiapterin-responsive PKU. 

"This is a really exciting time in PKU, with a new treatment coming out," said Suzanne Hollander, MS, RD, LDN, metabolic dietitian at Boston Children’s Hospital. "Sepiapterin is an oral Phe-lowering medication just recently approved for PKU in individuals of all ages. For individuals with PKU, historically, standard-of-care treatment has included a protein-restricted diet with low phenylalanine formula. More recently, there have been 2 Phe-lowering medications available on the market. These previous 2 medications and the diet do not always sustainably work for everyone to keep their Phe level within the target treatment range. That target treatment range is less than 360 µmol/L."

FDA approval follows the phase 3 APHENITY trial, in which sepiapterin demonstrated potential benefits such as increased dietary protein intake and reduced reliance on PKU-specific formula.

"Sepiapterin will become the third FDA-approved Phe-lowering medication. The approval is exciting because it offers an additional Phe-lowering option, and it may be appropriate for some people who have not achieved their treatment goals with the other 2 available medications and/or with the PKU diet," Hollander said. 

For metabolic care teams, the approval introduces new considerations for treatment planning. "Many individuals will still require a phenylalanine-restricted diet alongside this therapy. However, sepiapterin does offer a Phe-lowering effect that could allow individuals to liberalize their diet and eat more dietary phenylalanine and more dietary protein. For some people, it may lower their blood phenylalanine level enough that they could receive all the dietary protein they need from food, allowing them to discontinue use of the phenylalanine-restricted PKU formula. Anytime people on a restricted diet can liberalize their protein intake, it is a huge win."

Timothy Tramontana, MD, medical and biochemical geneticist at Cleveland Clinic Foundation, emphasized the broader impact. "The added benefit is that we currently have a drug called sapropterin, which we have been utilizing for a long time under the brand name Kuvan. Sepiapterin provides an alternative treatment to this, hopefully helping patients with extremely diminished levels of phenylalanine hydroxylase, the enzyme affected in this disorder. It is thought that patients even with null mutations or mutations that may be leaky—meaning there may be minimal to almost no enzyme—may benefit from this treatment. The drug has a chaperone effect; it can help with the conformation of the enzyme, which may be affected. Those with almost no enzyme activity may actually see benefit from it in reducing their phenylalanine levels compared with a drug like sapropterin or Kuvan." 

"It goes hand in hand; the approach has always centered on diet, with pharmacologic management as an adjunct to hopefully help liberalize diet. That approach has helped a number of people through the use of sapropterin and now with sepiapterin. The hope is to expand the number of patients who may be able to liberalize their diet and increase their protein intake. That is our goal for most of our patients: to help them live a more normal life in terms of intake. It may never be 100% protein unrestricted, but the hope is to liberalize it enough to allow increased intake," Tramontana added.

Both experts encouraged primary care providers to support continued specialty care. "For general practitioners working with individuals with PKU, we absolutely want you to encourage individuals with PKU in your practice to come to a PKU clinic, which may also be referred to as a metabolism or metabolic clinic. Even if someone has been away from clinic for many years, we always welcome people back so they can be made aware of these new treatment options," Hollander said.

"We are excited that there is another drug on the horizon that can help our patients live a more normal life and increase their protein intake," Tramontana added. "That is always exciting. General providers need to be aware that there are treatments available and should enlist the help of a biochemical geneticist. We work hand in hand with metabolic dietitians who are integral to the team and the management of these patients. Knowing that a new treatment may help patients further liberalize their protein intake is going to be important," Tramontana said. 

Suzanne Hollander, MS, RD, LDN; and Timothy Tramontana, MD, offer thoughts on newly FDA-approved sepiapterin for PKU.

"These younger individuals are at much higher risk of getting disease and having complications," said Tina Tan, MD, FIDSA, FPIDS, FAAP. 

Tina Tan, MD, comments on FDA expanded approval for MenQuadfi meningococcal disease vaccine

Last week, the FDA approved an expanded indication for Sanofi's MenQuadfi meningococcal vaccine—for the prevention of invasive meningococcal disease (IMD) caused by Neisseria meningitidis serogroups A, C, W, and Y—that now includes children aged 6 weeks to 23 months. The approval expanded on the previous indication for individuals aged 2 years or older.

A Sanofi spokesperson told our sister publication 

, because this decision by the FDA is an expansion, health care providers can start using the vaccine in infants aged 6 weeks or older, however, direct implementation depends on other factors.

"It is anticipated that the Centers for Disease Control and Prevention (CDC) Advisory Committee of Immunization Practices (ACIP) will vote on the use of MenQuadfi in infants and toddlers 6 weeks through 23 months of age during the ACIP meeting scheduled for next month, in June 2025," stated the spokesperson.

In the brief video above, Tina Tan, MD, FIDSA, FPIDS, FAAP, editor in chief, 

; president, Infectious Diseases Society of America (IDSA), provided comments on the expanded approval. 

"We know that over the last couple of years, there has been a significant increase in the amount of meningococcal disease that's being seen here in the United States," said Tan. "The majority of disease seen now is serotype Y, which this vaccine contains. Individuals at the highest risk are young infants, in addition to individuals like college students, people in military barracks, or teenagers who have other risk factors for disease. These younger individuals are at much higher risk of getting disease and having complications. This is not something that is new, because there are many countries that have recommendations to give a meningococcal vaccine to the younger pediatric population to protect them."

This video interview is the second part of a series highlighting the FDA approval of MenQuadfi

Click here for the fully-detailed article of the approval, including additional comments and clinical trial safety data

1. Fitch, J. FDA approves MenQuadfi meningococcal vaccine for children 6 weeks or older. Contemporary Pediatrics. May 27, 2025. Accessed May 27, 2025. https://www.contemporarypediatrics.com/view/fda-approves-menquadfi-meningococcal-vaccine-for-children-6-weeks-or-older

2. Parkinson, J. FDA approves an expanded indication for meningococcal vaccine for youngest pediatric population. Contagion. May 27, 2025. Accessed May 27, 2025. https://www.contagionlive.com/view/fda-approves-an-expanded-indication-for-meningococcal-vaccine-for-youngest-pediatric-population

Roflumilast foam 0.3% trial investigator Jennifer Soung, MD joined us to discuss the importance of this newly FDA-approved topical treatment for scalp and body psoriasis patients aged 12 years and older. 

Jennifer Soung, MD reacts to FDA approval of roflumilast foam 0.3%

FDA approved roflumilast (Zoryve; Arcutis Biotherapeutics) foam 0.3%

 to treat plaque psoriasis of the scalp and body in individuals aged 12 and older. Jennifer Soung, MD, director of clinical research at Southern California Dermatology and a clinical trial investigator, joined us to break down the approval and the impact it can have in the psoriasis treatment landscape.

"I'm super excited," said Soung. "Parents have been asking for many years, 'Is there anything else besides the topical steroid?' Prior to the approval of roflumilast, we really haven't had a highly effective nonsteroidal option.”

The foam formulation was approved based on findings from the phase 3 ARRECTOR trial and phase 2 Trial 204. In the ARRECTOR trial, 66.4% of participants using roflumilast foam achieved a Scalp-Investigator Global Assessment (S-IGA) Success compared to 27.8% using vehicle (

 < 0.0001), while Body-Investigator Global Assessment (B-IGA success) was achieved in 45.5% compared to 20.1% with vehicle (

Soung emphasized the psychosocial burden that visible skin disease places on younger patients, stating, "They’re much more susceptible to the psychosocial impacts of plaque psoriasis because it’s such a visible disease,” she said. “They tend to not want to do sports, or they don’t participate in social activities, so they’re much more withdrawn." 

The studies also measured improvement in itch, one of the most bothersome symptoms for patients. In ARRECTOR, 65.3% of patients treated with roflumilast experienced significant itch reduction compared to vehicle, with some scalp itch relief evident within 24 hours of the first dose (

 = 0.0164). Roflumilast foam 0.3% was well tolerated, with adverse events such as headache, diarrhea, and nausea occurring in fewer than 4% of patients. 

“One thing I love about Arcutis and the way they designed their clinical trials is they did their trials in adults and adolescents concurrently,” Soung noted. “That makes it easier for that pediatric patient and their families.”

Editors’ note: Jennifer Soung, MD has relevant disclosures with AbbVie, Alumis, Aslan, Amgen, Arcutis, Bristol Myers Squibb, Boehringer Ingelheim, Biopharma, Dermavant, Eli Lilly, Galderma, Incyte, Johnson & Johnson, KoBio Labs, LEO, Novartis, Ortho Dermatologics, Pfizer, Regeneron, Sanofi, and UCB. 

This is the second part of a series highlighting the FDA approval of roflumilast foam 0.3%. 

Click here for part 1, which includes full approval details.

Click here for part 3, which includes additional commentary from health care professionals. 

Fitch, J. FDA approves roflumilast foam 0.3% for scalp, body psoriasis. Contemporary Pediatrics. May 22, 2025. Accessed May 22, 2025. https://www.contemporarypediatrics.com/view/fda-approves-roflumilast-foam-0-3-for-scalp-body-psoriasis 

Roflumilast foam 0.3% trial investigator Jennifer Soung, MD, joined us to discuss the importance of this newly FDA-approved topical treatment for patients aged 12 years or older with scalp and body psoriasis.

Jennifer Soung, MD, reacts to the FDA approval of roflumilast foam 0.3%

A review of the clinical potential of PTC Therapeutics' sepiapterin as a novel treatment for phenylketonuria (PKU). 

PKU and sepiapterin data with Suzanne Hollander, MS, RD, LDN

Phenylketonuria (PKU), a rare genetic disorder that affects the body’s ability to metabolize phenylalanine (Phe), can cause severe neurocognitive and emotional complications without strict treatment.

“Without treatment, individuals with PKU would have very high blood Phe levels, and high blood Phe levels are a toxin to the brain, so that results in neurocognitive deficits and mood disorders," said Suzanne Hollander, MS, RD, LDN, senior clinical nutrition specialist at Boston Children’s Hospital, in the above video interview with 

PKU is typically diagnosed shortly after birth through newborn screening, and treatment begins immediately. The goal is to keep blood Phe levels under 360 micromoles per liter, according to Hollander.

“The current standard treatment that we use for PKU is a phenylalanine-restricted diet,” she said. “We pair that with a phenylalanine-restricted medical formula that provides a lot of the nutrients that would otherwise be deficient in the protein-restricted diet alone. The diet is both logistically challenging and clinically challenging. Sepiapterin is an exciting novel treatment. It works to lower levels of phenylalanine in the blood, which can improve clinical outcomes and also allow for individuals to eat more natural protein in their diet. Getting to expand their diet really can alleviate the treatment burden that goes along with PKU," added Hollander. 

Sepiapterin enhances the function of the enzyme that converts phenylalanine to tyrosine, lowering Phe levels and potentially enabling greater diet liberalization.

“Overall, participants in the trial were able to lower their Phe levels, and 84% of individuals reached the target range of less than 360,” said Hollander, highlighting findings from the Phase 3 APHENITY trial. “That’s really meaningful, because historically, those individuals often do not respond to the current oral phenylalanine-lowering therapy that’s on the market.” 

These data were supported by recent findings presented at the 2025 ACMG Annual Clinical Genetics Meeting and shared by PTC Therapeutics, showing that over 70% of trial participants had a genotype consistent with classical PKU and still responded to treatment. 

“From a dietary standpoint, the study was really exciting because we did a Phe tolerance assessment,” said Hollander. “For individuals who had their Phe level lowered with the medication to less than 360, they were able to participate in a portion of the study where we systematically monitored their Phe levels and then increased their dietary or natural protein.” Overall, participants saw a 126% increase in natural protein intake and a 50% reduction in their use of medical formula, a meaningful outcome given the burden the formula places on children and families.

"Seventy-three percent of the patients doubled their phenylalanine intake, and 34% tripled it. So on an individual basis, people were really able to liberalize their diet quite a bit," Hollander stated in the interview. "Additionally, those who came into the study on the current Phe lowering medication, BH4, they were able to increase their Phe intake, 52% of them doubled their Phe intake beyond what they had tolerated on BH4 therapy alone."

The phase 3 APHENITY trial results were recently published in 

, and the FDA has assigned a Prescription Drug User Fee Act date of July 29, 2025, to review sepiapterin for potential approval. If approved, the treatment could represent a significant step forward in both metabolic control and quality of life for individuals with PKU.

1. PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies. PTC Therapeutics. Press release. March 20, 2025. Accessed May 6, 2025.https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-presents-new-sepiapterin-data-ongoing-studies 

2. PTC Therapeutics Announces FDA Target Regulatory Action Date for Sepiapterin and Lancet Publication of Phase 3 Trial Results. Press release. October 14, 2025. Accessed May 6, 2025. https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-target-regulatory-action-date

A review of the clinical potential of PTC Therapeutics' sepiapterin as a novel treatment for phenylketonuria (PKU).

RX Review: Emerging research shaping the future of food allergy care in 2025

In the final part of this 5-part series, panelists discuss new allergy treatments such as Xolair, early immunotherapy, and their broader impact.

Drug Pipeline News

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