Infectious Disease
Livtencity (maribavir)
Maribavir is an antiviral that elicits its activity by competitive inhibition of the protein kinase activity of human cytomegalovirus (CMV) enzyme pUL97, which inhibits protein phosphorylation. It is indicated for treatment of posttransplant CMV infection/disease that is refractory to treatment with or without genotypic resistance (eg, ganciclovir, valganciclovir, cidofovir, foscarnet) in adults and pediatric patients aged 12 years and older who weigh at least 35 kg.
The phase 3 SOLSTICE trial randomized hematopoietic-cell and solid-organ transplant recipients 2:1 to receive maribavir (n = 235) or investigator-assigned therapy (IAT; valganciclovir/ganciclovir, foscarnet, or cidofovir [n = 117]). Significantly more patients in the maribavir versus IAT group achieved CMV clearance by Week 8 (55.7% vs 23.9%; P<.001) and maintained through Week 16 (18.7% vs 10.3%; P=.01). Clin Infect Dis. 2021 Dec 2
Fexinidazole
Fexinidazole is a nitroimidazole antiprotozoal drug indicated for treatment of African trypanosomiasis (HAT; sleeping sickness) caused by Trypanosoma brucei gambiense in adults and children aged 6 years and older who weigh at least 20 kg. It is indicated for stage 1 (hemolymphatic) and stage 2 (meningoencephalitic) HAT.
Results from 2 single-arm trials in adults and pediatric patients aged 6-15 years supported the approval by the FDA. Fexinidazole demonstrated efficacy of nearly 100% at 12 months. Lancet Glob Health. 2021 Jul;9(7)
TicoVac (tick-borne encephalitis vaccine)
Tick-borne encephalitis vaccine is indicated for adults and children aged 1 year and older for prevention of tick-borne encephalitis caused by Flaviviridae, including European or Western tick-borne encephalitis virus (transmitted by Ixodes ricinus), Siberian tick-borne encephalitis virus (transmitted by I persulcatus), and Far-Eastern tick-borne encephalitis virus, formerly known as Russian spring-summer encephalitis virus (transmitted by I persulcatus). It is administered as a 3-dose primary series.
Seropositivity rates at Day 21 after the third vaccination were above 99% for all age groups in Study 209 and above 98% in Study 213 and Study 690601. Three years after the primary 3-dose series, seropositivity ranged from 82.9 to 100% depending on age. Prescribing Information
Tembexa (brincidofovir)
Brincidofovir is a prodrug of cidofovir. Brincidofovir effectively penetrates cells via its lipid conjugate, releasing the nucleotide analog cidofovir, which then acts to inhibit viral replication. Cidofovir diphosphate selectively inhibits orthopoxvirus DNA polymerase–mediated viral DNA synthesis by incorporation of cidofovir into the growing viral DNA chain. This results in reductions in the rate of viral DNA synthesis. It is indicated for treatment of human smallpox disease caused by variola virus in adult and pediatric patients, including neonates.
The drug gained approval under the FDA’s animal rule. Approval was based on efficacy data in 2 lethal orthopoxvirus animal models of human smallpox disease: the rabbit pox model (New Zealand white rabbits infected with rabbit pox virus) and the mouse pox model (BALB/c mice infected with ectromelia virus). In the pivotal studies in each model, brincidofovir resulted in statistically significant survival benefit versus placebo following delayed treatment after animals were infected with a lethal viral dose. Tembexa prescribing information
COVID-19 Emergency Use Authorizations
Comirnaty ( COVID-19 vaccine, mRNA-Pfizer )
COVID-19 vaccine, mRNA-Pfizer is the first vaccine to gain full approval in the United States for prevention of COVID-19 disease for individuals aged 16 years and older. Since its August 2021 approval, the emergency use authorization for the vaccine has been amended to include children aged 5 years and older, third-dose administration for those aged 12 years and older who are immunocompromised, and booster doses for individuals aged 16 years and older.
Approval was based on a multinational phase 3 trial (n = 43,448). Vaccine efficacy was 95% against the original SARS-CoV-2 strain at 7 days after dose 2, and no serious safety concerns were observed. Updated data through 6 months (March 13, 2021) showed vaccine efficacy against COVID-19 was 91.3%. Vaccine variants (eg, delta) and waning efficacy prompted implementation of adding a booster dose to be administered 6 months after the 2-dose primary series. N Engl J Med 2021;385:1761-1773
Emergency use authorizations (EUAs) for COVID-19 vaccine, mRNA-Pfizer in children
Homologous booster dose authorized for adolescents aged 12-17 years at least 5 months after their primary 2-dose series with the Pfizer COVID-19 vaccine (not for heterologous use in this age group).
2-dose primary series approved for children aged 5 years and older.
Third primary-series dose at least 1 month after second dose in individuals aged 5 years and older who have undergone solid-organ transplantation or have a condition considered to have equivalent level of immunocompromise.
Additional COVID-19 EUAs
Oral antiviral
Paxlovid (nirmatrelvir/ritonavir) - Treatment of mild-to-moderate COVID-19 in high-risk adults and adolescents aged 12 years and older who test positive.
Monoclonal antibodies
Treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in high-risk adults and children, including neonates.
Postexposure prophylaxis for high-risk adults and children, including neonates who are not fully vaccinated or are not expected to mount an adequate immune response.
REGEN-COV (casirivimab/imdevimab)
Treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in high-risk individuals aged 12 years and older.
Postexposure prophylaxis (PEP) for high-risk individuals aged 12 years and older who are not fully vaccinated or are not expected to mount an adequate immune response.
Treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in high-risk individuals aged 12 years and older.
Evusheld (tixa genmab and cilgavimab)
Preexposure prophylaxis of individuals aged 12 years and older who are moderately to severely immunocompromised because of a medical condition or medications/treatment and may not mount an adequate immune response to COVID-19 vaccination or who have a history of severe adverse reactions to a COVID-19 vaccine and/or component(s).
Note: Depending on viral variants, distribution of certain monoclonal antibodies may be paused because of decreased efficacy.
Other pediatric infectious disease approvals
Dalvance (dalbavancin) - Indication for acute bacterial skin and skin structure infections (ABSSSI) caused by susceptible gram-positive bacteria in pediatric patients, including neonates.
Apretude (cabotegravir) - Every 2 month IM administration for preexposure prophylaxis (PrEP) in adults and adolescents weighing at least 35 kg to reduce the risk of sexually acquired HIV-1 infection.
Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide) - Indicated as a complete regimen for treatment of HIV-1 infection in adults and pediatric patients weighing at least 14 kg who have no ART treatment history or to replace a current ART regimen.
Natroba (spinosad) - New indication approved for scabies in adults and children aged 4 years and older.
Rapivab (peramivir) - Indication for acute uncomplicated influenza expanded to include children as young as 6 months.
Vaxchora (cholera vaccine) - Use for immunization against Vibrio cholerae serogroup 01 expanded to include children aged 2 years and older.
Cardiology
Evkeeza (evinacumab)
Evinacumab is a recombinant human monoclonal antibody that binds to and inhibits angiopoietin–like 3 (ANGPTL3). ANGPTL3 inhibits lipoprotein lipase and endothelial lipase, thereby reducing lipid metabolism. Evinacumab inhibits ANGPTL3 and results in increased lipid metabolism, leading to decreased low-density lipoprotein cholesterol (LDL-C), high-density lipoprotein cholesterol, and triglycerides. Evinacumab is indicated as an adjunct to other LDL-C–lowering therapies for homozygous familial hypercholesterolemia in adults and adolescents aged 12 years and older.
Approval was based on the phase 3 ELIPSE trial (n = 65). The study found that patients undergoing stable lipid-lowering treatment in whom an intravenous infusion of evinacumab was administered every 4 weeks achieved, by week 24, a 47.1% relative reduction in their LDL-C level, as compared to a 1.9% increase in patients given placebo (P<.001). N Engl J Med. 2020;383:711-720
Other pediatric cardiology approvals
Repatha (evolocumab) - Indicated as an adjunct to diet and other LDL-lowering therapies (eg, statins, ezetimibe, LDL apheresis) for the treatment of children aged 10 years and older with heterozygous or homozygous familial hypercholesterolemia (HeFH, HoFH) who require additional LDL-C reduction.
Xarelto (rivaroxaban) - Treatment of venous thromboembolism (VTE), or blood clots that form in the veins, and reduction in the risk of VTE recurring in pediatric patients from birth to younger than 18 years who have received at least 5 days of injectable or intravenous treatment for blood clots.
Arcalyst (rilonacept) - Interleukin-1 inhibitor for treatment of recurrent pericarditis and to reduce the risk of future recurrence in adults and children aged 12 years and older.
Pradaxa (dabigatran) - First oral anticoagulant approved for children to treat and prevent the recurrence of venous thromboembolic events.
Diovan (valsartan) - Indication for pediatric hypertension expanded to include children as young as 1 year.
Psychiatry
Azstarys (serdexmethylphenidate/dexmethylphenidate)
Serdexmethylphenidate/dexmethylphenidate is a fixed-dose combination of the prodrug of dexmethylphenidate, serdexmethylphenidate, and immediate-release dexmethylphenidate. This combination provides extended drug levels. It is indicated for attention deficit hyperactivity disorder (ADHD) in adults and children aged 6 years and older. Efficacy was assessed by Swanson, Kotkin, Agler, M-Flynn, and Pelham (SKAMP)–combined scores, as compared to baseline and at 0.5, 1, 2, 4, 8, 10, 12, and 13 hours post dose. Results showed statistically significant improvement of SKAMP-combined scores compared to placebo. Azstarys prescribing information
Qelbree (viloxazine)
Viloxazine is indicated for treatment of attention deficit hyperactivity disorder (ADHD) in children and adolescents aged 6-17 years. The mechanism of action by which viloxazine affects ADHD is unclear; however, it may be by selectively inhibiting norepinephrine reuptake. Approval was based on 3 phase-3 placebo-controlled trials that included over 1000 participants. Patients taking viloxazine had statistically significant improvement in ADHD rating scale 5 and Clinical Global Impressions I scores as compared to placebo. Qelbree prescribing information
Other pediatric psychiatry approvals
Rexulti (brexpiprazole) - Indication for schizophrenia expanded to include adolescents aged 13-17 years.
Kloxxado (naloxone intranasal) - New formulation that delivers 8 mg per actuation, compared with older products that deliver 2 mg or 4 mg per actuation.
Evekeo ODT (amphetamine) - Indication for attention deficit hyperactivity disorder (ADHD) expanded to include children as young as 3 years (previously approved for children aged 6-17 years).
Hematology-oncology
Rezurock (belumosudil)
Belumosudil is a rho-associated, coiled-coil–containing protein kinase (ROCK) inhibitor. It has been shown to down-regulate proinflammatory responses via regulation of STAT3/STAT5 phosphorylation and shifting Th17/Treg balance in ex vivo or in vitro human T-cell assays. It is indicated for graft-versus-host disease (GVHD) in patients aged 12 years and older after failure of at least 2 prior lines of systemic therapy.
Approval was based on safety and efficacy results from ROCKstar, a randomized, open-label, multicenter, phase 2 trial of 66 patients with chronic GVHD (cGVHD) who received 2 to 5 prior lines of systemic therapy. The median time from cGVHD diagnosis was 25.3 months, and 48% of patients had 4 or more organs involved. Overall, median follow-up was 14 months. The best ORR (95% CI) of belumosudil 200 mg QD and 200 mg BID was 74% (62-84%) and 77% (65-87%), respectively, with high response rates observed in all subgroups. Blood. 2021 Dec 2;138(22):2278-2289
Rylaze (asparaginase Erwinia chrysanthemi recombinant)
Asparaginase Erwinia chrysanthemi recombinant is indicated as part of a chemotherapeutic regimen for acute lymphoblastic leukemia or lymphoblastic lymphoma in adults and children aged 1 month or older who have developed hypersensitivity or silent inactivation to Escherichia coli–derived asparaginase.
Ryplazim (plasminogen)
Plasminogen is the first therapy approved for plasminogen deficiency type 1 (hypoplasminogenemia). It is a plasma-derived human plasminogen that temporarily increases plasminogen blood levels. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system, blood clot lysis, and extravasated fibrin clearance.
Other pediatric hematology-oncology approvals
Cabometyx (cabozantinib) – Indicated for locally advanced or metastatic differentiated thyroid cancer (DTC) in adults who have progressed following prior VEGFR-targeted therapy and who are radioactive iodine refractory or ineligible.
Keytruda (pembrolizumab) - Adjuvant treatment of pediatric patients aged 12 years and older with stage IIB, IIC, or III melanoma following complete resection.
Tasigna (nilotinib) - Indicated for newly diagnosed Ph+ chronic myeloid leukemia (Ph+ CP-CML) in children aged 1 year and older. Also indicated for children with Ph+ CP-CML who have accelerated phase-resistant disease or are intolerant to prior TKI therapy.
Rituxan (rituximab) - New indication in combination with chemotherapy for pediatric patients (6 months to <18 years) with previously untreated, advanced-stage, CD20-positive diffuse large B-cell lymphoma (DLBCL), Burkitt lymphoma (BL), Burkitt-like lymphoma (BLL), or mature B-cell acute leukemia (B-AL).
Jakafi (ruxolitinib) - Indicated for treatment of steroid-refractory acute graft-versus-host disease (GVHD).
Orencia (abatacept) - First drug indicated for the prophylaxis of acute graft-versus-host disease (aGvHD), in combination with a calcineurin inhibitor and methotrexate, in adults and pediatric patients aged 2 years and older undergoing hematopoietic stem cell transplantation (HSCT) from a matched or 1 allele-mismatched unrelated donor.
Injectafer (ferric carboxymaltose) - Indication for iron deficiency anemia expanded to include children aged 1 year and older.
Oxbryta (voxelotor) - Indication for sickle cell disease expanded to include children aged 4-11 years.
Vyxeos (cytarabine/daunorubicin liposomal) - Indication for newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes expanded to include children aged 1 year and older.
Lymphoseek (technetium Tc 99m tilmanocept) - Indication for lymphatic mapping expanded to include children aged 1 year and older with solid tumors for which this procedure is a component of intraoperative management.
Ferriprox (deferiprone) - Indication for transfusional iron overload expanded to include children aged 3 years and older with sickle cell anemia or other anemias.
Neurology
Amondys 45 (casimersen)
Casimersen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMO binds to exon 45 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Casimersen is indicated for Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 45 skipping.
The ESSENCE trial—a placebo-controlled confirmatory trial to support the casimersen approval—is ongoing and expected to conclude in 2024. In the study, patients who received casimersen showed a significantly greater increase in dystrophin protein levels from baseline to Week 48 of treatment, as compared to those who received placebo. Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)
Nulibry (fosdenopterin)
Fosdenopterin is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency type A (MoCD-A), a rare disease. Fosdenopterin provides an exogenous source of cyclic pyranopterin monophosphate (cPMP). MoCD-A is caused by a mutation in the molybdenum cofactor synthesis 1 gene (MOCS1), which causes a deficiency in molybdenum cofactor production. The exogenous intermediate substrate cPMP provided by the drug is subsequently converted to molybdenum cofactor, which is required for the activation of essential enzymes (eg, sulfite oxidase) to reduce the levels of neurotoxic sulfites. Decreased sulfite oxidase activity is thought to cause the severe and rapidly progressive CNS damage observed in patients with MoCD-A.
Other pediatric neurology approvals
Briviact (brivaracetam) ; Vimpat (lacosamide) - Indications for partial seizure expanded to include children aged 1 month and older (previously approved for those aged 4 years and older).
Fabrazyme (agalsidase) - Indication for Fabry disease expanded to include children aged 2 years and older (previously approved for those aged 8 years and older).
Carbaglu (carglumic acid) - Approval granted for adjunctive therapy to standard of care for acute hyperammonemia due to propionic acidemia or methylmalonic acidemia in adults and children.
Gastroenterology
Bylvay (odevixibat)
Odevixibat is a first-in-class ileal bile acid transport inhibitor (IBATi). It acts locally in the distal ileum to decrease reuptake of bile acids and increase clearance of bile acids through the colon, thereby reducing bile acid serum concentration. It is indicated for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) who are aged 3 months and older.
Approval by the FDA was supported by data from the Demonstrate Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis (PEDFIC) Types 1 and 2 phase 3 trials. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, odevixibat showed improvement of pruritus and serum bile acids as compared to placebo. PEDFIC 2, a long-term, open-label phase 3 extension study, showed sustained reductions in serum bile acids as well as improvements in pruritus assessments, growth, and other markers of liver function in patients treated for up to 48 weeks. AASLD The Liver Meeting 2021. 2021 Nov 12-15
Livmarli (maralixibat)
Maralixibat is another ileal bile acid transport inhibitor (IBATi) that gained approval for cholestatic pruritus in adults and children aged 1 year and older with Alagille syndrome. FDA approval was based on the ICONIC study and 5 years of data from supportive studies in 86 patients with Alagille syndrome. On average, patients who were given maralixibat for 22 weeks maintained pruritus reduction, whereas those in the placebo group who were withdrawn from maralixibat after week 18 returned to baseline pruritus scores by week 22. Lancet. 2021 Oct 30;398(10311):1581-1592 .
Other pediatric gastroenterology approvals
Humira (adalimumab) - Indication for ulcerative colitis expanded to include children aged 5 years and older.
Epclusa (sofosbuvir/velpatasvir) - Indication for chronic hepatitis C virus infection expanded to include children as young as 3 years.
Mavyret (glecaprevir/pibrentasvir) - Indication expanded to include children aged 3 years and older with chronic hepatitis C virus infection.
Endocrinology
Nexviazyme (avalglucosidase alfa)
Avalglucosidase is a recombinant acid alpha-glucosidase (GGA) indicated for late-onset Pompe disease in patients aged 1 year and older. GGA is deficient in patients with Pompe disease, which leads to a buildup of glycogen in skeletal and cardiac muscle cells, causing weakness and premature death from respiratory failure or heart failure.
Approval was based on the COMET phase 3 trial that compared avalglucosidase alfa (n = 51) with alglucosidase alfa (n = 49). Clinically meaningful improvement with avalglucosidase-alfa therapy over alglucosidase alfa was observed in respiratory function, ambulation, and functional endurance. Lancet Neurol. 2021 Dec;20(12):1012-1026
Voxzogo (vosoritide)
Vosoritide, an analog of C-type natriuretic peptide (CNP), is indicated to increase linear growth in pediatric patients aged 5 years or older who have achondroplasia with open epiphyses.
CNP binds to natriuretic-peptide receptor B (NPR B), which induces synthesis of cGMP (cyclic guanosine monophosphate) molecules, which, in turn, inhibits the MAPK pathway. Inhibition of the MAPK pathway leads to increased extracellular matrix (ECM), which, in conjunction with chondrocytes, serves as a template for bone via endochondral ossification. Achondroplasia is caused by a mutation in the gene that encodes for fibroblast growth factor receptor 3 (FGFR3), causing it to be permanently active; FGFR3 signaling activates 2 intracellular signaling cascades that lead to a lower proliferation and differentiation of bone growth plate chondrocytes, through the STAT-1 pathway, and to a lower production of ECM through the MAPK pathway.
Approval was supported by outcomes of a global randomized, double-blind, placebo-controlled phase 3 study evaluating efficacy and safety and the open-label extension of this phase 3 study. The study enrolled 121 children aged 5-14 years with achondroplasia. Baseline mean annual growth velocity (AGV) in the placebo and vosoritide groups was 4.06 cm/yr and 4.26 cm/yr, respectively. At week 52, the change from baseline in AGV was -0.17 cm/yr for the placebo-treated patients and 1.40 cm/yr for the vosoritide-treated patients, resulting in a statistically significant improvement in AGV of 1.57 cm/yr. After the 52-week double-blind, placebo-controlled, phase 3 study, 58 patients initially randomized to vosoritide enrolled in an open-label extension. Improvement in AGV was maintained at 2 years. Lancet. 202 Sep 5;396(10252):685-692 ; Genet Med. 2021 Dec;23(12):2443-2447
Rethymic (allogeneic processed thymus tissue)
Allogeneic processed thymus tissue is the first therapy approved to reconstitute immunity in children with congenital athymia (ie, DiGeorge syndrome).
Approval was based on 10 prospective single-arm, open-label studies that included 105 patients from 1993 to 2020. Survival rates were analyzed, with the longest follow-up period of 25.5 years. In the Efficacy Analysis Set (EAS), Kaplan-Meier estimated survival rates (95% CI) were 77% (0.670-0.841) at 1 year and 76% (0.658-0.832) at 2 years. For patients who were alive at 1 year post tissue implantation, the estimated long-term survival rate was 94% at a median follow-up of 10.7 years. For the patients in the clinical trials, naive T-cell levels were measured using flow cytometry at 6, 12, and 24 months after implantation. Patients in the clinical trials started out with very few naive T cells, but naive CD4 and CD8 T cells began to reconstitute over the first year, with a durable increase through the second year. Reductions in the number of infections over time during the first 2 years after treatment were statistically significant (P<0.001). Prescribing information.
Other pediatric endocrinology approvals
Bydureon, Bydureon BCise ( exenatide injectable suspension) - Indicated as an adjunct.
Gvoke (glucagon) - Indicated for severe hypoglycemia in patients with diabetes treated with insulin. Approved for patients aged 2 years and older (previously for those aged 6 years and older).
Pulmonology
Tezspire (tezepelumab)
Tezepelumab is a first-in-class human monoclonal antibody immunoglobulin G2-lambda that inhibits thymic stromal lymphopoietin (TSLP). It is indicated for add-on maintenance treatment of severe asthma in adults and pediatric patients aged 12 years and older.
Approval was based on the NAVIGATOR phase 3, multicenter, randomized, double-blind, placebo-controlled trial. Patients (aged 12 to 80 years) were randomly assigned to receive tezepelumab (210 mg) or placebo subcutaneously every 4 weeks for 52 weeks. Of the 1061 patients who underwent randomization, 529 were assigned to receive tezepelumab and 532 to receive placebo. The annualized rate of asthma exacerbations was 0.93 with tezepelumab and 2.10 with placebo (P<0.001). Additionally, in patients with a blood eosinophil count less than 300 cells per microliter, the annualized rate was 1.02 with tezepelumab and 1.73 with placebo (P<0.001). At week 52, improvements were greater with tezepelumab than with placebo with respect to the prebronchodilator FEV1 (0.23 vs 0.09 liters; P<0.001). N Engl J Med. 2021 May 13;384(19):1800-1809
Other pediatric pulmonology approvals
Dupixent (dupilumab) - Indication for moderate-to-severe asthma expanded to include children aged 6-11 years.
ArmonAir Digihaler (fluticasone inhaled) - Indication for asthma expanded to include children aged 4 years and older (previously for those 12 years and older).
Dermatology
Opzelura (ruxolitinib topical) - First JAK inhibitor topical dosage form. Indicated for topical short-term and noncontinuous long-term treatment of mild-to-moderate atopic dermatitis in nonimmunocompromised patients aged 12 years and older.
Cosentyx (secukinumab) - Indicated for moderate-to-severe plaque psoriasis in patients aged 6 years and older who are candidates for systemic therapy or phototherapy.
Twyneo (tretinoin/benzoyl peroxide) - New topical combination for acne vulgaris in patients aged 9 years and older.
Pain Management
Exparel (bupivacaine liposomal) - New indication for postsurgical local analgesia expanded to include children aged 6 years and older.
Zipsor (diclofenac) - Indication for acute mild-to-moderate pain now includes adolescents.
Other pediatric approvals
Estetrol/drospirenone - Indicated for use by females of reproductive potential to prevent pregnancy. The combination is the first to contain estetrol (E4), a synthetic analog of native estrogen that is present during pregnancy. It is selective for nuclear estrogen receptor (ER)–alpha and ER-beta. Treatment results in the decrease of follicle-stimulating hormone and luteinizing hormone, ultimately leading to ovulation suppression.
Dasiglucagon - Ready-to-use, stable glucagon analog indicated for severe hypoglycemia in pediatric and adult patients with diabetes mellitus. It is a glucagon receptor agonist that increases blood glucose concentration by activating hepatic glucagon receptors, thereby stimulating glycogen breakdown and release of glucose from the liver.
Prograf (tacrolimus) - FDA approval for prophylaxis of organ rejection in lung transplant recipients in combination with other immunosuppressants.
Verkazia (cyclosporine ophthalmic) - Indicated for vernal keratoconjunctivitis in adults and children aged 4 years and older.
Ragwitek (ragweed allergen extract) - Indication for immunotherapy for short ragweed pollen–induced allergic rhinitis expanded to include children as young as 5 years.
Children’s Astepro Allergy (azelastine) - The 0.15% nasal spray is now available as an over-the-counter treatment for seasonal/perennial rhinitis in children aged 6 years and older.
Myrbetriq (mirabegron) - FDA approval for neurogenic detrusor overactivity in children aged 3 years and older.
Botox (onabotulinumtoxinA) - New indication approved for neurogenic detrusor overactivity in children aged 5 years and older who have an inadequate response to or are intolerant of anticholinergic medications.
Cosentyx (secukinumab) - Indication expanded for moderate-to-severe plaque psoriasis to include children aged 6 years and older who are candidates for systemic therapy or phototherapy.
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Cite this: FDA Approvals, Highlights, and Summaries: Pediatrics - Medscape - Feb 17, 2022.
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