The latest FDA approvals for children’s medications
The Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act have continued to provide us with more US Food and Drug Administration (FDA)-approved medications for pediatric patients and updates in labeling that help us safely and effectively use medications in children. This article summarizes labeling changes, new dosage forms, and new drug approvals from November 2021 to December 2022. Table 1 includes information on newly approved drugs indicated for use in pediatric patients, whereas Table 2 lists newly approved drugs for adults with the potential for use in pediatric patients. Table 3 describes new dosage forms for medications, and Table 4 lists labeling changes to existing drugs with implications for pediatric patients.
The use of biologic therapies for managing chronic pediatric diseases such as atopic dermatitis and asthma has evolved. Adbry (tralokinumab-ldrm)1 is a subcutaneous monoclonal antibody approved for the management of moderate to severe atopic dermatitis in adult patients. Results from clinical studies have demonstrated benefits from scores on the Investigator’s Global Assessment scale and the Eczema Area and Severity Index. Additionally, patients reported improved sleep scores and decreased itch,2 end points affecting the quality of life of patients with atopic dermatitis. Although tralokinumab-ldrm is not yet approved in pediatrics, the plan is to study it in hopes of expanding indications.
Other targets of biologic therapy for atopic dermatitis are the JAK inhibitors. Recent approvals that target the JAK pathway include Opzelura (ruxolitinib), Cibinqo (abrocitinib), and a labeling update to include adolescents for Rinvoq (upadacitinib).2 Approval for these agents was based on clinical outcomes of decreased itch improvements in skin clearance, extent of disease, and severity.3-5 Abrocitinib and upadacitinib are oral agents, and ruxolitinib is a topical agent, providing a more patient-friendly and easier to use dosage formulations for biologics in pediatric patients.
For the management of asthma, the novel agent Tezspire (tezepelumab-ekko) was approved in December 2021 for add-on maintenance management of asthma in adult and pediatric patients 12 years or older. Results from 2 pivotal trials, the Pathway trial and the NAVIGATOR trial (NCT03347279), led to the approval of the drug. Pediatric patients 12 years or older were included in the NAVIGATOR trial. Tezepelumab decreased all-comers exacerbations in the Pathway trial by 71% and by 56% in the NAVIGATOR trial. Decreases in hospitalizations, improvement in forced expiratory volume in the first second, and reductions in blood and sputum eosinophil and fractional exhaled nitric oxide levels were observed. In addition, an overall decrease in exacerbations occurred across all phenotypic styles of asthma. At this point, available biologics work for specific phenotypic styles, so tezepelumab is unique in that it is not phenotype dependent.6
Livtencity (maribavir) was approved in November 2021 and provided a significant advancement in the management of cytomegalovirus (CMV) in adults and pediatric patients 12 years or older weighing 35 kg or more with posttransplant CMV infection or disease refractory to alternative treatment. It has demonstrated efficacy in 56% of patients (n=131/235) compared with those treated with conventional antiviral therapies (24%; n=28/117) (adjusted difference: 33%; 95% CI, 23-43; P<.001).7 Prior to FDA approval, it was granted orphan drug designation by the FDA for management of clinically significant CMV viremia and disease in patients at risk and granted Breakthrough Therapy designation for management of CMV infection and disease in patients who received transplants whose disease was resistant or refractory to prior therapy. It is currently being studied as first-line management of CMV in patients who received stem cell transplants.8 A pediatric study is planned but has not yet started enrolling.9
Recent approval of first-in-class biologic agent Tzield (teplizumab-mzwv) presents a novel approach to the management of type 1 diabetes (T1D), becoming the first drug to delay disease progression. Administered as a single course consisting of once-daily intravenous infusions for 14 consecutive days, this agent is indicated to delay the onset of stage 3 T1D in patients 8 years or older with stage 2 T1D. Approval was based on the results of a phase 2, randomized, double-blind, placebo-controlled trial that demonstrated superior efficacy of teplizumab in delaying onset to diagnosis vs placebo (48.4 vs 24.4 months) in relatives of T1D patients at high risk of developing disease.10,11
Other novel treatments from this year include the approval of several gene therapy cellular products, effectively changing the treatment paradigm for certain chronic diseases. One of these agents, Zynteglo (betibeglogene autotemcel), was approved for the treatment of adult and pediatric patients with β-thalassemia (all genotypes) who require regular red blood cell transfusions. Approval was based on ongoing phase 3 studies, in which 89% of patients achieved transfusion independence for a continuous period of 12 months or greater. This presents a major shift in treatment options for this patient population, potentially eliminating the need for allogeneic hematopoietic stem cell transplantation.12
New drug dosage forms
As more medications continue to receive expanded pediatric indications, new dosage formulations for ease and feasibility of administration have been increasingly in demand. Several new oral liquid preparations were introduced, notably Xarelto (rivaroxaban) 1-mg/mL oral suspension, following pediatric approval in 2021.13 Furthermore, new formulations of preexisting liquid products were introduced for several commonly prescribed medications. Norliqva (amlodipine) 1-mg/mL oral solution presents an alternative to the preexisting amlodipine suspension, Katerzia 1 mg/mL. This new formulation differs in that it does not require shaking upon administration or refrigeration.14,15 To further expand administration options for patients and their caregivers, a new prefilled 40-mg subcutaneous syringe was introduced for Nucala (mepolizumab), indicated for the management of severe eosinophilic asthma. This the first option available for home administration in eligible patients aged 6 to 11 years.16 Lastly, Xelstrym (dextroamphetamine) transdermal patch presents the first topical formulation of dextroamphetamine approved for the management of attention deficit hyperactivity disorder in children 6 years or older.17
Labeling changes
In 2022, we continued to observe expanded indications for younger pediatric age groups. Despite initial clinical off-label use, these updated labels help support the use of medications in children safely and effectively. Below are highlights of some updates of expanded indications in pediatric patients.
Smoflipid (lipid injectable emulsion) has been used in neonates for a few years. Despite a lack of FDA approval, it is used in first-line treatment in many instances. FDA approval for neonates born preterm and at term was based on safety and efficacy evidence from multiple studies ranging from 1 to 4 weeks and one study following neonates beyond 4 weeks.
Multiple medications for HIV, particularly combination products, are now approved for use in younger pediatric patients. Genvoya (elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide) and Descovy (emtricitabine/tenofovir alafenamide) are both now approved for pediatric patients aged as young as 2 years. Triumeq PD (abacavir/dolutegravir/lamivudine) is now available in a dispersible tablet and indicated in children who weigh 10 kg or more. Pifeltro (doravirine) tablets and Delstrigo (doravirine/lamivudine/tenofovir disoproxil fumarate) tablets are approved in patients who weigh 35 kg or more. Similarly, Cabenuva (cabotegravir/rilpivirine) injection and Vocabria (cabotegravir) tablets are new agents approved for use in patients 12 years or older and have expanded labels for pediatric patients who weigh 35 kg or more.18,19
Conclusion
Novel treatments approved in 2022 provide improved therapies for patients with acute and chronic illnesses. For the pediatric patient population, novel therapies have the potential to change the long-term outcomes of chronic diseases such as T1D. New gene therapies have transformed the management of certain diseases. We continue to gain more information on the use of medications in pediatric patients through labeling changes.
Tzield (teplizumab-mzwv)10
Disease-modifying diabetic agent, monoclonal antibody
To delay the onset of stage 3 type 1 diabetes in adults and pediatric patients 8 years or older who currently have stage 2 type 1 diabetes
For use with MRI to detect and visualize lesions with abnormal vascularity in the CNS and body in adult and pediatric patients 2 years or older
Xenpozyme (olipudase alfa-rpcp)21
Enzyme replacement therapy
Hydrolytic lysosomal sphingomyelin–specific enzyme for management of non-CNS manifestations of acid sphingomyelinase deficiency in adults and pediatric patients
For the management of seizures associated with CDKL5 deficiency disorder in patients 2 years or older
Tezspire (tezepelumab-ekko)6
For management of severe asthma as add-on maintenance therapy in adults and pediatric patients 12 years and older
For management of post-transplant CMV infection/disease that is refractory to management (with or without genotypic resistance) with ganciclovir,valganciclovir, cidofovir, or foscarnet in adults and pediatric patients 12 years or older who weigh 35 kg or more
To improve growth with achondroplasia and open epiphyses in patients 5 years or older
For management of cholestatic pruritus in patients 1 year or older with Alagille syndrome
Xelstrym transdermal system (dextroamphetamine)17
For pediatric patients aged 6 to 17 years with ADHD
Opdualag (nivolumab and relatlimab-rmbw)25
For reduction of the risk of ototoxicity associated with cisplatin in pediatric patients 1 month or older with localized, nonmetastatic solid tumors
Pedmark (sodium thiosulfate)28
For reduction of the risk of ototoxicity associated with cisplatin in pediatric patients 1 month or older with localized, nonmetastatic solid tumors
Skysona (elivaldogene autotemcel)27
Autologous hematopoietic stem cell, self-inactivating Lenti-D lentiviral vector
To slow the progression of neurologic dysfunction in boys aged 4 to 17 years with early, active CALD
Zyntelgo (betibeglogene autotemcel)12
Autologous hematopoietic stem cell–based gene therapy
For the treatment of adults and pediatric patients with β-thalassemia who require regular RBC transfusions
Priorix (measles, mumps, and rubella vaccine, live)28
For active immunization to prevent measles, mumps, and rubella in patients 1 year or older.
ADHD, attention deficit hyperactivity disorder; CALD, cerebral adrenoleukodystrophy; CMV, cytomegalovirus; CNS, central nervous system; RBC, red blood cell.
Rolvedon (eflapegrastim-xnst)29
To decrease the incidence of infection with nonmyeloid malignant tumors in adults
Sotyktu (deucravacitinib)30
For the treatment of adults with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy
Spevigo (spesolimab-sbzo)31
Dermatologic agent, IL inhibitor
For the management of generalized pustular psoriasis flares in adults
For the management of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
For topical management of plaque psoriasis in adults
As an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus
For the management of hemolytic anemia in adults with PKR deficiency
Enjaymo (sutimlimab-jome)36
To decrease the need for red blood cell transfusion because of hemolysis with cold agglutinin disease in adults
Dermatologic agent, JAK inhibitor
Management of refractory, moderate to severe atopic dermatitis in adults whose disease is not adequately controlled with other therapies
For the management of insomnia characterized by difficulties with sleep onset and/or sleep maintenance in adults
Adbry (tralokinumab-ldrm)1
Dermatologic agent, IL inhibitor
For management of moderate to severe AD in adults whose disease is not well controlled with topical prescription therapies
Small interfering RNA therapy
To manage heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease as an add-on therapy in adults
Besremi (ropeginterferon alfa-2b-njft)39
For management of polycythemia vera in adults
Peptide receptor antagonist
For the preventive management of episodic migraine in adults
Rebyota (fecal microbiota, live-jslm)41
Suspension for rectal use
For prevention of recurrence of CDI in adults following antibiotic treatment
Hemgenix (etranacogene dezaparvovec-drlb)42
Adeno-associated virus vector-based gene therapy
For treatment of adults with hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes
AD, atopic dermatitis; CDI, Clostridioides difficile infection.
40-mg prefilled syringe for home administration
For treatment of children aged 6 to 11 years who have severe eosinophilic asthma
Takhzyro (lanadelumab-flyo)43
300-mg/2-mL prefilled syringe
For prevention of hereditary angioedema attacks in children 12 years and older
For management of human smallpox disease
Menveo (meningococcal [groups A, C, Y, and W-135]oligosaccharide diptheria CRM197 conjugate vaccine)45
For prevention of meningococcal disease in children 10 years and older
Rotarix (rotavirus vaccine, live, oral)46
For prevention of rotavirusgastroenteritis caused by G1 and non-G1 types (G3, G4 and G9) in infants
Oral suspension (1 mg/mL)
For management of VTE and reduction in the risk of recurrent VTE in pediatric patients and thromboprophylaxis in pediatric patients 2 years or older with congenital heart disease after the Fontan procedure
Oral suspension (70 mg/mL)
For treatment of pediatric patients 1 year or older with cGVHD after failure of 1 or more lines of systemic therapy
Triumeq PD (abacavir/dolutegravir/lamivudine)48
For management of HIV-1 infection in patients weighing 10 kg or more
Oral suspension (5 mg/mL)
For the management of spasticity resulting from multiple sclerosis, particularly for the relief of flexor spasms and concomitant pain, clonus, and muscular rigidity
For the management of hypertension in children 6 years or older
Oral solution (100 mg/5 mL)
For adjunctive therapy in patients 16 years or older with epilepsy
Thyroid hormone replacement
For management of hypothyroidism and pituitary thyrotropin suppression in adults and pediatric patients, including neonates
cGVHD, chronic graft-vs-host disease; VE, venous thromboembolism.
For management of obesity in adults
Approved in patients 12 years or older
Potassium channel blocker
For management of LEMS in adults
Approved in pediatric patients 6 years or older
Lyumjev (insulin lispro-aabc)
For treatment of adults with type 1 and type 2 diabetes
Approved in children with diabetes 1 year or older
Orkambi (lumacaftor/ivacaftor)
Cystic fibrosis transmembrane regulator
For treatment of patients aged 2-5 years with cystic fibrosis who have 2 F508del mutations
Approved in patients aged 12-14 months
For treatment of patients 6 years or older with moderate to severe atopic dermatitis and adults with eosinophilic esophagitis
Safety and effectiveness established in pediatric patients 6 months or older with moderate to severe atopic dermatitis and in patients 12 years or older for eosinophilic esophagitis
For the management of active psoriatic arthritis in adults
Approved in pediatric patients 6 years or older
Xofluza (baloxavir marboxil)
For postexposure prophylaxis of influenza in adults
Approved in patients 5 years or older
For management of seizures associated with Dravet syndrome in patients 2 years or older
Approved for additional indication of management of seizures associated with Lennox-Gastaut syndrome in patients 2 years or older
For the management of active lupus nephritis in adults
Approved for the treatment of children aged 5-17 years
Three-day regimen for the prevention of nausea and vomiting in adults
Approved in pediatric patients aged 6 months to 17 years
Zerbexa (ceftolozane/tazobactam)
For the management of complicated intraabdominal infections and complicated urinary tract infections in adults (safety and effectiveness established)
Approved in all pediatric patients
Triumeq (abacavir/dolutegravir/lamivudine52
For management of HIV-1 infection in adult patients
For the management of HIV-1 infection in patients weighing 25 kg or more
Comirnaty (COVID-19 vaccine, mRNA)
For active immunization to prevent COVID-19 caused by SARS-CoV-2 in patients 16 years or older
Approved in patients aged 12-15 years
Qsymia (phentermine/ topiramate)
For management of chronic weight in adult patients in addition to a reduced-calorie diet and exercise
Approved for management of chronic weight in pediatric patients 12 years or older who have obesity, defined as a BMI of 95th percentile or greater when standardized for age and sex
Vaxneuvance (pneumococcal 15-valent conjugate vaccine)
For the prevention of pneumococcal disease in adults
Approved for the prevention of pneumococcal disease in patients 2 months or older
For the management of refractory, moderate to severe atopic dermatitis in patients whose disease is not adequately controlled with other systemic drug products including biologics or when use of those therapies is inadvisable
Approved in patients 12 years or older
For the management of COVID-19 in adults who are hospitalized or not hospitalized and are at high risk for progression to severe COVID-19, including hospitalization or death
Approved in pediatric patients 28 days or older and weighing 3 kg or more
For the management of spinal muscular atrophy in patients 2 months or older
Approved in all patients with spinal muscular atrophy
CellCept (mycophenolate mofetil)
For the prophylaxis of organ rejection in adults with heart or liver transplants and in adult and pediatric patients with kidney transplants in combination with other immunosuppressants
Expanded indication to include pediatric patients 3 years or older with liver and heart transplants
For anticoagulation in all adult patients
Indicated for the management of VTE and reduction in the risk of recurrent VTE in pediatric patients after 5 days or more of initial parenteral (injected or intravenous) anticoagulant treatment and for thromboprophylaxis (prevention of blood clots and blood clot–related events) in children 2 years or older with congenital heart disease who have undergone the Fontan procedure
Smoflipid (lipid injectable emulsion)
For adult patients as a source of calories and essential fatty acids for parenteral nutrition when oral or enteral nutrition is not possible, insufficient, or contraindicated
Expanded indication to all pediatric patients including neonatal patients born at term and preterm
NephroScan kit (technetium Tc99m succimer)
Indicated for use as an aid in the scintigraphic evaluation of renal parenchymal disorders
Indicated for use as an aid in the scintigraphic evaluation of renal parenchymal disorders in pediatric patients, including neonates born at term
Genvoya (elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide)
For the management of HIV-1 in patients 6 years or older
Management of HIV-1 in patients 2 years or older
Descovy (emtricitabine/tenofovir alafenamide)
For the management of HIV-1 in patients weighing 35 kg or more
Management of HIV-1 infection in pediatric patients 2 years or older weighing 14 kg or more
Ryaltris nasal spray (olopatadine hydrochloride/mometasone furoate)
For management of symptoms associated with seasonal allergic rhinitis in adults
Indicated for pediatric patients 12 years or older
Solosec oral granules (secnidazole)
For management of trichomoniasis and bacterial vaginosis in adults
Indicated for pediatric patients 12 years or older
Pifeltro tablets (doravirine)
For management of HIV-1 infection in adults
Indicated for pediatric patients weighing 35 kg or more
Delstrigo tablets (doravirine/lamivudine/tenofovir disoproxil fumarate)
For management of HIV-1 infection in adults
Indicated for pediatric patients weighing 35 kg or more
Opdualag injection (nivolumab/relatlimab-rmbw)
For management of unresectable or metastatic melanoma in adults
Indicated for pediatric patients 12 years or older weighing 40 kg or more
For the management of CLL in adults
Indicated for pediatric patients 1 year or older with cGVHD after failure of 1 or more lines of systemic therapy
Cabenuva injection (cabotegravir/rilpivirine)
For management of HIV-1 infection in adults
Indicated for pediatric patients 12 years or older weighing 35 kg or more
Vocabria tablets (cabotegravir)
For management and preexposure prophylaxis of HIV-1 infection in adults
Indicated for adolescents 12 years or older weighing 35 kg or more
Zulresso injection (brexanolone)
Antiviral agentAntiviral agent
For management of postpartum depression in adults
Indicated for patients 15 years or older
Halaven injection (eribulin mesylate)
For management of metastatic breast cancer and liposarcoma in adult patients
Treatment of pediatric patients with relapsed or refractory solid tumors and lymphomas
For treatment of adults with MDS subtypes: RA or RARS and CMMoL
Treatment of pediatric patients 1 month or older with newly diagnosed JMML
For the treatment of adults with unresectable or metastatic melanoma with BRAF V600E or V600K mutations as single agent and in combination with dabrafenib
Management (in combination with dabrafenib) of unresectable or metastatic solid tumors with BRAF V600E mutation that have progressed following prior treatment in adults and children 6 years and older
For the management of adults with unresectable or metastatic melanoma with BRAF V600E or V600K mutations as single agent and in combination with trametinib
Management (in combination with trametinib) of unresectable or metastatic solid tumors with BRAF V600E mutation that have progressed following prior treatment in adults and children 6 years and older
For the treatment of patients with metastatic NSCLC whose tumors are ALK+ or ROS1+
Treatment of adults and pediatric patients 1 year or older with unresectable, recurrent, or refractory IMT that is ALK+
Adcetris (brentuximab vedotin)
For management (in combination with chemotherapy) of adult Hodgkin and anaplastic large cell lymphoma and of cHL at high risk of relapse or progression as post autologous HSCT consolidation
Treatment of pediatric patients 2 years or older with previously unmanaged high-risk cHL in combination with doxorubicin, vincristine, etoposide, prednisone, and cyclophosphamide
For management of metastatic NSCLC in adults who experience disease progression during or following platinum-containing chemotherapy
As a single agent for the treatment of adult and pediatric patients 2 years or older with unresectable or metastatic ASPS
ASPS, alveolar soft part sarcoma; BMI, body mass index; cGVHD, chronic graft-vs-host disease; cHL, classical Hodgkin lymphoma; CLL, chronic lymphocytic leukemia; CMMoL, chronic myelomonocytic leukemia; HSCT, hematopoietic stem cell transplantation; IMT, inflammatory myofibroblastic tumor; JMML, juvenile myelomonocytic leukemia; LEMS, Lambert-Eaton myasthenic syndrome; MDS, myelodysplastic syndrome; NSCLC, non–small cell lung cancer; RA, refractory anemia; RARS, RA with ring sideroblasts.
References
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