Each year, the FDA approves several treatments—whether they be vaccines, topical agents, biologics, or monoclonal antibodies—that aim to help the pediatric population. From dermatology to rare disease, 2024 brought more than 25 pediatric FDA approvals into the collective pediatric treatment toolbox for general providers and specialists alike. But before we get into our “top” approvals for the year, let’s highlight some key areas of pediatric health care that saw important approvals.

After delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics, Inc) was approved in 2023 as the first gene therapy to treat pediatric patients aged 4 to 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene, givinostat (Duvyzat; Italfarmaco SpA) oral medication was approved on March 22, 2024. The approval made givinostat the first nonsteroidal drug approved to treat patients with all genetic variants of DMD, markingtreatment progress for the rare neurological disorder has been made over the past 2 years.^1-3

In the infectious disease space, FluMist (Influenza Vaccine Live; AstraZeneca) was further approved for the prevention of influenza disease caused by subtypes A and B for self- or caregiver administration among individuals aged 2 to 49 years on September 20, 2024. FluMist was initially approved for use in 2003 for individuals aged 5 to 49 years, and the FDA approved the use of the nasal vaccine in children as young as 2 years in 2007.⁴

“Making it available for in-home use should increase the use of this vaccine and influenza vaccine; however, it will be important that the selling of this vaccine be monitored, as you do not want the wrong individuals receiving the vaccine,” Tina Tan, MD, FAAP, FIDSA, FPIDS, who is Contemporary Pediatrics’ editor in chief, a professor of pediatrics at Northwestern University Feinberg School of Medicine, and a pediatric infectious diseases attending at the Ann & Robert H. Lurie Children’s Hospital of Chicago in Illinois, said at the time of the expanded approval.⁴

In the mental and behavioral health space, one key approval this year was clonidine hydrochloride (Onyda XR; Tris Pharma, Inc) to treat attention-deficit/hyperactivity disorder (ADHD) as a monotherapy or as an adjunctive therapy to approved central nervous system stimulant medications for patients 6 years and older. The once-a-day extended-release oral suspension with nighttime dosing is the first and only nonstimulant ADHD medication approved in the US and the only approved nonstimulant ADHD medication with nighttime dosing, which became available in early October.⁵

Click here for a discussion on clonidine hydrochloride.

To close out the year, Contemporary Pediatrics is highlighting the top 3 FDA approvals for pediatric health care below. Click here for full FDA-related coverage throughout the year, including approvals, complete response letters, new data, and other pipeline updates.

Dupilumab (Dupixent; Regeneron Pharmaceuticals, Inc/Sanofi)

First treatment indicated specifically for children with eosinophilic esophagitis

Dupilumab was approved for eosinophilic esophagitis (EoE) on January 25, 2024, for patients aged 1 to 11 years weighing at least 33 lb (15 kg). Dupilumab treatment is beneficial in several ways but also serves as an important approval for the chronic, progressive disease that is driven by type 2 inflammation that damages the esophagus.⁶

“This approval serves an unmet need,” Theresa Bingemann, MD, an associate professor of pediatrics and medicine and program director of the allergy and immunology fellowship training program at the University of Rochester School of Medicine and Dentistry in New York, said at the time of the approval. “This provides another option for patients [who] are still symptomatic on current treatments. It also adds another treatment to the armamentarium for the treatment of EoE and gives families more options to decide what treatment fits best with their goals and values. Improved treatment may reduce the feeding difficulties that are often seen in the youngest kids.”⁶

The approval was granted based on data from the phase 3 EoE KIDS trial (NCT0439435; parts A and B), which evaluated the safety and efficacy of dupilumab in children aged 1 to 11 years with EoE.⁶ Of the children who received a higher dose of dupilumab at tiered dosing regimens based on weight (n = 32), 66% achieved histological disease remission, which was the primary end point of the study, compared with 3% of patients who achieved remission with placebo (n = 29).¹

Seventeen of 32 patients (53%) treated with dupilumab in parts A and B of the study sustained histological remission at week 52. Remission was also achieved at week 52 for 8 of 15 children (53%) who switched to dupilumab treatment in part B of the study.⁶

Additionally, at 16 weeks, “a greater decrease in the proportion of days with [1] or more signs of EoE based on [the] Pediatric EoE Sign/Symptom Questionnaire-Caregiver version was observed in children treated with [dupilumab]” compared with placebo, according to a statement from Sanofi.⁶

Epinephrine nasal spray (neffy; ARS Pharmaceuticals, Inc)

First nasal spray for treatment of anaphylaxis

Nearly a year after a complete response letter was issued for neffy at the request of a pharmacokinetic/pharmacodynamic study to assess repeat doses of neffy compared with repeat doses of an epinephrine injector, the nasal spray received approval from the FDA on August 9, 2024. The spray is indicated for the treatment of type 1 allergic reactions, including anaphylaxis, in adult and pediatric patients who weigh at least 66 lb (30 kg).^7,8

“Anaphylaxis is life-threatening, and some people, particularly children, may delay or avoid treatment due to fear of injections,” Kelly Stone, MD, PhD, an associate director in the Division of Pulmonology, Allergy, and Critical Care at the FDA’s Center for Drug Evaluation and Research, said in a statement at the time of approval. “The availability of epinephrine nasal spray may reduce barriers to rapid treatment of anaphylaxis. As a result, neffy provides an important treatment option and addresses an unmet need.”⁷

“We know that a lot of patients are reluctant to either inject themselves or inject their children with epinephrine because of needle phobia,” Thomas Casale, MD, a professor of medicine and pediatrics, and chief of clinical and translational research in the Division of Allergy and Immunology at the University of South Florida in Tampa, said in a previous interview with Contemporary Pediatrics. “Having alternatives is very important.”⁸

Approval was based on comparable epinephrine blood concentration data following administration of neffy or approved epinephrine injection products demonstrated in 4 studies comprising 175 healthy adults without anaphylaxis. Similar increases in blood pressure and heart rate were observed for neffy compared with injection products, a pair of critical epinephrine effects when treating anaphylaxis.⁷

Roflumilast cream 0.15% (Zoryve; Arcutis Biotherapeutics, Inc)

Mild to moderate atopic dermatitis

On July 9, 2024, roflumilast cream 0.15% was approved by the FDA to treat mild to moderate atopic dermatitis (AD) in adults and children aged 6 years and older.⁹ The FDA’s decision is supported by the positive results of several clinical trials that demonstrated the efficacy and safety of roflumilast 0.15%.

In a pair of identical, phase 3, parallel-group, double-blind, and vehicle-controlled trials, the safety and efficacy of roflumilast 0.15% was compared with vehicle and applied once daily for 4 weeks. The INTEGUMENT-1 (NCT04773587) and INTEGUMENT-2 (NCT04773600) trials featured more than 1300 adults and children aged 6 years or older with mild to moderate AD.^9,10

Both studies met the primary end point of Investigator’s Global Assessment (IGA) success, defined as a Validated IGA-AD score of clear (0) or almost clear (1) plus a 2-grade improvement from baseline at week 4 (INTEGUMENT-1: 32% roflumilast cream vs 15.2% vehicle [P < .0001]; INTEGUMENT-2: 28.9% roflumilast cream vs 12% vehicle [P < .0001]).⁹

In addition, more than 40% of adults and children aged 6 years and older treated with roflumilast cream achieved a 75% reduction according to the Eczema Area and Severity Index at week 4 (INTEGUMENT-1: 43.2% vs 22% [P < .0001]; INTEGUMENT-2: 42% vs 19.7% [P < .0001]).¹⁰

“I’m pretty excited to get a new nonsteroidal medicine that we can put in our regimens of care,” Lawrence Eichenfield, MD, a professor of dermatology and pediatrics and vice chair of the Department of Dermatology at the University of California, San Diego, and chief of pediatric and adolescent dermatology at Rady Children’s Hospital-San Diego, said in a previous interview with Contemporary Pediatrics.¹⁰

“Now we have a new, well-formulated…water-based emollient formulation with a very well-designed topical nonsteroid—in this case, the [phosphodiesterase-4]for roflumilast—that works well on the skin,” Eichenfield said. “The product goes into the lipid layers and has shown very nice anti-inflammatory effects, with an impact on eczema and…on itch. [This allowed] a significant number of patients to make it to these important end points that we see in the clinical study.”¹⁰

Editor’s note:

Each year, the FDA approves several treatments across various indications and patient populations, with every approval important for that population. There are no true “top” approvals when it comes to the better care and treatment of pediatric patients across the entire spectrum of health care. The above list of 3 FDA approvals that took place in 2024 is based on editorial discussions with pediatric health care experts, the novelty of the treatments, and the expected impact on the disease states.

References:

1. Sarepta Therapeutics announces FDA approval of Elevidys, the first gene therapy to treat Duchenne muscular dystrophy. Press release. Sarepta Therapeutics; June 22, 2023. Accessed November 1, 2024. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene

2. FDA approves nonsteroidal treatment for Duchenne muscular dystrophy. Press release. FDA; March 21, 2024. Accessed November 1, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy?utm_medium=email&utm_source=govdelivery

3. Sarepta Therapeutics announces expanded US FDA approval of Elevidys to Duchenne muscular dystrophy patients ages 4 and above. Press release. Sarepta Therapeutics; June 20, 2024. Accessed November 1, 2024. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-expanded-us-fda-approval-elevidys

4. FDA approves nasal spray influenza vaccine for self- or caregiver-administration. Press release. FDA; September 20, 2024. Accessed November 1, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-nasal-spray-influenza-vaccine-self-or-caregiver-administration?utm_medium=email&utm_source=govdelivery

5. Tris Pharma receives U.S. FDA approval for once-daily Onyda XR (clonidine hydrochloride) extended-release oral suspension, the first-and-only liquid non-stimulant ADHD medication. Press release. Tris Pharma; May 29, 2024. Accessed November 1, 2024. https://www.businesswire.com/news/home/20240529072742/en/Tris-Pharma-Receives-U.S.-FDA-Approval-for-Once-Daily-ONYDA-XR-clonidine-hydrochloride-Extended-Release-Oral-Suspension-the-First-and-Only-Liquid-Non-Stimulant-ADHD-Medication

6. Dupixent FDA approved as first and only treatment indicated for children aged 1 year and older with eosinophilic esophagitis (EoE). Press release. Sanofi; January 25, 2024. Accessed November 1, 2024. https://www.sanofi.com/en/media-room/press-releases/2024/2024-01-25-19-30-00-2817342

7. FDA approves first nasal spray for treatment of anaphylaxis. Press release. FDA; August 9, 2024. Accessed November 1, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-nasal-spray-treatment-anaphylaxis

8. Fitch J. FDA approves neffy as first nasal spray to treat anaphylaxis. Contemporary Pediatrics. August 9, 2024. Accessed November 1, 2024. https://www.contemporarypediatrics.com/view/fda-approves-neffy-as-first-nasal-spray-to-treat-anaphylaxis

9. FDA approves Arcutis’ Zoryve (roflumilast) cream 0.15% for the treatment of atopic dermatitis in adults and children down to 6 years of age. Press release. Arcutis Biotherapeutics, Inc. July 9, 2024. November 1, 2024. https://www.arcutis.com/fda-approves-arcutis-zoryve-roflumilast-cream-0-15-for-the-treatment-of-atopic-dermatitis-in-adults-and-children-down-to-6-years-of-age/

10. Fitch J. FDA approves roflumilast cream 0.15% for atopic dermatitis in patients aged 6 years and up. Contemporary Pediatrics. July 9, 2024. Accessed November 1, 2024. https://www.contemporarypediatrics.com/view/fda-approves-roflumilast-cream-0-15-atopic-dermatitis-patients-aged-6-years-older