publications

The World Health Organization (WHO) issued its first global guidelines addressing the integrated management of obesity in children and adolescents. The guidance strongly recommends structured interventions that include healthy nutrition, physical activity and behavior-change strategies, delivered either individually or through a comprehensive multimodal program. Digital health interventions may also be used, provided they include appropriate parent or caregiver involvement (conditional recommendation). For children younger than 10 years of age, the WHO does not recommend pharmacotherapy, bariatric surgery or weight-loss devices. For adolescents 10–19 years of age, use of pharmacotherapy is considered only when intensive lifestyle interventions have not achieved adequate results. Bariatric surgery should be reserved for adolescents with severe obesity and considered only under carefully defined clinical circumstances.

A retrospective cross-sectional study published in Pediatrics evaluated trends in glucagon-like peptide-1 receptor agonist (GLP-1 RA) prescribing among children 8–11 years of age with obesity and without diabetes using a large United States (U.S.) electronic health record database that included more than 3.5 million children. Data were sourced from the Epic Cosmos Data Science Virtual Machine and analyzed in the form of repeated annual cohorts. The analysis included prescriptions for the GLP-1 RAs liraglutide (Saxenda), semaglutide (Wegovy) and tirzepatide (Zepbound). Obesity was defined as a body mass index (BMI) at or above the 95th percentile for age and sex according to the Centers for Disease Control and Prevention (CDC) 2000 Growth Charts. The investigators found that GLP-1 RA prescribing increased dramatically between 2019 and 2026. By 2026, 9.3% of children in the study population had received a GLP-1 RA prescription, compared with 0.03% in 2019, demonstrating a 310-fold increase (P<0.001). Prescribing was more common among older children, females, children with lower social vulnerability, children with severe obesity and those with obesity-related comorbidities. The most frequently prescribed initial GLP-1 RA was semaglutide (Wegovy). The authors noted that, although overall use across the entire study period remained low (0.6%), the rapid growth in prescribing suggests increasing off-label use of GLP-1 RAs in younger children. Wegovy and Saxenda are the only FDA-approved GLP-1 RAs for children and adolescents 12–17 years of age for weight management. Additional evidence regarding the safety, effectiveness and appropriate role of these agents in this age group is warranted.

A retrospective claims analysis published in Childhood Obesity evaluated nutritional deficiencies and nutrition therapy/counseling (NT/C) in pediatric patients receiving GLP-1 RAs. From an initial Inovalon Insights Claims Database population of over 109 million individuals, 2,031 pediatric patients 10–17 years of age who initiated a GLP-1 RA between January 1, 2017, and December 31, 2022, were included in the analysis. Patients with type 1 diabetes mellitus (T1DM) or diagnoses related to nutritional deficiency or associated complications in the six months prior were excluded. GLP-1 RAs initiated during the study period included dulaglutide, exenatide, liraglutide, lixisenatide and semaglutide (tirzepatide was not FDA-approved during the study period). Patients in the identified cohort (n=2,031) had a mean age of 15 years, and the majority were female (61.5%) and had obesity (62.6%) and/or type 2 diabetes mellitus (T2DM; 67.2%). The most prescribed GLP-1 RA was liraglutide (78.6%), followed by dulaglutide (10.4%) and semaglutide (9.1%). Patients were identified as having a nutritional deficiency based on the International Classification of Diseases, 10th edition (ICD-10) codes for up to one year following GLP-1 RA initiation. Nearly 17% of patients were diagnosed with at least one nutritional deficiency or deficiency-related complication within one year of GLP-1 RA initiation. Vitamin D deficiency (12.4%) was the most frequently diagnosed, followed by nutritional anemia (1.55%) and iron-deficiency anemia (1.44%). Despite these risks, nutrition therapy and counseling were infrequently used, with only 23.3% of patients receiving an NT/C visit within six months of GLP-1 RA dispensing; the mean time to the first NT/C visit was 149 days following GLP-1 RA initiation.

The FDA launched an Expedited Investigational New Drug (IND) Pilot program intended to shorten the time required to initiate first-in-human clinical trials. By pairing drug sponsors with qualified research institutions and enabling rolling review of IND application components, the FDA hopes to streamline early drug development while maintaining regulatory oversight.
The Food and Drug Administration (FDA) released a transparency roadmap outlining initiatives to increase public visibility into its regulatory decision-making processes. The roadmap focuses on five key areas: disclosure of Complete Response Letters (CRLs), greater transparency throughout the investigational and marketing application lifecycle, expanded use and communication of new approach methodologies and novel biomarkers, development of more patient- and consumer-centered product labeling, and increased access to compliance and inspection records.
The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) met on October 1, 2026, to review and recommend influenza virus strains for inclusion in vaccines for the 2027 Southern Hemisphere influenza season. The committee recommended that egg-based trivalent influenza vaccines contain H1N1, H3N2 and influenza B strains. Although VRBPAC’s recommendations are advisory, the FDA frequently incorporates them into its final vaccine strain selections.
The FDA granted clearance to MODI, a mobile app from DreaMed Diabetes that uses continuous glucose monitoring (CGM) and insulin data to provide automated, personalized insulin dose recommendations for adults with T2DM using insulin injections. MODI is the first FDA-cleared technology to support automated insulin optimization for both basal-only and multiple-daily-injection regimens, potentially enabling more timely insulin adjustments between healthcare visits.

The 2026 Annual Report to the Nation on the Status of Cancer has been published in Cancer. Developed collaboratively by the CDC, the National Cancer Institute, the American Cancer Society and the North American Association of Central Cancer Registries, the report found that overall U.S. cancer mortality continued to decline from 2019 to 2023, driven largely by significant progress in lung cancer prevention, early detection and treatment. Despite this progress, lung cancer remains the leading cause of cancer-related death and the second most diagnosed cancer among both men and women. While lung cancer incidence and mortality rates have decreased, overall cancer incidence increased slightly from 2018 to 2022 because of increasing rates of breast cancer in women and prostate cancer in men, the most commonly diagnosed cancers in their respective populations.
The CDC released its annual report on the leading causes of death in the United States for 2024. The top 10 causes of death remained largely unchanged from 2023, with heart disease and cancer continuing to rank first and second, respectively. Also included in the top 10 causes are accidents; cerebrovascular disease; chronic lower respiratory disease; Alzheimer’s disease; diabetes mellitus; nephritis, nephrotic syndrome and nephrosis; chronic liver disease/cirrhosis; and intentional self-harm. Collectively, the top 10 causes account for more than 70% of all deaths.
The CDC issued a Health Alert Network advisory following recent increases in human rabies exposures and reported errors in rabies post-exposure prophylaxis (PEP) administration. Clinicians should conduct careful exposure risk assessments and adhere to established PEP recommendations to ensure appropriate use of rabies vaccines and human rabies immune globulin while avoiding unnecessary treatment and administration errors.
The CDC published a report in Morbidity and Mortality Weekly Report (MMWR) on 2025 adolescent (13–17 years of age) vaccination coverage based on data from the National Immunization Survey. Tetanus toxoid, reduced diphtheria toxoid, and acellular pertussis vaccine (Tdap) and quadrivalent meningococcal conjugate vaccine (MenACWY) coverage remained near 90%, while human papillomavirus (HPV) vaccination coverage remained stagnant for the fourth consecutive year and continued to trail other routinely recommended adolescent vaccines.

The Kidney Disease: Improving Global Outcomes 2026 Clinical Practice Guideline for the Management of Anemia in Chronic Kidney Disease (CKD) updated the organization's 2012 recommendations. The new guideline includes eight graded recommendations for clinical practice and addresses the diagnosis of anemia in CKD, providing evidence-based recommendations on the use of iron therapy, erythropoiesis-stimulating agents (ESAs), hypoxia-inducible factor–prolyl hydroxylase inhibitors (HIF-PHIs) and red blood cell (RBC) transfusions.
The American Academy of Pediatrics (AAP) released updated clinical practice guidelines for the diagnosis and management of urinary tract infections (UTIs) in children 8 days to 5 years of age. Previous recommendations on this topic were published in 2011 and retired in 2021. The updated guidance includes a comprehensive clinical practice guideline accompanied by three technical reports.
The AAP published a clinical report on the care of infants with congenital cytomegalovirus (CMV), expanding on existing Red Book guidance. The report is intended to assist healthcare professionals (HCPs) in identifying risk factors and clinical signs of congenital CMV, facilitate appropriate newborn evaluation and guide treatment.
The American Society of Hematology (ASH) published updated guidelines for the diagnosis of iron deficiency. The guidance includes five recommendations that address diagnostic approaches across key populations, including children, adults, menstruating individuals, pregnant individuals and adults with inflammation.

Drug Approvals

Specialty

Sept. 11, 2026 — apitegromab-mstn (Isembyld)

Biologics License Application (BLA) approval; Fast Track, Orphan Drug and Rare Pediatric Disease designations
Recombinant monoclonal antibody targeting proforms of myostatin
Indicated for the treatment of spinal muscular atrophy (SMA) in adults and pediatric patients ≥2 years of age who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment
Injection: 150 mg/3 mL (50 mg/mL) solution in a single-dose vial
Recommended dosage is weight-based (10 mg/kg) and administered by an HCP once every four weeks as an intravenous (IV) infusion over approximately 60–120 minutes
Approval was based on data from a 52-week, randomized, double-blind, placebo-controlled trial (SAPPHIRE; n=188) enrolling type 2 and type 3 SMA patients 2–21 years of age who were unable to move or walk independently (nonambulatory at baseline) and were receiving an approved SMN2-targeted treatment (nusinersen or risdiplam); the primary endpoint was the mean change from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE; 33 scored activities, with a total maximum score of 66 and higher scores indicating better motor function) in SMA patients; 156 patients 2–12 years of age were evaluated at 52 weeks in the primary efficacy analysis; apitegromab 10 mg/kg demonstrated a nominally statistically significant treatment effect on the HFMSE compared with placebo (least-squares mean difference [LSMD] versus placebo, 2.2; P=0.01); additionally, a higher proportion of apitegromab-treated patients had a ≥3-point improvement on the HFMSE at one year compared with placebo
Other FDA-approved therapies for SMA include the SMN2-directed antisense oligonucleotide (ASO) nusinersen (Spinraza; intrathecal administration) and the SMN2 splicing modifier risdiplam (Evrysdi; oral, once-daily administration, with or without food); both of these products are indicated for SMA in pediatric and adult patients; the adeno-associated virus (AAV) vector-based gene therapy onasemnogene abeparvovec is available in two formulations: a single-dose intrathecal injection (Itvisma) and a single-dose IV infusion (Zolgensma); Itvisma is FDA-approved for the treatment of SMA in adult and pediatric patients ≥2 years of age with confirmed mutation in the survival motor neuron 1 (SMN1) gene and Zolgensma is indicated for the treatment of pediatric patients <2 years of age with SMA with biallelic mutations in the SMN1 gene; Isembyld is the first FDA-approved treatment directly targeting muscle loss; in SAPPHIRE, adjunctive Isembyld demonstrated improvement in motor function compared with placebo in nonambulatory patients with SMA receiving background SMN2-targeted therapy
Isembyld is available from Scholar Rock

Sept. 17, 2026 — rebisufligene etisparvovec-hopf (Fayuvi)

BLA approval; Breakthrough Therapy, Fast Track and Orphan Drug designations; Priority Review Voucher (PRV) received for the approval
Adeno-associated virus serotype 9 (AAV9)-based gene therapy
Indicated for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA; also known as Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function
Suspension for IV infusion: 1×10¹³ vector genomes (vg)/mL; each vial contains 2 mL of extractable volume
Recommended dose is administered by an HCP as a single-dose, slow IV infusion over approximately one hour, with dosing based on vg/kg of body weight; administration should occur in a setting with personnel and equipment immediately available for management of severe infusion and hypersensitivity reactions; one day before the infusion, corticosteroids should be administered and continued for at least eight weeks
Approval was based on data from a multicenter, open-label, single-arm, single-dose, dose-escalation study (Transpher A; n=17); the difference in mean change in Bayley-III Cognitive Scale raw scores (range, 0–91, with higher scores indicating higher skill level) between 24 and 60 months of age was evaluated in patients treated with rebisufligene etisparvovec compared with an external, natural history control (n=27); gain or maintenance of developmental skills was demonstrated in the rebisufligene etisparvovec patients, as compared with loss of developmental skills, which was observed in the untreated, natural history group (mean treatment difference, 23.5; 95% confidence interval [CI], 17.2–29.9; P<0.0001)
Fayuvi is the first FDA-approved treatment for pediatric patients with this ultra-rare, fatal lysosomal storage disease that leads to neurodegeneration; children with MPS IIIA exhibit progressive developmental delay and lose cognitive, language and motor function over time, resulting in early mortality (median life expectancy, 15 years); the substrate heparan sulfate accumulates in cells due to a deficiency in the lysosomal enzyme sulfamidase; as an AAV9 gene therapy, Fayuvi provides a functional copy of the N-sulfoglucosamine sulfohydrolase (SGSH) gene to cells, leading to production of sulfamidase, which subsequently breaks down heparan sulfate in lysosomes
Fayuvi is available from Ultragenyx Pharmaceutical through qualified treatment centers

Sept. 23, 2026 — lirafugratinib (Lyrfigtu)

New Drug Application (NDA) approval; Assessment Aid, Breakthrough Therapy, Orphan Drug and Priority Review designations; Real-Time Oncology Review (RTOR)
Kinase inhibitor
Indicated for the treatment of adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma (CCA) harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement
Capsules: 20 mg and 30 mg
Recommended dosage is taken orally once daily until disease progression or unacceptable toxicity
Approval was based on data from a multicenter, open-label, single-arm trial (REFOCUS; n=116) evaluating patients with unresectable or metastatic CCA who were naïve to fibroblast growth factor receptor (FGFR) inhibitor therapy but had previously received chemotherapy or chemoimmunotherapy; the primary endpoints were objective response rate (ORR) and duration of response (DOR); the median time to response was 1.9 months (range, 1.1–10.9 months) and the ORR was 46% (95% CI, 36%–55%), with the majority of responses being partial responses; the median DOR was 11.8 months (95% CI, 7.5–13 months)
The National Comprehensive Cancer Network (NCCN) biliary tract cancer guidelines list futibatinib (Lytgobi; Accelerated Approval for select patients with intrahepatic CCA), pemigatinib (Pemazyre; Accelerated Approval for same indication as Lyrfigtu) and erdafitinib (Balversa; not FDA-approved for this CCA) as Category 2A treatment options for CCA with FGFR2 gene fusions or rearrangements for use in the subsequent-line setting if there is disease progression; Lyrfigtu is a selective, oral, small-molecule inhibitor of FGFR2 that is specifically indicated for use in the second-line CCA setting
Lyrfigtu will be available from Elevar Therapeutics; launch is expected in the fourth quarter of 2026

Sept. 25, 2026 — tavapadon (Juvmo)

NDA approval; first selective partial agonist at the dopamine D1 and D5 receptor subtypes (D₁/D₅) approved for adults with Parkinson's disease
Non-ergot dopamine agonist
Indicated for the treatment of Parkinson’s disease in adults
Oral tablets: 0.25 mg, 1 mg, 5 mg, 10 mg and 15 mg
Recommended dosage is taken orally once daily, with or without food; a titration regimen is required to reach the maintenance dose at Day 41 (or later); titration is based on tolerability and clinical response; further titration is required to reach the maximum recommended daily dosage
Approval was based on data from three clinical studies conducted in adults with Parkinson’s disease; the TEMPO-1 (n=529) and TEMPO-2 (n=304) trials were 27-week, randomized, multicenter, double-blind, placebo-controlled studies that evaluated patients who were not taking oral levodopa; the primary endpoint was the change from baseline in the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II score (13 questions related to activities of daily living; scoring, 0 indicates normal and 4 indicates maximal severity, for a maximum [worst] score of 52) at Week 26; for TEMPO-1, the LSMD for MDS-UPDRS Part II score with tavapadon versus placebo was -2.5 (95% CI, -3.3 to -1.7) for the 5 mg dose and -2.6 (95% CI, -3.4 to -1.7) for the 15 mg dose (P<0.0001 for both comparisons); for TEMPO-2, patients in the tavapadon arm were titrated to receive 5–15 mg (based on tolerability); at Week 26, the LSMD versus placebo in the MDS-UPDRS Part II score was -1.5 (95% CI, -2.4 to -0.6; P=0.0007); TEMPO-3 (n=507) was a 27-week, double-blind, randomized, placebo-controlled, flexible-dose trial that assessed patients receiving concurrent levodopa; it evaluated the primary endpoint of change from baseline in the total daily “on” time without troublesome dyskinesia at Week 26 based on the self-completed home diary for motor function status (Hauser diary); the LSMD versus placebo was 1.1 hours (P<0.0001)
Other FDA-approved therapies for Parkinson’s disease include levodopa and dopamine agonists (e.g., pramipexole [Mirapex], ropinirole [Requip], rotigotine [Neupro]); levodopa is a dopamine precursor and is considered a first-line treatment option for Parkinson’s disease when combined with carbidopa; pramipexole, ropinirole and rotigotine act at the D₂/D₃ receptors
Juvmo is available from AbbVie

Sept. 25, 2026 — zilurgisertib (Atebrioz)

NDA approval; Fast Track, Orphan Drug and Priority Review designations; Rare Pediatric Disease PRV received for this approval
Activin receptor-like kinase-2 (ALK2) inhibitor
Indicated to reduce the volume of total new heterotopic ossification (HO) in adults and pediatric patients ≥12 years of age with fibrodysplasia ossificans progressiva (FOP)
Oral tablets: 25 mg
Recommended starting dosage is taken orally once daily, with or without food; tablets can be administered whole or crushed; if crushed, four tablets (100 mg) should be mixed with four tablespoons (60 mL) of water, orange juice, applesauce or plain yogurt
Approval was based on data from a randomized, double-blind, placebo‑controlled trial (PROGRESS; n=63) that evaluated the change from baseline in total new HO lesion volume, as assessed by whole body computed tomography (CT) scans in 28 adults and 35 pediatric patients ≥12 years of age; patients in the zilurgisertib study arm had an average 3.2 cm³ reduction in total new HO lesion volume compared with placebo patients, who had a 24.6 cm³ increase at Week 24 (Hodges-Lehmann treatment difference, -15.8 cm³; 95% CI, -32.1 to -6)
FOP is an ultra-rare, genetic condition resulting in connective tissues (e.g., muscles, tendons, ligaments) gradually turning into bone, leading to reduced mobility, physical deformities, severe disability and early mortality; FOP is caused by a mutation in the activin A receptor type 1 (ACVR1) gene, a receptor involved in bone growth; Atebrioz is the third FDA-approved therapy for FOP; other FDA-approved therapies include the retinoid palovarotene (Sohonos) — an oral capsule indicated for reducing new HO lesion volume in adults and female children ≥8 years of age and male children ≥10 years of age with FOP — and garetosmab-grts (Pasatru), an activin signaling inhibitor that binds activin A, stopping its ability to activate FOP-mutant ACVR1; Pasatru is indicated to reduce formation of new HO lesions and clinician-assessed flare-ups in adults with FOP; Atebrioz is a selective inhibitor of both wild-type ALK2 and pathogenic ALK2, resulting in inhibition of ALK2-mediated signaling and reduction in endochondral bone formation
Atebrioz is available from Mirum Pharmaceuticals/Incyte

Sept. 28, 2026 — tiratricol (Emcitate)

NDA approval; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review and Rare Pediatric Disease designations; FDA also awarded a Rare Pediatric Disease PRV in connection with the approval
Thyroid hormone receptor agonist
Indicated for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley syndrome); not recommended for the treatment of primary hypothyroidism
Tablets for oral suspension: 350 mcg
Recommended dosage is individualized based on body weight, total triiodothyronine (T3) concentrations, and clinical signs and symptoms of thyrotoxicosis; tablets must be dispersed in room-temperature drinking water prior to oral or enteral tube administration
Approval was based on data from two clinical studies in patients with MCT8 deficiency, including a multicenter, single-arm, open-label, Phase 2, 12-month trial (n=46) and a multicenter, randomized, double-blind, placebo-controlled withdrawal trial (ReTRIACt; n=15); in the open-label study, tiratricol significantly reduced elevated serum total T3 concentrations compared with baseline (primary endpoint); in ReTRIACt, continued tiratricol treatment maintained control of serum T3 concentrations from baseline, whereas treatment withdrawal led to a significant increase in serum T3 concentrations (treatment difference, 64.8 ng/dL; 95% CI, 44.7–84.8 for placebo versus tiratricol arm)
Boxed warning: not for the treatment of obesity or for weight loss
MCT8 deficiency is a rare, X-linked disorder caused by pathogenic variants in the SLC16A2 gene, resulting in impaired transport of thyroid hormone into the brain; affected individuals experience severe neurodevelopmental impairment, hypotonia, inability to walk independently, intellectual disability, feeding difficulties, tachycardia, muscle wasting and other complications related to chronic peripheral thyrotoxicosis; Emcitate can enter cells independently of MCT8, enabling thyroid hormone receptor activation, which modulates hypothalamic-pituitary-thyroid axis signaling and thereby reduces circulating T3 in patients with MCT8 deficiency; Emcitate is the first FDA-approved treatment for MCT8 deficiency in the United States
Emcitate is available from Egetis Therapeutics

Sept. 11, 2026 — cabozantinib (Omcazio)

505(b)(2) NDA approval; Omcazio has demonstrated bioequivalence to cabozantinib tablets (Cabometyx) at a lower dose; unlike Cabometyx, which requires administration on an empty stomach (≥1 hour before or ≥2 hours after eating), Omcazio can be taken with or without food
Kinase inhibitor
Indicated for the treatment of (1) patients with advanced renal cell carcinoma (RCC); (2) patients with advanced RCC, as a first-line treatment in combination with nivolumab; (3) patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib; and (4) adult and pediatric patients ≥12 years of age with previously treated, unresectable, locally advanced or metastatic, well-differentiated extra-pancreatic neuroendocrine tumors (epNET)
Oral capsules: 11.5 mg, 23 mg and 34.5 mg; cannot be substituted on a mg-to-mg basis with other cabozantinib products
Recommended dosage is dependent on indication for use and is taken orally once daily, with or without food
Boxed warning: risk of serious adverse reactions or reduced effectiveness due to medication errors
Omcazio will be available from Handa Oncology; the launch timeframe is to be determined (TBD)

Oct. 1, 2026 — no trade name (trabectedin)

505(b)(2) NDA approval; other trabectedin products, including Yondelis (1 mg powder for injection) and Evdi (1 mg/20 mL solution for injection), have the same FDA-approved indication as this product
Alkylating drug
Indicated for the treatment of adults with unresectable or metastatic liposarcoma or leiomyosarcoma who received a prior anthracycline-containing regimen
For injection: 1 mg as lyophilized powder in a single-dose vial
Recommended dosage is based on body surface area and is administered by an HCP as a 24-hour IV infusion, every three weeks, through a central venous line; continue treatment until disease progression or unacceptable toxicity; premedication with IV dexamethasone should be given 30 minutes prior to each infusion
Trabectedin will be available from Ever Pharma; the launch timeframe is TBD

Sept. 15, 2026 – insulin aspart-fsan (Garzulys)

Meitheal Pharmaceuticals; BLA approval; insulin aspart-fsan (Garzulys) in the 10 mL (100 units/mL) multidose vial for IV and subcutaneous (SC) use and the 3 mL (100 units/mL) single-patient-use prefilled pen for SC use have received FDA approval as an interchangeable biosimilar to the same presentation of insulin aspart (Novolog)
Previously, Garzulys was only approved as a biosimilar to Novolog; Garzulys is the second interchangeable biosimilar to Novolog

Sept. 25, 2026 — denosumab-adet (Degevma)

BLA approval; 120 mg/1.7 mL (70 mg/mL) injection in a single-dose vial for SC use has received FDA approval as an interchangeable biosimilar to the same presentation of denosumab (Xgeva)
Degevma is approved for all indications as the reference drug, Xgeva
Degevma will be available from Teva Pharmaceuticals; launch is expected in the coming months

Sept. 11, 2026 — leniolisib (Joenja)

Pharming Technologies; kinase inhibitor; Orphan Drug, Priority Review and Rare Pediatric Disease designations; first FDA-approved treatment for pediatric patients 4–11 years of age with activated phosphoinositide 3-kinase delta syndrome (APDS); the FDA also approved new 40 mg and 50 mg oral tablet strengths
Expanded indication: APDS in pediatric patients ≥4 years of age weighing ≥27 kg; previously indicated for APDS in adults and pediatrics ≥12 years of age
Dosage for pediatric patients 4 to <12 years of age is weight-based (27 to <38 kg, 38 to <45 kg, ≥45 kg) and is administered orally twice daily, approximately 12 hours apart, with or without food

Sept. 18, 2026 — abemaciclib (Verzenio)

Eli Lilly and Company; kinase inhibitor; Assessment Aid designation; FDA also approved the Guardant360 CDx assay as a companion diagnostic device to identify eligible patients for treatment
New indication: in combination with imlunestrant (Inluriyo) for treatment of adults with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, estrogen receptor-1 (ESR1)-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy
Dosage is administered orally twice daily, with or without food, until disease progression or unacceptable toxicity; premenopausal/perimenopausal women and men should also be treated with a gonadotropin-releasing hormone (GnRH) agonist in accordance with current clinical practice standards
Other indications are detailed in the product label

Sept. 18, 2026 — imlunestrant (Inluriyo)

Eli Lilly and Company; ER antagonist; Assessment Aid designation; the FDA also approved the Guardant360 CDx assay as a companion diagnostic device to identify eligible patients for treatment
New indication: in combination with abemaciclib (Verzenio) for treatment of adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy
Dosage is administered orally once daily, on an empty stomach, at least two hours before or one hour after food, until disease progression or unacceptable toxicity
Other indication: monotherapy for treatment of adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer, as detected by an FDA-authorized test, with disease progression following at least one line of endocrine therapy

Sept. 18, 2026 — levacetylleucine (Aqneursa)

IntraBio; modified amino acid; Orphan Drug and Priority Review designations; first drug to be approved for ataxia in patients with ataxia-telangiectasia (A-T)
New indication: ataxia in adults and pediatric patients with A-T weighing ≥15 kg
Administered by mixing contents of Aqneursa packet(s) with water to form a suspension for oral or feeding-tube administration; dosage is determined using patient’s actual body weight (15 to <25 kg, 25 to <35 kg or ≥35 kg) and is administered two or three times daily, with or without food; when administered orally, Aqneursa packet(s) can also be mixed with orange juice, almond milk or yogurt, in addition to water
Other indication: neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients weighing ≥15 kg

Sept. 24, 2026 — belzutifan (Welireg)

Merck; hypoxia-inducible factor inhibitor; Assessment Aid designation
New indication: in combination with lenvatinib (Lenvima) for treatment of adults with advanced renal cell carcinoma with a clear cell component (ccRCC) following a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor
Dosage is administered orally once daily, with or without food, until disease progression or unacceptable toxicity
Other indications are detailed in the product label

Sept. 24, 2026 — lenvatinib (Lenvima)

Eisai; kinase inhibitor; Assessment Aid designation
New indication: in combination with belzutifan (Welireg) for treatment of adults with advanced ccRCC following a PD-1 or PD-L1 inhibitor
Dosage is administered orally once daily, with or without food, until disease progression or unacceptable toxicity
Other indications are detailed in the product label

Sept. 25, 2026 — baricitinib (Olumiant)

Eli Lilly and Company; Janus kinase (JAK) inhibitor
Expanded indication: treatment of severe alopecia areata in pediatric patients ≥12 years of age; previously only indicated for severe alopecia areata in adults
Administered orally once daily, with or without food; higher dosage can be considered for patients with nearly complete or complete scalp hair loss, with or without substantial eyelash or eyebrow hair loss, and for those with an inadequate response to the lower dosage; for patients taking the higher dosage, dosage should be reduced once an adequate response is achieved
Other indications are detailed in the product label

Sept. 25, 2026 — obinutuzumab (Gazyva)

Genentech; cluster of differentiation (CD) 20-directed cytolytic antibody; Breakthrough Therapy, Orphan Drug and Priority Review designations
New indication: to reduce the risk of relapse in adults and pediatric patients ≥2 years of age with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome who are in complete remission
Dosage is administered as a weight-based (<45 or ≥45 kg) IV infusion by an HCP on Days 1 and 15 and Weeks 24 and 26; premedication with an IV glucocorticoid, acetaminophen and an antihistamine is recommended
Other indications are detailed in the product label

Sept. 28, 2026 — rituximab-arrx (Riabni)

Amgen; CD20-directed cytolytic antibody; biosimilar to rituximab (Rituxan)
Expanded indication: in combination with glucocorticoids for treatment of granulomatosis with polyangiitis (GPA; Wegener’s granulomatosis) and microscopic polyangiitis (MPA) in pediatric patients ≥2 years of age; previously only indicated for GPA and MPA in adults
Administered as an IV infusion by an HCP; induction treatment is administered once weekly for four weeks, with IV methylprednisolone given prior to the infusion and oral steroids continued according to clinical practice; follow-up treatment in patients who achieved disease control with induction treatment, in combination with glucocorticoids, is administered as two infusions separated by two weeks, followed by one infusion every six months thereafter based on clinical evaluation
Other indications are detailed in the product label

Sept. 28, 2026 — rituximab-pvvr (Ruxience)

Pfizer; CD20-directed cytolytic antibody; biosimilar to rituximab (Rituxan)
Expanded indication: in combination with glucocorticoids for treatment of GPA (Wegener’s granulomatosis) and MPA in pediatric patients ≥2 years of age; previously only indicated for GPA and MPA in adults
Administered as an IV infusion by an HCP; induction treatment is administered once weekly for four weeks, with IV methylprednisolone given prior to the infusion and oral steroids continued according to clinical practice; follow-up treatment in patients who achieved disease control with induction treatment, in combination with glucocorticoids, is administered as two infusions separated by two weeks, followed by one infusion every six months thereafter based on clinical evaluation
Other indications are detailed in the product label

Sept. 28, 2026 — mavacamten (Camzyos)

Bristol Myers Squibb; cardiac myosin inhibitor; Orphan Drug designation; first FDA-approved treatment for children with symptomatic obstructive hypertrophic cardiomyopathy (oHCM); FDA also approved new 1 mg capsule strength
Expanded indication: treatment of adults and pediatric patients weighing ≥30 kg with symptomatic oHCM to improve functional capacity and symptoms; previously only indicated for adults with New York Heart Association (NYHA) class II-III oHCM
Dosage is administered orally once daily, with or without food; initial dose is based on weight (30 to <45 kg or ≥45 kg); subsequent dose titrations should be individualized based on clinical status and echocardiographic assessment of patient response

Sept. 29, 2026 — tocilizumab-anoh (Avtozma)

Celltrion; interleukin-6 (IL-6) receptor antagonist; interchangeable biosimilar to tocilizumab (Actemra)
Expanded indication: for coronavirus disease 2019 (COVID-19) in hospitalized pediatric patients ≥2 years of age who are receiving systemic corticosteroids and require supplemental oxygen, noninvasive or invasive mechanical ventilation or extracorporeal membrane oxygenation (ECMO); previously indicated for this use in adults
Administered by IV infusion over 60 minutes, with dosing based on body weight (<30 or ≥30 kg); after the first dose, if no improvement or worsening, one additional injection can be given a minimum of 8 hours after the initial infusion
Other indications are detailed in the product label

Oct. 2, 2026 — pirtobrutinib (Jaypirca)

Eli Lilly and Company; kinase inhibitor; Assessment Aid designation
New indication: treatment of adults with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) with no known 17p deletion
Administered orally once daily, with or without food, until disease progression or unacceptable toxicity
Other indications are detailed in the product label

Oct. 6, 2026 — remibrutinib (Rhapsido)

Novartis Pharmaceuticals; kinase inhibitor; first treatment approved for symptomatic dermographism
Expanded indication: treatment of symptomatic dermographism and chronic spontaneous urticaria (CSU) in adults who remain symptomatic despite use of H1 antihistamines; previously approved for CSU only
Dosage is administered orally twice daily, with or without food

Oct. 7, 2026 — tucatinib (Tukysa)

Seagen, a subsidiary of Pfizer; kinase inhibitor; Assessment Aid designation
New indication: in combination with trastuzumab (Herceptin) and pertuzumab (Perjeta) for maintenance treatment of adults with unresectable, locally advanced or metastatic HER2-positive breast cancer following induction treatment
Administered orally twice daily, with or without food, until disease progression or unacceptable toxicity
Other indications are detailed in the product label

Oct. 8, 2026 — atezolizumab (Tecentriq)

Genentech; PD-L1-blocking antibody; Assessment Aid and Priority Review designations; Project Orbis review
New indication: in combination with a fluoropyrimidine and oxaliplatin for adjuvant treatment of adults and pediatric patients ≥2 years of age with stage III mismatch repair deficient (dMMR) colon cancer
Administered IV by an HCP every two weeks (adults and pediatrics), every three weeks (adults and pediatrics) or every four weeks (adults only), in combination with chemotherapy, for six months; then administered IV by an HCP as monotherapy for six months or until disease recurrence or unacceptable toxicity
Other indications are detailed in the product label

Oct. 8, 2026 — atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza)

Genentech; atezolizumab is a PD-L1-blocking antibody and hyaluronidase-tqjs is an endoglycosidase; Assessment Aid and Priority Review designations; Project Orbis review
New indication: in combination with a fluoropyrimidine and oxaliplatin for adjuvant treatment of adults and pediatric patients ≥12 years of age weighing ≥40 kg with stage III dMMR colon cancer
Dosage is administered as an SC injection by an HCP every three weeks, in combination with chemotherapy, for six months; then administered SC by an HCP as monotherapy for six months or until disease recurrence or unacceptable toxicity
Other indications are detailed in the product label

Traditional

Sept. 24, 2026 — insulin efsitora alfa-gobe (Onswik)

BLA approval; second FDA-approved once weekly long-acting insulin analog
Long-acting basal insulin analog
Indicated as an adjunct to diet and exercise to improve glycemic control in adults with T2DM
Not recommended for use in patients with T1DM due to increased risk of severe hypoglycemia
Solution for injection: 3 mL single-patient-use KwikPen containing either 1,500 units (500 units/mL [U-500]) or 3,000 units (1,000 units/mL [U-1,000])
Recommended dosage is administered once weekly, on the same day each week, via SC injection into the abdomen, upper arm, thigh or buttocks; dosage should be individualized and adjusted based on metabolic needs, glucose monitoring results and glycemic goals
Approval was based on data from four clinical trials; QWINT-1 (n=795) was a 52-week, Phase 3, open-label, randomized, treat-to-target trial that evaluated adults with T2DM who were starting basal insulin therapy and found insulin efsitora was noninferior to once-daily insulin glargine based on a reduction in mean glycated hemoglobin level (HbA1c) at 52 weeks (-1.2% versus -1.1%; difference, 0%; 95% CI, -0.2–0.1); QWINT-2 (n=928) was a 52-week, Phase 3, parallel-design, open-label, randomized, treat-to-target trial that evaluated insulin-naïve adults with T2DM and found insulin efsitora was noninferior to once daily insulin degludec based on a mean reduction in HbA1c at 52 weeks (-1.2% versus -1.2%; difference, -0.1%; 95% CI, -0.2–0); QWINT-3 (n=986) was a 78-week, Phase 3, open-label, randomized, treat-to-target trial that evaluated adults with T2DM currently being treated with basal insulin therapy and found insulin efsitora was noninferior to insulin degludec at Week 26 based on a mean HbA1c reduction (-0.8% versus -0.7%; difference, -0.1%; 95% CI, -0.2–0); QWINT-4 (n=730) was a 26-week, open-label, randomized, treat-to-target trial that evaluated adults with T2DM on once- or twice-daily basal insulin therapy and mealtime insulin and found insulin efsitora to be noninferior to insulin glargine based on a mean HbA1c reduction at Week 26 (-1% versus -1%, difference, 0%; 95% CI, -0.1–0.1)
Insulin icodec-abae (Awiqli) is also a long-acting insulin analog that carries the same indication as Onswik and is also given SC once weekly; it is supplied in a single-patient-use FlexTouch prefilled pen containing 700 units (1 mL pen), 1,050 units (1.5 mL pen) and 2,100 units (3 mL pen) of 700 unit/mL (U-700) insulin
Onswik will be available from Eli Lilly in the coming months

None

Sept. 16, 2026 — finerenone (Kerendia)

Bayer HealthCare Pharmaceuticals; nonsteroidal mineralocorticoid receptor antagonist (nsMRA)
New indication: to reduce the urinary albumin-to-creatinine ratio (UACR), which is expected to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline and end-stage kidney disease in adults with chronic kidney disease (CKD) associated with T1DM
Dosage is taken orally once daily, with or without food; starting dosage is based on patient’s eGFR and serum potassium thresholds; dosage should be increased after four weeks based on eGFR and serum potassium
Other indications are detailed in the product label

First generic drug launches

Oct. 5, 2026 — riociguat (Adempas)

MSN Laboratories/Zydus Lifesciences launched oral tablets (0.5 mg, 1 mg, 1.5 mg, 2 mg and 2.5 mg) generic to Bayer HealthCare Pharmaceuticals’ Adempas
Soluble guanylate cyclase (sGC) stimulator indicated for the treatment of adults with: (1) persistent/recurrent chronic thromboembolic pulmonary hypertension (CTEPH; WHO Group 4) after surgical treatment or inoperable CTEPH to improve exercise capacity and WHO functional class and (2) pulmonary arterial hypertension (PAH; WHO Group 1) to improve exercise capacity, improve WHO functional class and delay clinical worsening
Recommended dosage is taken orally three times a day; tablets can be crushed and mixed with water or soft foods for patients who have difficulty swallowing
U.S. annual sales for Adempas in 2025 were $1,074 million

AAP American Academy of Pediatrics
AAV adeno-associated virus

AAV9 adeno-associated virus serotype 9

ACVR1 activin A receptor type 1

ALK2 activin receptor-like kinase 2

APDS activated phosphoinositide 3-kinase delta syndrome
ASH American Society of Hematology

ASO antisense oligonucleotide

A-T ataxia-telangiectasia
BLA Biologics License Application

BMI body mass index

CCA cholangiocarcinoma

ccRCC clear cell renal cell carcinoma

CD20 cluster of differentiation 20

CDC Centers for Disease Control and Prevention

CGM continuous glucose monitoring

CI confidence interval

CKD chronic kidney disease
CLL chronic lymphocytic leukemia
CMV cytomegalovirus
COVID-19 coronavirus disease 2019

CRL Complete Response Letter
CSU chronic spontaneous urticaria
CT computed tomography

CTEPH chronic thromboembolic pulmonary hypertension

D1/D5 dopamine D1 and D5 receptor subtypes

dMMR mismatch repair deficient
DOR duration of response

ECMO extracorporeal membrane oxygenation
eGFR estimated glomerular filtration rate
epNET extra-pancreatic neuroendocrine tumor

ER estrogen receptor

ESA erythropoiesis-stimulating agent

ESR1 estrogen receptor 1
FDA Food and Drug Administration

FGFR fibroblast growth factor receptor
FGFR2 fibroblast growth factor receptor 2

FOP fibrodysplasia ossificans progressiva
GLP-1 RA glucagon-like peptide-1 receptor agonist

GnRH gonadotropin releasing hormone
GPA granulomatosis with polyangiitis
H1N1 influenza A strain H1N1
H3N2 influenza A strain H3N2
HbA1c glycated hemoglobin level
HCC hepatocellular carcinoma
HCP healthcare professional

HER2 human epidermal growth factor receptor 2

HFMSE Hammersmith Functional Motor Scale Expanded
HIF-PHI hypoxia-inducible factor–prolyl hydroxylase inhibitor
HO heterotopic ossification

HPV human papillomavirus

iCCA intrahepatic cholangiocarcinoma
ICD-10 International Classification of Diseases, 10th edition

IL-6 interleukin-6
IND Investigational New Drug
IV intravenous

JAK Janus kinase

KDIGO Kidney Disease: Improving Global Outcomes

LSMD least-squares mean difference
MCT8 monocarboxylate transporter 8
MDS-UPDRS Movement Disorder Society-sponsored revision of the Unified Parkinson’s Disease Rating Scale
MenACWY quadrivalent meningococcal conjugate vaccine

MMWR Morbidity and Mortality Weekly Report

MPA microscopic polyangiitis

MPS IIIA mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A)

NCCN National Comprehensive Cancer Network
NDA New Drug Application

nsMRA nonsteroidal mineralocorticoid receptor antagonist
NT/C nutrition therapy/counseling

NYHA New York Heart Association
oHCM obstructive hypertrophic cardiomyopathy
ORR objective response rate

PAH pulmonary arterial hypertension

PD-1 programmed cell death receptor-1

PD-L1 programmed death-ligand 1
PEP post-exposure prophylaxis

PRV Priority Review Voucher

RBC red blood cell
RCC renal cell carcinoma

RTOR Real-Time Oncology Review

SC subcutaneous

sGC soluble guanylate cyclase
SGSH N-sulfoglucosamine sulfohydrolase gene (encodes the sulfamidase enzyme)
SLL small lymphocytic lymphoma
SMA spinal muscular atrophy
SMN1 survival motor neuron 1
SMN2 survival motor neuron 2

T1DM type 1 diabetes mellitus

T2DM type 2 diabetes mellitus

T3 triiodothyronine

TBD to be determined

Tdap tetanus toxoid, reduced diphtheria toxoid and acellular pertussis vaccine

U.S. United States
UACR urinary albumin-to-creatinine ratio

UTI urinary tract infection
vg vector genomes
VRBPAC Vaccines and Related Biological Products Advisory Committee

WHO World Health Organization

Editor-in-Chief: Maryam Tabatabai, PharmD

Executive Editor: Anna Schreck Bird, PharmD

Deputy Editors: Nicole Kjesbo, PharmD, BCPS; Olivia Pane, PharmD, CDCES

The content in this publication is not a substitute for professional medical advice. For questions regarding any medical condition, or if you need medical advice, please contact your healthcare provider.
All brand names are property of their respective owners.

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