publications

Aug. 13, 2026 — iberdomide (Zenbexus)

New Drug Application (NDA) approval; Accelerated Approval, Assessment Aid, Breakthrough Therapy, Orphan Drug and Priority Review designations; Project Orbis review
Cereblon-modulating protein degrader
Indicated in combination with daratumumab/hyaluronidase-fihj (Darzalex Faspro) and dexamethasone (Dd) for the treatment of adults with multiple myeloma (MM) who have received at least one prior line of therapy including a proteasome inhibitor and an immunomodulatory agent
Accelerated Approval was based on minimal residual disease (MRD)-negative complete response (CR) at any time; therefore, continued approval may require demonstration of clinical benefit in confirmatory trial(s)
Oral capsules: 0.75 and 1 mg
Recommended dosage is taken orally once daily, with or without food, on Days 1–21 of repeated 28-day cycles until disease progression or unacceptable toxicity
Approval was based on data from the two-stage, randomized, multicenter, open-label Phase 3 trial (EXCALIBER-RRMM; n=939) that evaluated adults with relapsed or refractory MM (RRMM) who had previously received 1–2 prior lines of therapy; MRD-negative CR at any time was evaluated as the primary endpoint of the first 420 patients randomized to iberdomide 1 mg in combination with Dd or the comparator arm of daratumumab/hyaluronidase-fihj, bortezomib and dexamethasone (DVd); at any time, the MRD-negative CR was 41% in the study arm that received iberdomide + Dd compared with 21% in the DVd study arm (P<0.0001)
Boxed warning for embryo-fetal toxicity and serious venous and arterial thromboembolism
The National Comprehensive Cancer Network (NCCN) MM guidelines list iberdomide (Zenbexus)/dexamethasone plus daratumumab as a Category 2A (other recommended regimen) for relapsed/refractory disease after 1–3 prior therapies; the guidelines also recommend several other regimens as options in this setting
Zenbexus is available from Bristol Myers Squibb through a restricted distribution program called Zenbexus Risk Evaluation and Mitigation Strategy (REMS)

Aug. 19, 2026 — garetosmab-grts (Pasatru)

Biologics License Application (BLA) approval; Breakthrough Therapy, Fast Track, Orphan Drug and Priority Review designations
Activin signaling inhibitor
Indicated to reduce formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP)
Solution for injection: 300 mg/5 mL (60 mg/mL) in a single-dose vial
Recommended starting dosage is a weight-based intravenous (IV) infusion administered by a healthcare professional (HCP) over 60 minutes once every four weeks; dosage can be decreased due to tolerability
Approval was based on data from the randomized, double-blind, placebo-controlled, multicenter Phase 3 study (OPTIMA; n=63) that evaluated adults with FOP; the primary endpoint was the number of new HO lesions that formed by Week 56, as detected using low-dose, full-body computed tomography (CT) imaging; at Week 56, garetosmab significantly reduced the number of new HO lesions compared with placebo at both the recommended starting dose (10 mg/kg) and the tolerability-reduced dose (3 mg/kg); two new lesions among 23 patients receiving the starting dose and one new lesion among 19 patients receiving the reduced dose, compared with 19 new lesions among 21 patients receiving placebo) resulting in a 90% reduction for the recommended starting dose and a 94% reduction for the reduced dose compared with placebo (P<0.05 for both doses)
FOP is an ultra-rare, genetic condition resulting in connective tissues (e.g., muscle, 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; Pasatru binds activin A and stops its ability to activate FOP-mutant ACVR1; it is the second FDA-approved therapy for this condition; the retinoid palovarotene (Sohonos) is an oral capsule indicated for reducing the volume of new HO in adults and children ≥8 years of age for females and ≥10 years of age for males with FOP
Pasatru is available from Regeneron

Aug. 19, 2026 — pariglasgene brecaparvovec-opnr (Genglycos)

BLA approval; Accelerated Approval, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations; Rare Pediatric Disease Priority Review Voucher
Adeno-associated virus (AAV) vector-based gene therapy
Indicated to reduce daily cornstarch intake as an adjunct to nutritional management in adult and pediatric patients ≥8 years of age with glycogen storage disease type Ia (GSDIa)
Accelerated Approval was based on a decrease in daily cornstarch intake; therefore, continued approval for this use may require demonstration of benefit in confirmatory clinical trial(s)
Suspension for IV infusion: 3 × 10¹³ genome copies (gc)/mL; supplied as a kit with 3–15 single-dose vials
Recommended dosage is administered as a single-dose IV infusion dosed in gc per kg of body weight; baseline testing for pre-existing antibodies to AAV8 should be conducted and baseline liver-function tests should be assessed before infusion; acetaminophen and nonsedating antihistamines should be administered as premedication 30–60 minutes before the infusion; two weeks following administration, prophylactic corticosteroids should be administered for a minimum of eight weeks
Approval was based on data from the randomized, double-blind, placebo-controlled Phase 3 GlucoGene study (n=46) in patients ≥8 years with GSDIa; the primary endpoint was the percentage change from baseline to Week 48 in cornstarch intake; patients who received pariglasgene brecaparvovec-opnr demonstrated a statistically significant reduction in the least-squares average daily cornstarch intake from baseline (41% versus 10% for placebo; P<0.0001)
GSDIa is an ultra-rare, inherited disorder caused by a genetic mutation in the glucose-6-phosphatase gene (G6PC) that leads to the absence of the glucose-6-phosphatase (G6Pase) enzyme responsible for release of glucose from glycogen stores in the liver; as a result of the enzyme deficiency, the body’s ability to maintain stable blood glucose between meals, during fasting and in periods of metabolic stress is severely compromised, leading to potentially life-threatening hypoglycemic episodes and serious, long-term metabolic complications; traditional management strategies for GSDIa include frequent meals, avoidance of simple sugar-containing foods, medical monitoring, and continuous consumption of uncooked/specially formulated cornstarch to provide a slow-digesting complex carbohydrate for prevention of hypoglycemia; this approach can lead to hyperglycemia in an effort to avoid hypoglycemia; Genglycos is the first FDA-approved therapy for patients with GSDIa and is administered as a one-time gene therapy that provides a functional human copy of the G6PC gene to the liver to aid in restoration of the deficient G6Pase enzyme, which may help restore the body’s ability to utilize glycogen stored in the liver to maintain blood glucose levels
Genglycos is available from Ultragenyx Pharmaceutical through a national network of Qualified Treatment Centers

Aug. 26, 2026 — daraxonrasib (Rasonque)

NDA approval; Breakthrough Therapy, Orphan Drug and Priority Review designations; Commissioner’s National Priority Voucher (CNPV) pilot program
RAS GTPase family inhibitor
Indicated for the treatment of adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or who are not candidates for multiagent systemic therapy
Oral tablets: 100 mg and 150 mg
Recommended dosage is administered orally once daily, with or without food; prophylactic and concurrent medications should be administered as recommended in the Rasonque labeling to decrease the risk of dermatologic reactions
Approval was based on a randomized, open-label, multicenter Phase 3 clinical study (RASolute 302; n=500) that evaluated patients with metastatic pancreatic adenocarcinoma with disease progression after receiving one prior line of systemic therapy, which included either a fluoropyrimidine-based or gemcitabine-based regimen; patients received either daraxonrasib orally once daily or physician’s choice of standard of care (SOC) chemotherapy regimens that were continued until disease progression or unacceptable toxicity; the primary endpoint was overall survival (OS) and progression-free survival (PFS) in patients with a RAS G12 mutation (91.8% of enrolled patients had these mutations); the median OS was 13.2 months with daraxonrasib compared with 6.6 months with SOC chemotherapy in the RAS G12 population (hazard ratio, 0.4; P<0.001); the median PFS in the RAS G12 population was 7.3 months with daraxonrasib compared with 3.5 months with SOC chemotherapy (hazard ratio, 0.45; P<0.001)
Most newly diagnosed pancreatic cancer cases in the United States are pancreatic adenocarcinomas, and although this represents only 3.2% of all cancer diagnoses, pancreatic adenocarcinoma continues to be associated with a high mortality rate due to late diagnosis, aggressive presentation and historically limited response to conventional therapies; traditional treatment options in the recurrent disease setting are based on whether prior therapy was gemcitabine- or fluoropyrimidine-based; in patients with pancreatic ductal carcinoma, an oncogenic RAS mutation exists in >90% of cases; Rasonque is a first-in-class oral RAS(ON) multiselective inhibitor for both mutant and wild-type RAS and is included in NCCN guidelines as a Category 1 recommendation in the subsequent therapy setting for locally advanced/metastatic disease and a Category 2A or 2B recommendation (depending on performance status) in the first-line setting for metastatic disease
Rasonque is available from Revolution Medicines

Aug. 27, 2026 — brepocitinib (Lisraya)

NDA approval; Orphan Drug and Priority Review designations
Dual Janus kinase (JAK) and tyrosine kinase 2 (TYK2) inhibitor
Indicated for the treatment of dermatomyositis in adult patients
Not recommended for use in combination with other JAK inhibitors, other TYK2 inhibitors or biologic disease-modifying antirheumatic drugs (DMARDs)
Oral tablets: 30 mg
Recommended dosage is taken once daily, administered orally, with or without food
Approval was based on data from a randomized, double-blind, multicenter, placebo-controlled Phase 3 study (VALOR; n=241) that evaluated the primary endpoint of Total Improvement Score (TIS; score range, 0–100; higher score demonstrates greater improvement) at Week 52 in patients receiving standard therapies for dermatomyositis (with tapering of glucocorticoids); the TIS is a validated composite myositis index assessing muscle enzymes, both physician and patient assessments of overall health, and changes in muscle strength, physical function, skin and other disease activity; at Week 52, patients who received brepocitinib 30 mg once daily demonstrated significantly higher mean TIS scores compared with placebo (46.5 versus 31.2, respectively; treatment difference, 15.3; 95% confidence interval [CI], 6.7–24; P<0.001) indicating greater clinical response and disease control with brepocitinib
Boxed warning for serious infections, mortality, malignancy, major adverse cardiovascular events (MACE) and thrombosis
Dermatomyositis is a rare systemic autoimmune disease involving chronic inflammation that leads to progressive damage to the muscles, skin, lungs, joints, heart and gastrointestinal tract; management approaches are dependent on the presence and severity of myositis and the type and extent of skin and organ involvement; systemic glucocorticoids have historically been considered a cornerstone for treatment and are utilized in combination with steroid-sparing immunomodulatory agents (e.g., methotrexate, azathioprine, mycophenolate mofetil, hydroxychloroquine); IV immunoglobulin (IVIG) (e.g., Octagam 10%) is FDA-approved for dermatomyositis in adults and used for patients with severe/refractory disease; as a selective JAK/TYK2 inhibitor, Lisraya provides a new, oral targeted therapeutic approach for patients with dermatomyositis
Lisraya is available from Priovant Therapeutics

Aug. 28, 2026 — rusfertide (Mimrylo)

NDA approval; Breakthrough Therapy, Fast Track, Orphan Drug and Priority Review designations
Hepcidin mimetic
Indicated for the treatment of erythrocytosis in adults with polycythemia vera (PV)
Lyophilized powder in single-dose vials for reconstitution: 9.5 mg, 19 mg, 28 mg, 41 mg and 54 mg
Recommended starting dosage is given once weekly by subcutaneous (SC) injection with dose adjustments based on efficacy and safety; a complete blood count (CBC) should be performed every 2–4 weeks or as clinically indicated during dose modifications; patients and/or caregivers can administer following proper training
Approval was based on data from a multicenter, randomized, double-blind, placebo-controlled Phase 3 study (VERIFY; n=293) that evaluated adults with PV who were receiving SOC therapy (phlebotomy alone or phlebotomy plus one or more cytoreductive agents) and still required frequent phlebotomies; the primary endpoint was the proportion of patients between Week 20 and 32 of the study that achieved a response (defined as not being eligible for a phlebotomy); phlebotomy was based on a confirmed hematocrit ≥45% that is ≥3% (absolute) points higher than the baseline hematocrit or a hematocrit ≥48%; significantly more rusfertide patients (76.9%) achieved a response compared with those who received placebo (32.9%) resulting in a common risk difference of 43.8% (95% CI, 33.5%–54.2%; P<0.0001)
Other FDA-approved therapies for PV include the SC interferon alfa-2b (Besremi) indicated for adults with PV and the oral kinase inhibitor ruxolitinib (Jakafi/Jakafi XR) indicated for PV in adults with an inadequate response to or intolerance of hydroxyurea; as a first-in-class hepcidin mimetic, Mimrylo mimics the endogenous hormone hepcidin, resulting in regulation of iron levels and a subsequent decrease in red blood cell production
Mimrylo will be available from Takeda Pharmaceuticals; the launch timeframe is to be determined (TBD)

Sept. 3, 2026 — zilganersen (Zanvastro)

NDA approval; Breakthrough Therapy, Fast Track and Orphan Drug designations; Rare Pediatric Disease Priority Review Voucher; first FDA-approved treatment for Alexander disease
Glial fibrillary acidic protein (GFAP)-directed antisense oligonucleotide (ASO)
Indicated for the treatment of Alexander disease in pediatric and adult patients
Solution for injection: 56 mg/2.8 mL (20 mg/mL) in single-dose vials; supplied with artificial cerebrospinal fluid diluent (21 mL) in a single-dose vial for dilution of Zanvastro
Recommended dosage is administered every three months intrathecally using a lumbar puncture by or under the direction of HCPs experienced in performing lumbar punctures; administered as an intrathecal bolus injection over 1–3 minutes
Approval was based on data from a multicenter, randomized, double-blind, 60-week clinical study (n=49) in pediatric and adult patients with Alexander disease 2–65 years of age, and in an open-label substudy (n=4) in patients <2 years of age; the primary endpoint was evaluated in Stratum 1, which included patients (1) ≥5 years of age and, (2) if ≥18 years of age, onset of motor symptoms/signs within five years, and (3) demonstrated abnormality in gross motor skills; the primary endpoint was the difference in the mean percentage change in gait speed from baseline to Week 61, as assessed by the 10-meter walk test (10MWT) with zilganersen compared with control; there was a statistically significant difference in gait speed with zilganersen (-2.1%) compared with control (-35.4%) for an adjusted least squares mean difference of 33.3% (95% CI, 1.44–65.25, P=0.041); for patients 2–4 years of age, motor function was evaluated using the Gross Motor Function Measure-88 subscales for standing (Dimension D) and for walking, running and jumping (Dimension E); zilganersen-treated patients (n=4) exhibited improvement in the Dimensions D and E combined score, whereas patients who received control (n=3) exhibited decline
Zanvastro is the first disease-modifying treatment for pediatric and adult patients with Alexander disease, an ultra-rare, progressive, frequently fatal neurological disease caused by mutations in the GFAP gene that result in overproduction and accumulation of GFAP; this mutation leads to abnormal protein aggregates, known as Rosenthal fibers, inside astrocytes in certain regions of the central nervous system (CNS), resulting in damaged neurons and myelin; neurological manifestations include seizures, developmental regressions, difficulty walking, muscle weakness and increased intracranial pressure; as an ASO, Zanvastro decreases the production of abnormal GFAP protein, reducing accumulation in astrocytes and potentially slowing further neurological damage; prior to approval of Zanvastro, management was primarily symptomatic
Zanvastro is available from Ionis Pharmaceuticals

Sept. 4, 2026 — camizestrant (Etcamah)

NDA approval; Accelerated Approval, Assessment Aid and Breakthrough Therapy designations; Project Orbis review; the FDA also approved the companion diagnostic device, Guardant360 CDx assay, to identify patients with breast cancer with estrogen receptor 1 (ESR1) mutations
Estrogen receptor (ER) antagonist
Indicated in combination with a cyclin-dependent kinase (CDK) 4/6 inhibitor (abemaciclib [Verzenio], palbociclib [Ibrance] or ribociclib [Kisqali]) for the treatment of adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer upon detection of ESR1 mutation during aromatase inhibitor and CDK4/6 inhibitor therapy, based on an FDA-authorized test
Accelerated Approval was based on PFS, as measured from detection of ESR1 mutation; therefore, continued approval for this use may require demonstration of clinical benefit in a confirmatory trial
Oral tablets: 75 mg
Recommended dosage is taken orally once daily, with or without food
Approval was based on data from a randomized, double-blind, placebo-controlled, multicenter trial (SERENA-6; n=315) that evaluated switching to camizestrant versus continuing an aromatase inhibitor, in combination with a CDK4/6 inhibitor, in adults with ER-positive, HER2-negative locally advanced or metastatic breast cancer with detectable ESR1 mutations; the primary endpoint of median PFS was assessed at an interim analysis with a median follow-up of 12.6 months and was found to be significantly improved in the camizestrant plus CDK4/6 inhibitor arm (16 months) compared with the aromatase inhibitor plus CDK4/6 inhibitor arm (9.2 months) corresponding to a hazard ratio for disease progression or death of 0.44 (95% CI, 0.31–0.6; P<0.0001)
Boxed warning for arrhythmia risk with concomitant use of QTc interval prolonging drugs
The NCCN invasive breast cancer guidelines list elacestrant (Orserdu), imlunestrant (Inluriyo) or vepdegestrant (Veppanu) for HR-positive, HER2-negative, recurrent unresectable (local or regional) or metastatic breast cancer patients with an ESR1 mutation (all Category 2A, other recommended regimens) in certain settings; ESR1 mutations are the most common acquired resistance mutation during therapy with an aromatase inhibitor plus a CDK4/6 inhibitor; at initial diagnosis, <5% of patients have an ESR1 mutation, however after disease progression on an aromatase inhibitor, almost 40% of patients will exhibit this type of mutation; Etcamah is a next-generation, oral selective estrogen receptor degrader (SERD) and complete ER antagonist with efficacy in patients with emergent ESR1 tumor mutations
Etcamah is available from AstraZeneca Pharmaceuticals

Aug. 26, 2026 — leuprolide acetate (Vobrig)

505(b)(2) NDA approval
Gonadotropin releasing hormone (GnRH) agonist
Indicated for the treatment of central precocious puberty in pediatric patients
Solution for injection: 28 mg/2.8 mL single-patient-use prefilled pen
Recommended starting dosage is based on body weight and is given SC once daily by patients or caregivers following proper training; the daily dosage should be increased if total down regulation is not reached with the starting dose; to ensure adequate suppression, monitor with a GnRH stimulation test, basal luteinizing hormone (LH) levels or serum concentrations of sex steroid levels; bone age and height should be assessed every 6–12 months; discontinue Vobrig at the appropriate age for puberty onset
Vobrig will be available from Sun Pharmaceutical Industries; the launch timeframe is TBD

July 31, 2026 — rituximab-cdxx (Zimrixby)

BLA approval; 100 mg/10 mL (10 mg/mL) injection in a single-dose vial for IV use and 500 mg/50 mL (10 mg/mL) injection in a single-dose vial for IV use have received FDA approval as biosimilar to the same presentations of rituximab (Rituxan)
Zimrixby is approved for most of the same indications as reference drug Rituxan, with the exception that Rituxan carries additional indications for (1) patients ≥2 years of age with granulomatosis with polyangiitis (GPA; Wegener’s granulomatosis) and microscopic polyangiitis (MPA) in combination with glucocorticoids; (2) pediatric patients ≥6 months of age with mature B-cell non-Hodgkin lymphoma (NHL) and mature B-cell acute leukemia (previously untreated, advanced stage, cluster of differentiation [CD]20-positive, diffuse large B-cell lymphoma [DLBCL], Burkitt lymphoma [BL], Burkitt-like lymphoma [BLL] or mature B-cell acute leukemia [B-AL] in combination with chemotherapy); and (3) moderate-to-severe pemphigus vulgaris in adults
Zimrixby will be available from Fresenius Kabi USA; the launch timeframe is TBD

Aug. 20, 2026 — pegcetacoplan (Empaveli)

Apellis Pharmaceuticals; complement inhibitor
Indication update: indication for adults and pediatric patients ≥12 years of age with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) updated to describe additional benefit of reducing loss of kidney function; indication previously only included benefit of proteinuria reduction
Dosage is administered SC twice weekly via a commercially available infusion pump or single-use, on-body injector; dosing for pediatric patients is weight based (<35 kg, 35–49 kg or ≥50 kg); patients/caregivers may administer following proper training by an HCP
Other indication: paroxysmal nocturnal hemoglobinuria (PNH) in adults

Aug. 24, 2026 — nipocalimab-aahu (Imaavy)

Janssen Biotech; neonatal Fc receptor blocker; Fast Track, Orphan Drug and Priority Review designations; first drug to be approved for warm autoimmune hemolytic anemia (wAIHA)
New indication: wAIHA in adult and pediatric patients ≥12 years of age currently or previously treated with corticosteroids
Administered via IV infusion by an HCP every four weeks; dosage is weight based and is given over ≥30 minutes for the initial dose and over ≥15 minutes for maintenance doses
Other indication: generalized myasthenia gravis (gMG) in adult and pediatric patients ≥12 years of age who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Aug. 25, 2026 — tislelizumab-jsgr (Tevimbra)

BeOne Medicines; programmed cell death receptor-1 (PD-1)-blocking antibody; Assessment Aid, Priority Review and Real-Time Oncology Review (RTOR) designations; Project Orbis review
New indication: in combination with zanidatamab-hrii (Ziihera) and fluoropyrimidine- and platinum-containing chemotherapy in adults for first-line treatment of HER2-positive (immunohistochemistry [IHC] 3+ or IHC 2+/in situ hybridization [ISH]+), unresectable, locally advanced or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma, as detected by an FDA-authorized test
Dosage is administered by an HCP as an IV infusion every two, three, four or six weeks, in combination with Ziihera and fluoropyrimidine- and platinum- containing chemotherapy, until disease progression or unacceptable toxicity; initial infusion rates vary from 60 to 120 minutes, based on dosage; subsequent infusion rates can be gradually decreased to 30 minutes if prior infusions are tolerated
Other indications are detailed in the product label

Aug. 25, 2026 — zanidatamab-hrii (Ziihera)

Jazz Pharmaceuticals; bispecific HER2-directed antibody; Assessment Aid, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review and RTOR designations; Project Orbis review; FDA also approved two companion diagnostic devices — PATHWAY anti-HER-2/neu (4B5) Rabbit Monoclonal Primary Antibody and VENTANA HER2 Dual ISH DNA Probe Cocktail (Ventana Medical Systems/Roche Diagnostics) — for identifying eligible patients for treatment with Ziihera
New indication: treatment of gastroesophageal adenocarcinoma (1) in combination with fluoropyrimidine- and platinum-containing chemotherapy and tislelizumab-jsgr (Tevimbra) as first-line treatment for adults with HER2-positive (IHC 3+ or IHC 2+/ISH+), unresectable, locally advanced or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma, as detected by an FDA-approved test, and (2) in combination with fluoropyrimidine- and platinum-containing chemotherapy as first-line treatment for adults with HER2-positive (IHC 3+), unresectable, locally advanced or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma, as detected by an FDA-approved test
Administered by an HCP as a weight-based (<70 kg or ≥70 kg) IV infusion every two or three weeks, in combination with fluoropyrimidine- and platinum-containing chemotherapy (with or without Tevimbra), until disease progression or unacceptable toxicity; first infusion is administered over 120 minutes; the second infusion can be administered over 90 minutes, and subsequent infusions can be given over 60 minutes if previous infusions were well-tolerated
Other indication: Accelerated Approval for adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer, as detected by an FDA-authorized test

Aug. 27, 2026 — lenacapavir (Sunlenca)

Gilead Sciences; human immunodeficiency virus type 1 (HIV-1) capsid inhibitor
New indication: oral loading as a component of the initiation regimen for bictegravir/lenacapavir (Bixlenvo), a complete regimen for treatment of HIV-1 infection, in adults to replace the current antiretroviral (ARV) regimen in virologically suppressed patients (HIV-1 ribonucleic acid [RNA] <50 copies/mL) on a stable ARV regimen with no known or suspected resistance to the individual components of Bixlenvo
Administered as two tablets taken orally on Days 1 and 2 as part of an initiation regimen in combination with Bixlenvo
Other indication: treatment of HIV-1 infection, in combination with other ARV(s), in heavily treatment-experienced adults with multidrug resistant HIV-1 whose current ARV regimen is failing due to resistance, intolerance or safety considerations

Aug. 28, 2026 — ropeginterferon alfa-2b-njft (Besremi)

PharmaEssentia; interferon alfa-2b
New indication: essential thrombocythemia in adults
Administered as an SC injection every two weeks, with initial dose escalation during Weeks 2 and 4; maintenance dose should be given every two weeks unless dose modification is needed for safety or tolerability; patients/caregivers may administer if deemed appropriate by an HCP
Other indication: polycythemia vera in adults

Aug. 28, 2026 — ustekinumab (Stelara)

Janssen Biotech; human interleukin-12 and -23 antagonist; Orphan Drug designation
Expanded indication: moderately to severely active ulcerative colitis (UC) in pediatric patients ≥2 years of age; previously, the UC indication was limited to adults
Induction dosage for pediatric patients weighing ≥10 kg is a single IV infusion with dosage based on patient’s body weight at the time of dosing (10–25 kg, 26–55 kg, 56–85 kg, >85 kg); maintenance dosage is weight based (10–35 kg or >35 kg) and is administered SC eight weeks after the initial IV dose and every eight weeks thereafter; administration by an HCP is recommended for pediatric patients
Other indications are detailed in the product label

Sept. 9, 2026 — sevabertinib (Hyrnuo)

Bayer Healthcare; kinase inhibitor; Accelerated Approval; Assessment Aid, Breakthrough Therapy, Orphan Drug and Priority Review designations; Project Orbis review
Expanded indication: adults with locally advanced or metastatic non-squamous NSCLC whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-authorized test; previously only approved for this indication in patients who had received prior systemic therapy
Administered orally twice daily with food until disease progression or unacceptable toxicity

Traditional

None

Aug. 27, 2026 — bictegravir/lenacapavir (Bixlenvo)

NDA approval; provides a once-daily single tablet regimen for virologically suppressed adults with HIV-1
Fixed-dose combination of bictegravir, an HIV-1 integrase strand transfer inhibitor (INSTI), and lenacapavir, an HIV-1 capsid inhibitor
Indicated as a complete regimen for treatment of HIV-1 in adults to replace the current ARV regimen in virologically suppressed patients (HIV-1 RNA <50 copies/mL) on a stable ARV regimen with no known or suspected resistance to the individual components of Bixlenvo
Oral tablet: 75 mg bictegravir and 50 mg lenacapavir
Recommended dosage includes a two-day initiation regimen of one oral Bixlenvo tablet plus two oral lenacapavir (Sunlenca) tablets, followed by a maintenance dose of one Bixlenvo tablet once daily
Bixlenvo is available from Gilead Sciences

Aug. 18, 2026 — lerodalcibep-liga (Lerochol)

LIB Therapeutics; proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor; FDA also approved a new 300 mg/1.2 mL single-dose autoinjector presentation
Indication update: indication as adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH), updated to state that cardiovascular (CV) outcomes trials have shown that reducing LDL-C lowers risk of MACE in adults at increased risk when treated with statins or monoclonal antibody PCSK9 inhibitors as an add-on to statin therapy
Administered as a once-monthly SC injection; patients/caregivers can administer following proper training
New autoinjector presentation is expected to be available by January 2027

Aug. 25, 2026 — dolutegravir tablets for oral suspension (Tivicay PD)

ViiV Healthcare; HIV-1 INSTI; Priority Review designation
Expanded indication: HIV-1 infection in newborns from birth up to 4 weeks of age weighing ≥2 kg that are treatment-naïve, or treatment-experienced but INSTI-naïve, in combination with other ARV agents; previously only approved for adults and pediatric patients ≥4 weeks of age weighing ≥3 kg
Recommended dosage in term neonates (≥37 weeks of gestation) <4 weeks of age and weighing ≥2 kg is based on age; administered orally once every other day from birth to 14 days and once daily from 15 days to <4 weeks; tablets should be swallowed whole or dispersed in water

Aug. 27, 2026 — tirzepatide (Mounjaro)

Eli Lilly and Company; glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist; first and only dual GIP/GLP-1 receptor agonist to be approved for CV risk reduction
New indication: to reduce the risk of MACE — including CV death, nonfatal myocardial infarction (MI) or nonfatal stroke — in adults with type 2 diabetes mellitus (T2DM) who are at high risk for these events
Administered as an SC injection once weekly; Mounjaro should be initiated at the lowest dosage, and dosage should be gradually escalated after at least four weeks on the current dose
Other indication: adjunct to diet and exercise to improve glycemic control in adults and pediatric patients ≥10 years of age with T2DM

Sept. 9, 2026 — aminolevulinic acid hydrochloride (Ameluz)

Biofrontera; porphyrin precursor
New indication: treatment of superficial basal cell carcinoma (sBCC) in adults, in combination with photodynamic therapy (PDT) using a BF-RhodoLED or RhodoLED XL lamp, a narrowband, red light illumination source
Administered topically by an HCP as a 1-mm-thick layer to sBCC lesion(s), in combination with PDT, in two sessions spaced 1–2 weeks apart; should be applied to cover lesion(s) and to include about 5–10 mm of surrounding skin; treatment is a multistage process requiring administration of both Ameluz and a BF-RhodoLED or RhodoLED XL light; lesions that have not completely resolved after three months should be retreated with two additional sessions, spaced 1–2 weeks apart
Other indication: lesion- and field-directed treatment of actinic keratosis of mild-to-moderate severity on the face and scalp in adults, in combination with PDT using a BF-RhodoLED or RhodoLED XL lamp

First generic drug launches

Aug. 26, 2026 — nisoldipine (Sular)

Avet Pharmaceuticals/AMTA launched oral extended-release tablets (8.5 mg, 17 mg) generic to Azurity Pharmaceutical’s Sular
Dihydropyridine calcium channel blocker indicated for the treatment of hypertension; may be used alone or in combination with other antihypertensive agents
Recommended dosage is taken orally once daily with the dosage individualized based on blood pressure response; tablets should be taken on an empty stomach, either one hour prior to or two hours after a meal
United States annual sales for Sular in 2025 are <$10 million

10MWT 10-meter walk test
AAFP American Academy of Family Physicians
AAN American Academy of Neurology
AAP American Academy of Pediatrics
AAV adeno-associated virus
AChR acetylcholine receptor
ACOG American College of Obstetricians and Gynecologists

ACVR1 activin A receptor type 1

AASM American Academy of Sleep Medicine

AHA American Heart Association
AHS American Headache Society
ARV antiretroviral
ASA American Stroke Association

ASO antisense oligonucleotide
B-AL B-cell acute leukemia
BL Burkitt lymphoma
BLA Biologics License Application

BLL Burkitt-like lymphoma

C3G C3 glomerulopathy
CBC complete blood count

CD20 cluster of differentiation 20

CDC Centers for Disease Control and Prevention

CDK cyclin-dependent kinase

CI confidence interval

CNPV Commissioner's National Priority Voucher

CNS central nervous system
COVID-19 coronavirus disease 2019

CR complete response
CRC colorectal cancer
CT computed tomography
CV cardiovascular

Dd daratumumab/hyaluronidase-fihj and dexamethasone

DKA diabetic ketoacidosis
DLBCL diffuse large B-cell lymphoma
DM dermatomyositis
DMARDs disease-modifying antirheumatic drugs

DOSE Nonfatal Drug Overdose Surveillance and Epidemiology System

DTaP diphtheria, tetanus and acellular pertussis
DVd daratumumab/hyaluronidase-fihj, bortezomib and dexamethasone

ER estrogen receptor

ES Endocrine Society
ESR1 estrogen receptor 1
FDA Food and Drug Administration

FOP fibrodysplasia ossificans progressiva
G6Pase glucose-6-phosphatase (enzyme)
G6PC glucose-6-phosphatase (gene)
gc genome copies
GFAP glial fibrillary acidic protein
GIP glucose-dependent insulinotropic polypeptide
GLP-1 glucagon-like peptide-1

gMG generalized myasthenia gravis
GnRH gonadotropin releasing hormone
GPA granulomatosis with polyangiitis
GSDIa glycogen storage disease type Ia
GTPase guanosine triphosphatases
HCP healthcare professional

HeFH heterozygous familial hypercholesterolemia
HER2 human epidermal growth factor receptor 2
HIV-1 human immunodeficiency virus type 1
HO heterotopic ossification

HR hormone receptor
IC-MPGN immune-complex membranoproliferative glomerulonephritis
IDSA Infectious Diseases Society of America
IHC immunohistochemistry

INSTI integrase strand transfer inhibitor
ISH in situ hybridization
IV intravenous

IVIG intravenous immunoglobulin
JAK Janus kinase

KRAS Kirsten rat sarcoma viral oncogene homologue

LDL-C low-density lipoprotein cholesterol

LH luteinizing hormone
MACE major adverse cardiovascular events

MI myocardial infarction

MM multiple myeloma

MMR measles, mumps and rubella

MPA microscopic polyangiitis
MRD minimal residual disease

mRNA messenger ribonucleic acid

MuSK muscle-specific tyrosine kinase

NCCN National Comprehensive Cancer Network
NDA New Drug Application

NHL non-Hodgkin lymphoma

NSCLC non-small cell lung cancer

NVSS National Vital Statistics System
OS overall survival

PCSK9 proprotein convertase subtilisin/kexin type 9
PD-1 programmed cell death receptor-1
PDT photodynamic therapy

PFS progression-free survival

PCSK9 proprotein convertase subtilisin/kexin type 9
PD-1 programmed cell death protein 1

PET positron emission tomography

PFS progression-free survival

PNH paroxysmal nocturnal hemoglobinuria

PV polycythemia vera

RAS rat sarcoma
REMS Risk Evaluation and Mitigation Strategy
RMAT Regenerative Medicine Advanced Therapy

RNA ribonucleic acid

RRMM relapsed or refractory multiple myeloma
RSV respiratory syncytial virus

RTOR Real-Time Oncology Review

SARS-CoV-2 severe acute respiratory syndrome coronavirus 2
sBCC superficial basal cell carcinoma

SC subcutaneous

SERD selective estrogen receptor degrader

SOC standard of care
SUDORS State Unintentional Drug Overdose Reporting System

T2DM type 2 diabetes mellitus

TBD to be determined

TIS Total Improvement Score
TKD tyrosine kinase domain

TYK2 tyrosine kinase 2

UC ulcerative colitis

wAIHA warm autoimmune hemolytic anemia

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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