publications

Avacopan (Tavneos) update

The Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) has proposed that avacopan oral capsule (Tavneos) be withdrawn from commercialization in the United States. This conclusion is based on recent evidence indicating that Tavneos has not demonstrated efficacy for its FDA-approved indication, and because it has been determined that false statements were included in its initial approval application. Tavneos is a complement 5a receptor (C5aR) antagonist that was approved in 2021 as an adjunctive treatment for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (granulomatosis with polyangiitis [GPA] and microscopic polyangiitis [MPA]), in combination with standard therapy including glucocorticoids.

In January 2026, the FDA requested that ChemoCentryx (a subsidiary of Amgen) voluntarily withdraw Tavneos. This request was based on concerns regarding re-adjudication of nine patients in the ADVOCATE trial (n=331). According to the FDA, unblinded staff involved in the clinical trial altered the results to make the drug appear effective, even though the original analysis did not support this outcome. At the end of January, Amgen stated it did not intend to withdraw Tavneos from commercialization and was unaware of any patient data issues. Furthermore, the manufacturer confirmed ongoing support for Tavneos as an effective treatment for ANCA-associated vasculitis.

The FDA also stated ChemoCentryx violated FDA regulations by not submitting the original analysis as part of the initial approval application. Based on the findings, CDER has concluded they cannot support that Tavneos is effective for the FDA-approved use. A notice of opportunity for a hearing (NOOH) has been issued by the FDA to ChemoCentryx. Additionally, a Drug Safety Communication was issued by the FDA in March 2026 regarding concerns of serious drug-induced liver injury (DILI). Tavneos will remain commercially available until the manufacturer decides to remove it from market or the FDA Commissioner mandates its removal. If ChemoCentryx requests a hearing, the FDA Commissioner will evaluate the necessity of conducting a public hearing. After the hearing (if deemed necessary) concludes, the Commissioner will then determine the withdrawal of Tavneos. Amgen has published a FAQ stating Tavneos remains commercially available and there is no change to its approval status, use or availability.

Eli Lilly has announced topline results from the event-driven, randomized, open-label, Phase 3 ACHIEVE-4 trial, which evaluated orforglipron (Foundayo) compared to insulin glargine in adults with type 2 diabetes mellitus (T2DM) and obesity or overweight at increased cardiovascular (CV) risk (n=2,749). Foundayo is an oral glucagon-like peptide-1 (GLP-1) receptor agonist for weight management in adults with obesity or overweight in the presence of at least one weight-related comorbid condition. The ACHIEVE-4 study randomized patients who had taken at least one and no more than three oral anti-diabetic medications for at least 90 days prior to study start to receive either escalating doses of orforglipron or insulin glargine. Results showed that orforglipron was noninferior to insulin glargine for the primary endpoint of time to first occurrence of any major adverse CV events (MACE-4; myocardial infarction [MI], stroke, hospitalization for unstable angina or CV death). Additionally, orforglipron showed superior improvements in HbA1c and body weight from mean baseline to week 52 and through week 104 compared to insulin glargine.

The FDA announced a proposal to exclude semaglutide, tirzepatide and liraglutide from the 503B bulk drug substances list. These exclusions are based on the lack of clinical need for outsourcing facilities to compound these drugs from bulk substances. Clinical need is determined by patient safety and medical necessity under the law. In general, outsourcing facilities cannot compound drugs using bulk drug substances unless the substance is present on the 503B bulks list or the product is on the FDA drug shortage list. Prior to making a final determination regarding exclusion of these substances from the list, the FDA is collecting comments electronically through the Federal Register. Comments must be submitted by June 29, 2026, for consideration.

Following President Trump's Executive Order to accelerate access of treatments for serious mental illness, the FDA issued national priority vouchers for psilocybin for treatment-resistant depression and major depressive disorder and for methylone for post-traumatic stress disorder (PTSD). The FDA has also authorized an early phase clinical trial of noribogaine hydrochloride for alcohol use disorder to proceed following the Investigational New Drug (IND) submission. The Agency is planning to release final guidance with recommendations for evaluating serotonin-2A agonists and related products.
The FDA announced steps for implementation of real-time clinical trials (RTCT). First, two proof-of-concept clinical trials have been initiated; these trials will report endpoints and data signals to the FDA in real time. Second, a request for information (RFI) has been released on the proposed pilot program for RTCT that will launch in summer 2026.
The FDA is encouraging sponsors of FDA-approved testosterone replacement therapy (TRT) products to pursue a potential new indication for low libido in men with idiopathic hypogonadism. This announcement follows a December 2025 expert-panel meeting, where it was determined that TRT is a potentially safe and effective treatment for certain men with this condition, which is characterized by low testosterone levels without a known cause. FDA-approved TRT products are currently only indicated for men with hypogonadism related to known structural or genetic causes. The Agency stated that approval of a new indication remains contingent upon sponsors demonstrating effectiveness and ensuring that the benefits surpass the associated risks.
The Agency has expanded AI capabilities through the launch of Elsa 4.0, built within a FedRAMP High secure Google Cloud Platform (GCP) environment. Elsa serves as an internal AI platform for use by any FDA staffer. Additionally, the FDA streamlined over 40 application and data submission sources and systems throughout the entire Agency to form a new tool called HALO (Harmonized AI & Lifecycle Operations for Data). Elsa and HALO are in the process of being integrated to ease efficiency of use of both systems.
The FDA has issued a reminder to over 2,200 medical product companies and researchers of reporting requirements for clinical trial results. An internal analysis found that 29.6% of studies likely requiring mandatory reporting have not submitted results to ClinicalTrials.gov. Mandatory reporting applies to interventional studies with a connection to the U.S. and an FDA-regulated product that have passed the reporting deadline; Phase 1 and device feasibility studies are excluded. The FDA is requesting that certain sponsors and researchers voluntarily comply with the clinical trial results submission within one year after trial completion.

As of May 4, 2026, semaglutide (Ozempic) tablets in the strengths of 1.5 mg, 4 mg and 9 mg are commercially available in the U.S. This is the only oral GLP-1 approved for primary and secondary CV risk reduction of MI, stroke and death in adults with T2DM at high risk for these events, including those with existing heart disease. Semaglutide for T2DM has been available orally as Rybelsus, but this is a different formulation and set of doses.
The FDA has announced that tazemetostat (Tazverik) is being voluntarily withdrawn from commercialization in the U.S. It has been found that the risks of therapy outweigh the benefits due to an increased rate of hematologic second primary malignancies (SPMs). Tazverik was initially granted Accelerated Approval for patients ≥ 16 years of age with metastatic or locally advanced epithelioid sarcoma ineligible for complete resection. Subsequently, Accelerated Approval was granted for treatment of certain adults with relapsed or refractory follicular lymphoma. SPMs were a known risk (rate, 1.7%) at the time of approval; however, data from the Symphony 1 study demonstrated a rate of SPMs of over 5% across a median treatment duration of 15.8 months (range, 6.9 to 33 months).
The U.S. Department of Health and Human Services (HHS) has announced an action plan to mitigate psychiatric overprescribing. The plan includes a multifaceted approach combining education and outreach, program and policy initiatives and efforts that bridge research with practical application. The intent is to prevent unnecessary initiation of these medications and support tapering/discontinuation for those not receiving benefit. The announcement includes a Dear Colleague Letter emphasizing informed consent and shared decision-making as well as access to evidence-based non-drug therapies and when appropriate, deprescribing.

The American Academy of Dermatology (AAD) has published recommendations for prevention and management of pediatric atopic dermatitis (AD) in two separate guidelines, both published in the Journal of the American Academy of Dermatology. AAD’s prior guidelines on this subject were published in 2014. Moisturizing skin care is conditionally recommended to reduce the occurrence of AD; recommendations are also provided on the use of topical therapies, phototherapy and systemic therapies for treating AD in pediatric patients.
The American College of Physicians (ACP) published a guidance statement on screening for breast cancer in asymptomatic, average-risk adult females. The statement has been published in the Annals of Internal Medicine and includes five guidance statements.
The American Society of Hematology (ASH) has published a clinical practice guideline for diagnosis and management of severe and very severe acquired aplastic anemia. The panel issued 33 recommendations and four good practice statements with recommendations emphasizing prioritization of hematopoietic cell transplantation for younger patients who have an available matched sibling or unrelated donor and as a second-line therapy after failure of immunosuppressives.
The ASH and the International Society on Thrombosis and Haemostasis (ISTH) have published guidelines on anticoagulant prophylaxis of pediatric patients at risk of venous thromboembolism (VTE). Although high-quality data on anticoagulant prophylaxis for pediatric VTE prevention is minimal, the panel issued 12 recommendations, including conditional recommendations suggesting no anticoagulant prophylaxis for pediatric patients with solid cancer, trauma or critical illness.
The Institute for Clinical and Economic Review (ICER) has published a white paper on the Accelerated Approval pathway for prescription drugs, including information on its evolution as well as recent regulatory and market changes. This paper expands on an earlier ICER white paper from 2021 and features additional policies that could be considered to enhance the pathway even further.

Drug Approvals

Specialty

April 23, 2026 – lunsotogene parvec-cwha (Otarmeni, formerly known as DB-OTO)

Biologics License Application (BLA) approval; Accelerated Approval, Commissioner’s National Priority Voucher (CNPV), Fast Track, Orphan Drug, Rare Pediatric Disease, Regenerative Medicine Advanced Therapy (RMAT); approval marks the first dual adeno-associated virus (AAV) vector-based gene therapy to receive FDA approval and first in vivo gene therapy for otoferlin (OTOF)-related hearing loss; Otarmeni is also the first gene therapy and second new molecular entity approved under the CNPV pilot
Adeno-associated virus vector-based gene therapy
Indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 decibels Hearing Level [dB HL]) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear; Accelerated Approval based on improvement of hearing sensitivity assessed by average pure tone audiometry (PTA) at week 24; continued approval for this use may require demonstration of benefit in a confirmatory clinical trial
Limitations of use: not recommended in patients in whom preoperative imaging demonstrates that access to the inner ear is not feasible including those with abnormal mastoid pneumatization or clinically significant anatomic variations of the middle ear and inner ear
Sterile suspension for intracochlear infusion: nominal titer of 3 x 10¹³ vector genomes (vg)/mL in each single-dose vial (extractable volume of 0.63 mL)
Recommended dosage for each ear is 7.2 x 10¹² vg in a total volume of 0.24 mL, administered by a single-dose intracochlear infusion by a healthcare professional (HCP) under anesthesia
Approval was based on a single-arm, multicenter, ongoing clinical trial (CHORD) conducted in 24 pediatric patients 10 months old to 16 years of age with OTOF gene-associated profound sensorineural hearing loss (defined as >90 dB HL on pure tone audiometry averaged across 0.5, 1, 2, and 4 kHz and auditory brainstem response); 20 of the 24 patients were evaluable for efficacy; the primary endpoint was the achievement of a hearing sensitivity threshold of ≤ 70 dB HL assessed by average pure tone audiometry at 24 weeks after product administration; 80% of the 20 evaluable patients achieved the primary endpoint, a finding not anticipated in the natural history of the disease in the absence of therapeutic intervention
Half of congenital hearing loss is caused by genetic mutations with OTOF gene variants being responsible for 2% – 8% of inherited cases not associated with a specific condition or disease; only 50 newborns per year in the U.S. are born with OTOF-related hearing loss making it an ultra-rare condition; Otarmeni is a one-time biologic-device combination that allows for administration of the gene therapy as a single dose per ear surgically into the cochlea via a syringe/catheter that is connected to an infusion pump (intracochlear infusion); the drug delivers a functional copy of the OTOF gene to inner hair cells restoring OTOF production and auditory signaling; Otarmeni is the first disease modifying treatments available for OTOF-related deafness with related hearing loss considered to be permanent and managed with use of cochlear implants which can restore partial hearing, but with limited sound quality and speech recognition
Otarmeni will be available from Regeneron with launch timeframe to be determined (TBD); Regeneron will provide Otarmeni at no cost to eligible patients in the U.S. however this does not include the cost of administration which occurs under general anesthesia

May 1, 2026 – vepdegestrant (Veppanu)

New Drug Application (NDA) approval; Assessment Aid; first FDA-approved PROteolysis TArgeting Chimera (PROTAC), a type of heterobifunctional protein degrader therapy
Heterobifunctional protein degrader
Indicated for the treatment of adults with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2), 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; patients should be selected for treatment based on the presence of ESR1 mutation; FDA also approved the Guardant360 CDx as a companion diagnostic device for identifying patients with breast cancer with this mutation
Oral tablets: 100 mg and 200 mg
Recommended dosage is taken orally once daily with food
Approval was based on data from a randomized, open-label, active-controlled multicenter, Phase 3 trial (VERITAC-2; n=624) that enrolled adults with ER-positive, HER2-negative advanced breast cancer who had received one previous line of cyclin-dependent kinase (CDK) 4 and 6 (4/6) inhibitor therapy plus one line of endocrine therapy (and up to one additional line of endocrine therapy); patients were randomized 1:1 to receive vepdegestrant orally once every day of each 28-day cycle or fulvestrant intramuscularly on day 1 and day 15 of cycle 1 and on day 1 of subsequent cycles; in the patients with ESR1 mutations (n=270), the median progression-free survival (PFS) was significantly improved with vepdegestrant compared to fulvestrant (5 months versus 2.1 months, respectively; hazard ratio, 0.58; 95% confidence interval [CI], 0.43 to 0.78; p<0.001)
The National Comprehensive Cancer Network (NCCN) breast cancer guidelines include vepdegestrant as a category 2A other recommended option for patients with HR-positive, HER2-negative, ESR1 mutation breast cancer patients with disease progression after at least one line of endocrine therapy + CDK 4/6 inhibitor in the metastatic setting; other similarly recommended therapies include elacestrant (Orserdu) for patients with disease progression during or after a prior line of aromatase inhibitor plus CDK 4/6 inhibitor therapy in the adjuvant or metastatic setting and imlunestrant (Inluriyo) for patients with disease progression during or after a prior line of aromatase inhibitor with or without CDK 4/6 inhibitor therapy in the adjuvant or metastatic setting
Veppanu will be available from Pfizer with launch timeframe TBD

May 13, 2026 – sonrotoclax (Beqalzi)

NDA approval; Accelerated Approval, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, Project Orbis
B-cell lymphoma 2 (BCL-2) inhibitor
Indicated for the treatment of adults with relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor; Accelerated Approval based on response rate and durability of response; continued approval for this use may require demonstration of benefit in confirmatory clinical trials
Oral tablets: 1 mg, 5 mg, 20 mg, and 80 mg
Recommended dosage is taken orally once daily following completion of a four-week dose ramp-up schedule; tablets should be taken whole with food and water; prophylaxis for tumor lysis syndrome (TLS) should be provided; patients should be assessed for risk of TLS and whether hospitalization is needed for monitoring
Approval was based on a Phase 1/2, single-arm, multicenter clinical trial (n=103) enrolling adults with relapsed or refractory MCL who previously received anti-CD20–based therapy and a BTK inhibitor; patients enrolled had received a median of three prior lines of therapy (range, 1 to 8) with 89% of patients having received at least two prior lines of therapy and 60% having received at least three prior lines; the overall response rate was 52% (95% CI, 42 to 62) with 16% of patients achieving a complete response and 37% having a partial response; the median duration of response was 15.8 months and the median time to response was 1.9 months (range, 1.6 to 6.2)
According to the NCCN B-cell lymphoma guidelines, second-line or subsequent therapy options for MCL include preferred therapies such as covalent BTK inhibitors given continuously (acalabrutinib [Calquence] or zanubrutinib [Brukinsa]) or lenalidomide given continuously in combination with rituximab; other recommended regimens in this setting include the covalent BTK inhibitor ibrutinib (Imbruvica) with or without rituximab; there are a number of other potential therapies considered useful in certain circumstances as well as therapies specifically for progression after a prior covalent BTK inhibitor: non-covalent BTK inhibitor given continuously (pirtobrutinib [Jaypirca]) or T-cell mediated therapy (e.g., CAR T cell therapies or bispecific antibody therapies); Beqalzi is the only BCL2 inhibitor approved for relapsed/refractory MCL and provides a new mechanism for patients who have received at least two lines of systemic therapy, including BTK inhibitor
Beqalzi will be available from BeOne Medicines with launch timeframe TBD

April 24, 2026 – anifrolumab-fnia subcutaneous (SC) (Saphnelo)

BLA approval; previously approved as an intravenous (IV) formulation in a single-dose vial for IV infusion that requires administration by an HCP over about 30 minutes
Type I interferon (IFN) receptor antagonist
Indicated for the treatment of adults with moderate to severe systemic lupus erythematosus (SLE), who are receiving standard therapy
Solution for injection: 120 mg/0.8 mL in a single-dose prefilled syringe or single-dose autoinjector (pen)
Recommended SC dosage is given once every week; patients/caregivers can administer Saphnelo SC using the prefilled syringe or pen following proper training; to transition patients from IV administration to SC administration, administer the first SC injection approximately two weeks after the last IV infusion
Saphnelo autoinjector is available from AstraZeneca; launch timeframe for prefilled syringe is TBD

May 1, 2026 – ruxolitinib extended-release tablets (Jakafi XR)

NDA approval; approved also as an oral immediate-release tablet (5 mg, 10 mg, 15 mg, 20 mg and 25 mg) taken twice daily
Kinase inhibitor
Indicated for the treatment of: (1) intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis, and post-essential thrombocythemia myelofibrosis in adults, (2) polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea, (3) steroid-refractory acute graft-versus-host disease in adult and pediatric patients ≥ 12 years of age and (4) chronic graft-versus-host disease after failure of one or two lines of systemic therapy in adult and pediatric patients ≥ 12 years of age
Extended-release tablets: 11 mg, 22 mg, 33 mg, 44 mg and 55 mg
Recommended dosage is dependent on indication and is individualized based on safety and efficacy; for example, the starting dose for myelofibrosis is based on the patient’s baseline platelet count; Jakafi XR is taken orally once daily
Jakafi XR is available from Incyte

May 1, 2026 – trabectedin (Evdi)

505(b)(2) NDA approval; trabectedin (Yondelis) is also available as a 1 mg lyophilized powder in a single-dose vial for reconstitution (same indication as Evdi)
Alkylating drug
Indicated for the treatment of adults with unresectable or metastatic liposarcoma or leiomyosarcoma who received a prior anthracycline-containing regimen
Solution for injection: 1 mg/20 mL (0.05 mg/mL)
Recommended dosage is based on body surface area (BSA) given as a 24-hour IV infusion, every three weeks through a central venous line; premedication with dexamethasone is required prior to each infusion
Evdi will be available from Apotex with launch timeframe TBD

April 29, 2026 – insulin glargine-aldy (Langlara)

BLA approval; 100 units/mL in a 3 mL single-patient-use prefilled pen for SC use has received FDA approval as an interchangeable biosimilar to the same presentation of insulin glargine (Lantus); the FDA also approved unbranded biological product labeling
Langlara will be available from Sunshine Lake with launch timeframe TBD

May 7, 2026 – pegfilgrastim-pccg (Ennumo)

BLA approval; 6 mg/0.6 mL single-dose prefilled syringe for SC use has received FDA approval as a biosimilar to the same presentation of pegfilgrastim (Neulasta) 
Ennumo will be available from Accord with launch timeframe TBD

April 13, 2026 – sparsentan (Filspari)

Travere; endothelin and angiotensin II receptor antagonist; first FDA-approved therapy for the treatment of focal segmental glomerulosclerosis (FSGS)
New indication: to reduce proteinuria in adult and pediatric patients ≥ 8 years of age with FSGS without nephrotic syndrome
Administered orally once daily for 14 days, then increased to the recommended dose, as tolerated; dosing is weight based depending on if the patient is > 50 kg or ≤ 50 kg; taken with water prior to the morning or evening meal
Other indication: to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression

April 15, 2026 – ustekinumab (Stelara)

Janssen Biotech; human interleukin-12 and -23 antagonist
Expanded indication: treatment of pediatric patients ≥ 2 years of age with moderately to severely active Crohn’s disease; previously only indicated for moderately to severely active Crohn’s disease in adults
Administered as an IV induction dosage in pediatric patients ≥ 10 kg: single IV infusion using weight-based dosing; recommended SC maintenance dose is weight-based for patients weighing 10 kg to 35 kg or a fixed dose for patients weighing > 35 kg; administered eight weeks after the initial IV dose and then every eight weeks thereafter
Other indications are detailed in the product label

April 17, 2026 – secukinumab (Cosentyx)

Novartis; human interleukin-17A antagonist
Expanded indication: for the treatment of active ankylosing spondylitis (AS) in patients ≥ 12 years of age; previously, indicated for active AS in adults only
Administered as a weight-based SC injection at weeks zero, one, two, three, and four and every four weeks thereafter; Cosentyx can be administered by an adult caregiver for pediatric patients after proper training in SC injection technique
Other indications are detailed in the product label

April 20, 2026 – teplizumab-mzwv (Tzield)

Provention Bio; CD3-directed antibody; a Boxed Warning for viral reactivation (e.g., EBV, CMV) has also been added to the label
Expanded indication: to delay the onset of stage 3 type 1 diabetes (T1D) in pediatric patients between 1 to 8 years of age with stage 2 T1D; previously only indicated to delay the onset of stage 3 T1D in adults and pediatric patients aged ≥ 8 years old with stage 2 T1D
Administered as an IV infusion over a minimum of two hours in pediatric patients 1 to < 8 years of age (30-minute infusion for adults and pediatric patients ≥ 8 years old); dosing is based on BSA and is given once daily for 14 consecutive days, initiated with a five day titration schedule to the maintenance dose on day five (continued through day 14)

April 22, 2026 – dupilumab (Dupixent) 

Regeneron; interleukin-4 receptor alpha antagonist
Expanded indication: for chronic spontaneous urticaria (CSU) to include pediatric patients aged 2 years to < 12 years of age who remain symptomatic despite H1 antihistamine treatment (not indicated for other forms of urticaria); previously, the CSU indication was only for the treatment of adult and pediatric patients ≥ 12 years of age with CSU who remain symptomatic despite H1 antihistamine treatment
Administered as SC injection under the guidance of an HCP; patients/caregivers can administer following proper training; for pediatric patients 2 to 5 years of age the dose is based on body weight given every four weeks (no initial loading dose is required); dosage for pediatric patients 6 to 17 years of age is also based on body weight but includes an initial loading dose followed by a subsequent maintenance dose administered every two or four weeks
Other indications are detailed in the product label

May 8, 2026 – efgartigimod alfa-fcab (Vyvgart) and efgartigimod alfa and hyaluronidase-qvfc (Vyvgart Hytrulo)

Argenx; neonatal Fc receptor blocker (efgartigimod alfa) and an endoglycosidase (hyaluronidase); approval marks the first FDA-approved treatment for all serotypes of adults living with generalized myasthenia gravis (gMG)
Expanded indication: treatment of gMG in adults who are antiacetylcholine receptor antibody (AChR-Ab) seronegative; based on this, the indication has been revised to remove the qualifier of adults who are anti-acetylcholine receptor (AChR) antibody positive; the indication now states for the treatment of gMG in adults
Administered as a weight-based dose administered by an HCP via IV infusion over one hour (Vyvgart) once weekly for four weeks with subsequent treatment cycles being based on clinical evaluation; Vyvgart Hyturlo prefilled syringe can be administered by patients/caregivers following proper training in SC injection technique (Vyvgart Hyturlo vial for HCP administration only); prefilled syringe is administered over 20 to 30 seconds in cycles of once weekly injections for four weeks with subsequent treatment cycles being based on clinical evaluation
Other indication: chronic inflammatory demyelinating polyneuropathy (CIDP) (Vyvgart Hytrulo only)

May 8, 2026 – ocrelizumab (Ocrevus)

Genentech; CD20-directed cytolytic antibody; second FDA-approved therapy for pediatric patients with relapsing-remitting multiple sclerosis (MS)
New indication: for the treatment of relapsing-remitting MS in pediatric patients ≥ 10 years of age who weigh ≥ 25 kg
Administered as an IV infusion by an HCP; dosage is based on if the patient is a pediatric patient or adult and body weight for pediatric patients (25 kg < 35 kg versus ≥ 35 kg); the initial dose is split into two equal infusions administered two weeks apart; subsequent doses are administered every six months; patients should be monitored closely during the infusion and for at least one hour after the infusion by experienced HCPs with access to medical support for management of severe reactions (e.g., serious infusion reactions); patients should receive premedication with methylprednisolone (or an equivalent) and an antihistamine (e.g., diphenhydramine) before each infusion
Other indications are detailed in the product label

May 8, 2026 – zenocutuzumab-zbco (Bizengri)

Partner;  bispecific HER2- and HER3-directed antibody; Assessment Aid, Breakthrough Therapy, CNPV pilot, Orphan Drug, Priority Review
New indication: for the treatment of adults with advanced unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy; patients should be selected for treatment based on the presence of an NRG1 gene fusion
Administered as an IV infusion by an HCP over four hours; administer every two weeks until disease progression or unacceptable toxicity; premedications should be administered before each infusion to decrease the risk for infusion-related reactions; patients should be closely monitored for signs and symptoms of infusion-related reactions during the infusion, for at least one hour following completion of the first infusion and as clinically indicated
Other indications are detailed in the product label

May 13, 2026 – benralizumab (Fasenra)

AstraZeneca; interleukin-5 receptor alpha-directed cytolytic monoclonal antibody
New indication: treatment of adult and pediatric patients ≥ 12 years of age with hypereosinophilic syndrome (HES) without an identifiable nonhematologic secondary cause
Administered SC once every four weeks; Fasenra pen can be used for administration by a patient/caregiver following proper training
Other indications are detailed in the product label

May 13, 2026 – decitabine and cedazuridine (Inqovi)

Taiho Oncology; combination of a nucleoside metabolic inhibitor (decitabine) and a cytidine deaminase inhibitor (cedazuridine); Orphan Drug, Project Orbis; approval marks the first all-oral combination treatment for patients with acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy
New indication: in combination with venetoclax for the treatment of newly diagnosed AML in adults ≥ 75 years of age, or who have comorbidities that preclude use of intensive induction chemotherapy
Administered orally, on an empty stomach, once daily on day 1 through day 5 of each 28-day cycle
Other indication: for treatment of adults with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate1, intermediate-2, and high-risk International Prognostic Scoring System groups

Traditional

April 20, 2026 – doravirine and islatravir (Idvynso)

NDA approval; only FDA-approved two-drug, non-integrase strand transfer inhibitor (INSTI), tenofovir-free regimen
Combination of a HIV-1 nonnucleoside reverse transcriptase inhibitor (NNRTI) (doravirine) and a nucleoside analog reverse transcriptase inhibitor (NRTI) (islatravir)
Indicated as a complete regimen for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in those who are virologically-suppressed (HIV-1 RNA < 50 copies/mL) on a stable antiretroviral regimen with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine
Oral tablet: 100 mg doravirine and 0.25 mg islatravir
Recommended dosage is taken orally once daily with or without food in adults
Approval was based on data from two randomized, active-controlled, noninferiority studies (trial 051; n=551 and trial 052; n=513) conducted in virologically-suppressed patients with HIV-1; patients in these studies were randomized 2:1 to either switch to once daily doravirine/islatravir or remain on their current baseline therapy (trial 051: antiretroviral therapy (ART); trial 052: bictegravir/emtricitabine/tenofovir alafenamide [Biktarvy]); in trial 051 at week 48, 1.4% of patients treated with doravirine/islatravir had a viral load of ≥ 50 copies/mL of HIV-1 RNA compared with 4.9% of patients who continued on their baseline ART regimen (difference, -3.6%; multiplicity-adjusted 95% CI, -7.8 to -0.8); in trial 052 at week 48, 1.5% of patients in the doravirine/islatravir group and 0.6% of patients in the Biktarvy group had HIV-1 RNA viral load of ≥ 50 copies/mL (difference, 0.9%; multiplicity-adjusted 95% CI, -1.9% to 2.9%)
Idvynso provides a non-INSTI, complete two-drug regimen that is taken once daily and does not contain tenofovir; it has demonstrated non-inferiority to standard oral ART including the triple-drug regimen Biktarvy in virologically-suppressed adults with HIV
Idvynso is available from Merck Sharp & Dohme

None

April 27, 2026 – budesonide and formoterol fumarate (Symbicort Aerosphere)

AstraZeneca; combination of a corticosteroid (budesonide) and a long-acting beta2-adrenergic agonist (LABA) (formoterol fumarate); FDA also approved a new strength of the metered dose inhaler: 80 mcg/4.8 mcg (budesonide 80 mcg and formoterol fumarate 4.8 mcg) per actuation
New indication: the treatment of asthma in adult and pediatric patients ≥ 12 years of age; not indicated for relief of acute bronchospasm
Administered as two actuations twice daily via oral inhalation
Other indication: the maintenance treatment of chronic obstructive pulmonary disease (COPD) in adults

April 27, 2026 – budesonide, glycopyrrolate, and formoterol fumarate (Breztri Aerosphere)

AstraZeneca; combination of an inhaled corticosteroid (budesonide), an anticholinergic (glycopyrrolate) and a LABA (formoterol fumarate); FDA also approved a new strength of the metered dose inhaler: 160 mcg/18 mcg/4.8 mcg (budesonide 160 mcg, glycopyrrolate 18 mcg and formoterol fumarate 4.8 mcg) per actuation
New indication: the maintenance treatment of asthma in adult and pediatric patients ≥ 12 years of age; not indicated for the relief of acute bronchospasm
Administered as two inhalations of the new strength metered dose inhaler twice daily administered by oral inhalation
Other indication: the maintenance treatment of COPD in adults

April 29, 2026 – rosuvastatin (Crestor)

AstraZeneca; HMG Co-A reductase inhibitor (statin); homozygous familial hypercholesterolemia (HoFH) indication revised to remove wording for use as an adjunct to other LDL-C lowering therapies or alone if such treatments are unavailable and instead state as an adjunct to diet and exercise to reduce LDL-C in patients ≥ 7 years of age with HoFH; other LDL-C reduction indications revised to state as an adjunct to diet and exercise rather than diet only
Expanded indication: cardiovascular (CV) risk reduction indication updated to remove wording specifying for adults without established coronary heart disease who are at increased risk of CV disease based on age, high-sensitivity C-reactive protein (hsCRP) ≥ 2 mg/L, and at least one additional CV risk factor; the indication now reads to reduce the risk of major adverse CV events (CV death, nonfatal myocardial infarction, nonfatal stroke, or an arterial revascularization procedure) in adults at increased risk for CV events
Administered orally once daily; dosage is dependent on a patient’s indication for usage, LDL-C and individual risk for CV events
Other indications are detailed in the product label

April 30, 2026 – dextromethorphan hydrobromide/bupropion hydrochloride (Auvelity)

Axsome; combination of an uncompetitive N-methyl D-aspartate (NMDA) receptor antagonist and sigma-1 receptor agonist (dextromethorphan) and an aminoketone and cytochrome (CYP) 450 2D6 inhibitor (bupropion); approval marks the first non-antipsychotic FDA-approved treatment for this use; Breakthrough Therapy, Priority Review
New indication: for treatment of agitation associated with dementia due to Alzheimer's disease in adults
Administered as extended-release oral tablets; initiated at the lowest tablet strength taken once daily in the morning; on day eight, based on tolerability, increase to twice daily; on day 15, based on tolerability, increase to the highest tablet strength taken twice daily (maximum recommended dosage); no more than two doses should be taken within the same day
Other indication: major depressive disorder in adults

First generic drug launches

March 24, 2026 – fluticasone propionate (Flovent HFA)

Glenmark launched generic fluticasone propionate inhalation aerosol (44 mcg per actuation) to GSK’s Flovent HFA
Corticosteroid; indicated for the maintenance treatment of asthma as prophylactic therapy in adult and pediatric patients ≥ 4 years of age
Recommended dosage is based on prior asthma therapy and disease severity; taken via oral inhalation twice daily
Annual sales for Flovent in 2025 were < $10 million

April 6, 2026 – dapagliflozin propanediol (Farxiga)

Multiple manufacturers launched generic dapagliflozin oral tablets (5 mg, 10 mg) to AstraZeneca’s Farxiga
Sodium-glucose cotransporter 2 (SGLT2) inhibitor; indicated (1) to reduce the risk of hospitalization for heart failure in adults with type 2 diabetes mellitus (T2DM) and either established CV disease or multiple CV risk factors and (2) as an adjunct to diet and exercise to improve glycemic control in adults with T2DM
Recommended dosage is taken orally once daily
Annual sales for Farxiga in 2025 were $11,152 million

April 6, 2026 – dapagliflozin propanediol and saxagliptin hydrochloride (HCl) (Qtern)

MSN/Novadoz launched generic dapagliflozin/saxagliptin oral tablets (10 mg/5 mg) to AstraZeneca’s Qtern
Combination of a SGLT2 inhibitor (dapagliflozin) and a dipeptidyl peptidase-4 (DPP-4) inhibitor (saxagliptin HCl); indicated as an adjunct to diet and exercise to improve glycemic control in adults with T2DM
Recommended dosage is orally once daily in the morning with or without food
Annual sales for Qtern in 2025 are < $10 million

April 8, 2026 – dapagliflozin and metformin HCl (Xigduo XR)

Multiple manufacturers launched generic oral dapagliflozin/metformin extended-release tablets (5 mg/500 mg, 5 mg/1,000 mg, 10 mg/500 mg, 10 mg/1,000 mg) to AstraZeneca’s Xigduo XR
Combination of a SGLT2 inhibitor (dapagliflozin) and a biguanide (metformin HCl); indicated as an adjunct to diet and exercise to improve glycemic control in adults with T2DM; dapagliflozin, when used as a component of dapagliflozin/metformin is indicated in adults with T2DM to reduce the risk of (1) sustained eGFR decline, end-stage kidney disease, CV death and hospitalization for heart failure in patients with chronic kidney disease at risk of progression, (2) CV death, hospitalization for heart failure and urgent heart failure visit in patients with heart failure and (3) hospitalization for heart failure in patients with T2DM and either established CV disease or multiple CV risk factors
Recommended dosage is taken orally once daily in the morning with food
Annual sales for Xigduo XR in 2025 were $576 million

AAD American Academy of Dermatology

AAV adeno-associated virus

ACP American College of Physicians

AD atopic dermatitis

AI artificial intelligence

AML acute myeloid leukemia

ANCA anti-neutrophil cytoplasmic autoantibody

AS ankylosing spondylitis

ASH American Society of Hematology

BCL-2 B-cell lymphoma 2

BLA Biologics License Application

BSA body surface area

BTK Bruton’s tyrosine kinase

C5aR complement 5a receptor

CAR chimeric antigen receptor

CD cluster of differentiation

CDER Center for Drug Evaluation and Research

CDK cyclin-dependent kinase

CI confidence interval

CMML chronic myelomonocytic leukemia

CMV cytomegalovirus

CNPV Commissioner’s National Priority Voucher

CSU chronic spontaneous urticaria

CV cardiovascular

COPD chronic obstructive pulmonary disease

DILI drug-induced liver injury

EBV Epstein-Barr virus

ER estrogen receptor

eGFR estimated glomerular filtration rate

FAQ frequently asked question

FDA Food and Drug Administration

FSGS focal segmental glomerulosclerosis

GCP Google Cloud Platform

GLP-1 glucagon-like peptide-1

gMG generalized myasthenia gravis

GPA granulomatosis with polyangiitis

HALO Harmonized AI & Lifecycle Operations for Data

HbA1c hemoglobin A1c

HCP healthcare professional

HER2 human epidermal growth factor receptor 2

HES hypereosinophilic syndrome

HHS U.S. Department of Health and Human Services

HIV-1 human immunodeficiency virus-1

HMG Co-A hydroxymethylglutaryl-coenzyme A

HoFH homozygous familial hypercholesterolemia

hsCRP high-sensitivity C-reactive protein

ICER Institute for Clinical and Economic Review

IFN interferon

IgAN immunoglobulin A nephropathy

IND Investigational New Drug

INSTI integrase strand transfer inhibitor

ISTH International Society on Thrombosis and Haemostasis

LABA long-acting beta2-adrenergic agonist

LDL-C low-density lipoprotein cholesterol

MCL mantle cell lymphoma

MDS myelodysplastic syndromes

MPA microscopic polyangiitis

MS multiple sclerosis

NCCN National Comprehensive Cancer Network

NDA New Drug Application

NMDA N-methyl D-aspartate

NNRTI non-nucleoside reverse transcriptase inhibitor

NOOH notice of opportunity for a hearing

NRTI nucleoside analog reverse transcriptase inhibitor

PFS progression-free survival

PROTAC Proteolysis Targeting Chimera

PTA pure tone audiometry

PTSD post-traumatic stress disorder

RMAT Regenerative Medicine Advanced Therapy

RNA ribonucleic acid

RTCT real-time clinical trials

SLE systemic lupus erythematosus

SPM second primary malignancy

T1DM type 1 diabetes mellitus

T2DM type 2 diabetes mellitus

TBD to be determined

TLS tumor lysis syndrome

TRT testosterone replacement therapy

U.S. United States

VTE venous thromboembolism

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 health care provider.
All brand names are property of their respective owners.

About us

Corporate social responsibility

Solutions overview

Modern technology

Drug access

Clinical solutions

Condition management

Specialty drug management

Government solutions

Single-state programs

Read

Watch

Listen

Home Delivery

Specialty Pharmacy

Trending Topics & Drug Approvals: May 2026

Trending Topics

Hot topics

Drug Approvals

Specialty

Traditional

First generic drug launches

Login Portals

Compliance / Legal

Company