Quarterly Drug Pipeline: April 2026
Clinical insights and competitive intelligence on anticipated drugs in development
Editor-in-chief's message
Welcome to the Prime Quarterly Drug Pipeline! Dive into clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline.
Methodology
The drug pipeline is complex and fluid. Our talented and committed team of clinical and analytics experts are excited to bring you this robust publication after thoughtful research. Specialty and traditional drugs that are covered under the pharmacy and medical benefits are featured. New molecular entities, pertinent new and expanded indications for existing medications, biosimilars and regenerative medicines, such as gene and cellular therapies, are also profiled.
The Quarterly Drug Pipeline details both agents submitted for FDA review and those in Phase 3 studies with a likelihood to apply to the FDA. Our Deep Dives consider the evidence, the products’ potential to fill an unmet need or become the new standard of care and the ability to replace existing therapies.
A market agnostic financial forecast primarily from Evaluate is included for select agents to assist payers with assessing the potential budgetary impact of the pipeline. Five-year projected annual U.S. sales are forecasted for select agents.
Reflection
In the first quarter of 2026, the agency has approved 10 novel drugs which is 30% more that the number of approvals about the same time last year. New therapeutics in oncology, autoimmune disease, and mental health conditions are among some of this year’s novel approvals so far. Notably, there were several first-time approvals including a once-weekly basal insulin for type 2 diabetes, treatments for rare diseases such as Hunter syndrome, Menkes disease and cholestatic pruritus associated with primary biliary cholangitis, as well as a new gene therapy for an inherited immune deficiency. Wegovy HD – a high-dose version of injectable semaglutide – received approval for chronic weight management.
In 2026, two new molecular entities – an oral GLP-1 orforglipron (Foundayo) for weight loss and a first-time gene therapy lunsotogene parvec-cwha (Otarmeni) for a rare genetic hear loss – were approved under the FDA’s new Commissioner’s National Priority Voucher (CNPV) pilot. The FDA also issued national priority vouchers to three companies studying psychedelics for mental health conditions. This pilot program awards vouchers to products that align with critical national priorities.
While numbers do not tell the entire story, they represent innovation for patient care and have the potential to advance health for the American public.
On the horizon
The FDA decisions for specialty medications (76%) and for Orphan Drugs (37%) in the pipeline continue to grow for agents with applications submitted to the FDA. Eight agents submitted to the FDA are seeking FDA’s Accelerated Approval. Several approvals and additional voucher recipients are expected under the FDA’s CNPV pilot.
Second quarter 2026 may usher in notable approvals. Select highlights include:
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Two new therapeutics for breast cancer
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First treatment for hepatitis D virus
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An oral medication for resistant or uncontrolled hypertension
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New infusion for thyroid eye disease
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First-time biosimilar for Simponi for autoimmune conditions
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First new option for smoking cessation in over 20 years
We hope you enjoy the report!
Maryam Tabatabai
Associate Vice President, Clinical Information
Editorial team
Maryam Tabatabai, PharmD
Editor-In-ChiefAssociate Vice President, Clinical Information Data Scientist Principal
Carole Kerzic, RPh
Executive Editor
Drug Information Pharmacist Principal
Nicole Kjesbo, PharmD, BCPS
Executive Editor
Clinical Program Development Director Senior
Consultant panel
Samantha Decker, PharmD
Medical Pharmacy Clinical Pharmacist
Natalee Felten, PharmD, BCPS
Medical Pharmacy Clinical Pharmacist
Andrea Henry, PharmD, MBA, BCPS
Drug Information Pharmacist Principal
Danny Melson
Data Scientist Principal
Katie Owen, PharmD, BCOP
Clinical Program Pharmacist Principal
All brand names are property of their respective owners.
The drug pipeline is fluid; the dates and information within this publication are subject to change. Nothing herein is or shall be construed as a promise or representation regarding past or future events and Prime Therapeutics expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Prime Therapeutics.
Near-term pipeline
Deep dive
adrabetadex intrathecal
Proposed indications
Niemann-Pick disease type C1 (NPC1)
Clinical overview
Mechanism of action
Adrabetadex is a mixture of 2-hydroxypropyl-β-cyclodextrin isomers designed to increase intracellular cholesterol trafficking in Niemann-Pick disease type C (NPC).
Clinical trial(s)
An open-label, Phase 2b/3 trial evaluated adrabetadex in patients 4–21 years of age with NPC1. In parts A and B (NCT02534844; n=58), patients were randomized to receive adrabetadex or sham control for 52 weeks. Part C (NCT04958642; n=66) evaluated safety and tolerability during which all patients received adrabetadex (900 mg) treatment for up to 260 weeks or until the study was ended by the sponsor. Parts A (dose-finding) and B (900 mg) did not show a statistically significant difference between adrabetadex and sham in disease progression at 52 weeks as measured by the co-primary endpoints of the 4-item NPC severity scale (NPC-SS) assessing ambulation, cognition, fine motor, and swallowing and the clinician global impression change (CGIC). Given the lack of separation from sham on the primary endpoints in parts A and B, part C of the study was terminated early (2021). In part A and B of the study, more patients who received adrabetadex experienced serious adverse events compared to those who received sham treatment (52.6% versus 22.2%, respectively). Serious effects were similar to complications of NPC1 disease and included hearing loss, pneumonia, seizure, and difficulty swallowing. No deaths were reported. The part C safety evaluation reported hearing loss, balance problems, and tiredness with adrabetadex.
Neuronal biomarkers were evaluated in children with NPC1 in the Phase 2b/3 and open-label extension trials. CSF levels of 24(S)-hydroxycholesterol (OHC), an indicator of cholesterol trafficking, and calbindin D and fatty acid-binding protein 3 (FABP3), both of which are associated with neurodegeneration, were measured. From baseline to week 52, among adrabetadex-treated children, CSF 24(S)-OHC level increased by a median of 27.7% (p=0.0012) and CSF calbindin D and FABP3 levels decreased by a median of 18.3% (p=0.0117) and median of 40.5% (p=0.0079), respectively.
In addition, a survival analysis compared adrabetadex treatment in individuals (n=72) with infantile neurological onset NPC who participated in the Phase 2b/3 trial and open-label extension period and/or in an Expanded Access Program (EAP) to 119 matched external controls from the NIH Natural History study and three other major disease databases or publications. The analysis revealed a 5-year survival rate of 84% among patients treated with adrabetadex compared to 42% for external controls, translating to a 71% relative reduction in mortality risk (hazard ratio [HR], 0.289; p<0.0001) with adrabetadex. More favorable survival was reported with adrabetadex compared to control across the analyzed subgroups including: early infantile-onset NPC (age at onset < 2 years) (HR, 0.148; p<0.0001), late infantile-onset NPC (age at onset 2 to < 6 years) (HR, 0.344; p=0.457), with miglustat (HR, 0.361; p=0.0205) and without miglustat (HR, 0.139; p<0.0001).
In March 2026, an analysis of data from individuals with infantile-onset NPC in clinical trials and EAP (n=79) reported a significantly reduced annual rate of disease progression of 43% compared with pre-treatment rates of disease progression (4-item NPC-SS, 1.7 point decrease after 3 years of treatment; p=0.0055) among patients with infantile-onset NPC who were treated with adrabetadex (n=79).
In the clinical trial, adrabetadex was administered intrathecally every two weeks at doses titrated to 900 mg, 1,200 mg, or 1,800 mg in part A. A dose of 900 mg was used in parts B and C.
Place in therapy
NPC is an ultra-rare, inherited, lysosomal storage disorder. It is estimated to affect one in 100,000–150,000 individuals, with the disease occurring more frequently in people of French-Acadian descent. It is caused by mutations in the NPC1 gene (95% of cases) or NPC2 gene (5% of cases) that result in dysfunctional NPC proteins giving rise to abnormal accumulation of lipids in tissues and organs, including the brain. Clinical features include hepatosplenomegaly and progressive neurologic impairment affecting motor function, cognition, swallowing, and speech. Disease presentation and rate of progression depend on the age of onset of neurological symptoms, with earlier onset associated with more aggressive decline. Mean survival of 5.6 years is estimated for early-infantile-onset (onset < 2 years of age) and approximately 13.4 years for late-infantile-onset (onset 2 to < 6 years of age).
There is no cure for NPC. Miglustat has been used off-label for NPC in the United States and works by inhibiting glycolipid synthesis, with slowing of disease progression demonstrated in clinical trials. Its use may be considered in select patients with mild to moderate neurologic, psychiatric, or cognitive manifestations, ideally starting use at the time of onset of neurologic manifestations. FDA-approved agents for NPC are arimoclomol (Miplyffa), which prevents protein misfolding and maintains lysosomal stability and is indicated for use in combination with miglustat, and levacetylleucine (Aqneursa), a neuroprotective agent that reduces lysosomal dysfunction and neuroinflammation.
Adrabetadex may address the underlying pathology of NPC by restoring intracellular neuronal cholesterol trafficking. Although initial analysis of the Phase 2b/3 trial in patients 4–21 years of age with NPC did not demonstrate improvement in clinical disease activity with adrabetadex, additional data showed a significant reduction in the annual rate of disease progression, improvement in key NPC biomarkers consistent with increased cholesterol trafficking and reduced neuronal injury. Moreover, adrabetadex improved survival among children with infantile-onset NPC1 compared with matched external controls. If approved, adrabetadex may offer a treatment option that directly targets the underlying impairment in NPC.
FDA approval timeline
Aug, 17, 2026
FDA designations: Breakthrough Therapy, Orphan Drug, Priority Review, Rare Pediatric Disease
Financial forecast (reported in millions)
The financial forecast for adrabetadex is not currently available.
anitocabtagene autoleucel IV
Proposed indications
Relapsed or refractory multiple myeloma (RRMM) as fourth-line treatment
Clinical overview
Mechanism of actionAnitocabtagene autoleucel (anito-cel) is an autologous anti–B-cell maturation antigen (BCMA) CAR T-cell therapy with a D-domain binder. It attaches to BCMA on the surface of multiple myeloma cells, leading to CAR T-cell proliferation, cytokine secretion, and subsequent cytolytic destruction of BCMA-expressing tumor cells.
The ongoing, open-label, Phase 2 iMMagine-1 trial (NCT05396885) evaluated anito-cel in 117 adults with RRMM who had received at least three prior systemic treatments, including a proteasome inhibitor, immunomodulatory drugs, and an anti-CD38 antibody. Patients were excluded if they received prior BCMA directed therapy. At a median follow-up of 15.9 months, the ORR was 96%, with a complete response or stringent complete response (CR/sCR) rate of 74%. The median time to best response was 4.8 months. Among patients evaluable for MRD testing (n=96), 95% achieved MRD negativity (≤10⁻⁵ sensitivity) at a median time of one month. At 12, 18, and 24 months, PFS rates were 82.1%, 67.4%, and 61.7%, and OS rates were 94%, 88%, and 83%, respectively. The most common grade 3/4 TEAEs were neutropenia (66%), anemia (24%), thrombocytopenia (24%), and infection (9%). CRS and ICANS of any grade were reported in 85% and 8% of patients, respectively. Delayed immune effector cell-associated enterocolitis and non-ICANS neurotoxicity, such as Parkinsonism, cranial nerve palsies, and Guillain-Barré syndrome, were not observed.
Dosage and administration
In the IMMagine-1 trial, patients received a single dose of anito-cel 115×10⁶ CAR T-cells via IV infusion. Lymphodepleting chemotherapy was required in the days prior to the anito-cel infusion.
Place in therapy
Multiple myeloma (MM) is a malignant neoplasm of plasma cells that accumulate in bone marrow, leading to destruction and failure of the bone marrow. It is estimated that 36,000 new cases of MM and 10,850 deaths due to MM will occur in the United States in 2026. Patients are typically diagnosed with MM between the ages of 65–74 years. The 5-year relative survival rate is 81% for localized disease and 62% for distant disease.
Quadruplet therapy with an anti-CD38 monoclonal antibody (e.g., daratumumab [Darzelex], isatuximab [Sarclisa]), a proteasome inhibitor (e.g., bortezomib [Velcade]) an immunomodulating agent (e.g., lenalidomide [Revlimid]), and dexamethasone is the preferred primary treatment of newly diagnosed symptomatic MM in patients who are HSCT eligible. Primary therapy is followed by high-dose therapy and autologous HSCT. BCMA-directed CAR T-cell therapy with ciltacabtagene autoleucel (Carvykti) or idecabtagene vicleucel (Abecma) is recommended for relapsed or refractory disease after one to three prior therapies.
If approved, anito-cel would be the third BCMA-directed CAR T-cell indicated for RRMM. Unlike Carviykti and Abecma, which are indicated for second- or third-line use, anito-cel was submitted as a fourth-line therapy. Anito-cel utilizes a compact D-domain binder to target BCMA on the tumor cells rather than the single-chain binders used in other CAR T-cell products. The D-domain binder improves binding efficiency, allowing effective treatment with fewer CAR T cells and potentially offering a better safety profile compared to other CAR T products, although its full clinical benefit is not yet known. Anito-cel has not been compared to these products in head-to-head trials.
The Phase 3 iMMagine-3 trial is currently recruiting to compare anito-cel to SOC regimens in patients with RRMM who have received one to three prior lines of therapy, including an anti-CD38 antibody and an immunomodulatory drug. FDA filing is expected as early as 2027.
Additionally, in June 2025, the FDA removed the REMS requirements for CAR T-cell therapy, eliminating the need for certified treatment centers and reducing post-dose monitoring and facility‑proximity requirements. Approximately eight patients in the iMMagine-1 trial received anito-cel in an outpatient setting.
FDA approval timeline
FDA designations: Fast Track, Orphan Drug, Regenerative Medicine Advanced Therapy
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $50 |
| 2027 | $224 |
| 2028 | $365 |
| 2029 | $722 |
| 2030 | $1,023 |
atacicept SC
Proposed indications
Immunoglobulin A nephropathy (IgAN) (Berger's Disease)
Clinical overview
Mechanism of actionAtacicept is a recombinant Fc fusion protein that contains a transmembrane activator and calcium-modulating cyclophilin ligand interactor (TACI) receptor, which binds to and inhibits two key cytokines―B-cell activating factor (BAFF) and a proliferation-inducing ligand (APRIL)―that play a key role in IgAN pathogenesis.
Clinical trial(s)
The ongoing Phase 3 ORIGIN-3 trial (NCT04716231) is a randomized, double-blind, placebo-controlled trial evaluating atacicept in adults with IgAN. Eligible patients had a total urine protein excretion ≥ 1 g/24 hours or urine protein-to-creatinine ratio (UPCR) ≥ 1 g/g, an eGFR ≥ 30 mL/min/1.73 m² and were on a stable regimen of a RASi (e.g., ACEI, ARB). Atacicept was added to SOC therapy. Interim data (n=203) showed that atacicept led to a significantly greater reduction in the primary endpoint of percentage reduction from baseline on 24-hour UPCR compared to placebo at 36 weeks (45.7% versus 6.8%; difference, 41.8%; p<0.001). Atacicept also led to significant improvements compared to placebo in key secondary outcomes, including reductions in serum galactose-deficient IgA1 (Gd-IgA1) (-68% versus -3%; p<0.0001), which is a key biomarker of IgAN, and higher rates of hematuria resolution (81% versus 21%; p<0.0001). Data regarding impact on eGFR are anticipated in 2027 and estimated study completion is in 2028. Interim data show that atacicept was generally well tolerated, with infection rates similar to placebo (32% versus 28%, respectively). The detection of ADA with atacicept have not been reported.
Eligible adults with IgAN who received atacicept (25 mg, 75 mg or 150 mg) or placebo in the 36-week Phase 2b, blinded ORIGIN trial were enrolled in the open-label ORIGIN-EXTEND (NCT06674577) extension study, where they received atacicept 150 mg for an additional 60 weeks. Interim results showed that after 96 weeks of continuous atacicept therapy, UPCR decreased by 52%, Gd-IgA1 decreased by 66%, hematuria resolved in 75% of patients, and eGFR remained stable with a mean annualized slope of -0.6 mL/min/yr. Atacicept was well tolerated over the 96-week period.
Dosage and administration
In the ORIGIN-3 trial, atacicept 150 mg was self-administered SC once weekly.
FDA approval timeline
July 7, 2026
FDA designations: Breakthrough Therapy, Orphan Drug, Priority Review, seeking Accelerated Approval
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $32 |
| 2027 | $224 |
| 2028 | $486 |
| 2029 | $818 |
| 2030 | $944 |
povetacicept SC
Proposed indications
Immunoglobulin A nephropathy (IgAN) (Berger's Disease)
Clinical overview
Mechanism of actionPovetacicept is an Fc fusion protein of an engineered TACI domain that binds to and inhibits the BAFF and APRIL cytokines.
Clinical trial(s)
The ongoing, double-blind, placebo-controlled, Phase 3 RAINIER trial (NCT06564142) is evaluating povetacicept in 605 adults with IgAN. Enrolled patients had a 24-hour proteinuria excretion ≥1 g/day or 24-hour UPCR ≥ 0.75 g/g, an eGFR ≥ 30 mL/min/1.73m² and were on a stable regimen of a RASi (e.g., ACEI, ARB). Povetacicept was added to SOC therapy. Interim analysis (n=199) showed that povetacicept led to statistically significant 49.8% reduction in UPCR, the primary endpoint, compared to placebo (p<0.0001) at week 36. Povetacicept also led to significant improvements compared to placebo in key secondary outcomes, including reductions in serum Gd-IgA1 (-77.4% versus +9.1%; p<0.0001) and higher rates of hematuria resolution (85.1% versus 23.4%; p<0.0001). Povetacicept was generally safe and well tolerated. No opportunistic infection or discontinuation due to infection were reported. Anti-drug antibodies (ADAs) were observed; but had no impact on efficacy or safety of povetacicept.The RAINIER trial is anticipated to complete in early 2028.
Dosage and administration
In the RAINIER trial, povetacicept 80 mg was self-administered SC once every four weeks.
FDA approval timeline
4Q 2026
FDA designations: Breakthrough Therapy, Priority Review, seeking Accelerated Approval
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $0 |
| 2027 | $127 |
| 2028 | $352 |
| 2029 | $854 |
| 2030 | $1,206 |
IgAN, also known as Berger’s disease, is an autoimmune disorder of B-cell regulation. It is characterized by the accumulation of abnormal antibody immunoglobulin A (IgA) immune complexes in the kidneys, leading to inflammation and glomeruli damage. Approximately 50% of patients will progress to ESRD within 10–20 years of diagnosis. IgAN can occur at any age, but onset is typically between 10–40 years. It occurs more often in men as well as in individuals of Asian and Caucasian descent.
Proteinuria is an established modifiable predictor of IgAN progression, and emerging evidence shows that even lower levels of proteinuria than previously reported are associated with a substantial lifetime risk of kidney failure. Accordingly, the 2025 KDIGO guidelines on IgAN management (published October 2025) lowered the proteinuria goal from < 1 g/day to < 0.5 g/day, and ideally < 0.3 g/day. KDIGO endorses two simultaneous therapeutic strategies for IgAN:
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Use of systemic glucocorticoids, including delayed-release oral budesonide (Tarpeyo), to reduce the production of IgA-containing immune complex (IgA-IC) that drives glomerular injury.
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Managing the consequences of existing IgAN-induced nephron loss by controlling blood pressure and reducing glomerular hyperfiltration and proteinuria using a RASi or the dual endothelin-angiotensin receptor antagonist sparsentan (Filspari), with or without an SGLT2 inhibitor.
Of note, the endothelin receptor antagonist atrasentan (Vanrafia; approved 2025) and the complement inhibitor iptacopan (Fabhalta; approved 2024 for IgAN) were not covered in the 2025 KDIGO guidelines, although both agents are FDA-approved to reduce proteinuria in adults with IgAN who are at risk of rapid disease progression (UPCR ≥ 1.5 g/g).
B-cell targeted therapy is emerging as a new treatment approach for IgAN and has the potential to be used earlier in the treatment algorithm than traditional systemic corticosteroids since these agents target upstream pathogenic processes rather than broadly suppressing immunity. In November 2025, the first B-cell modulator─the anti-APRIL monoclonal antibody sibeprenlimab (Voyxact)―received Accelerated Approval for adults with IgAN who are at risk for disease progression. If approved, atacicept would be the first therapy, followed by povetacicept, to inhibit both APRIL and BAFF cytokines, although the clinical relevance of dual APRIL-BAFF blockage remains uncertain. In non-comparative trials, atacicept, povetacicept and Voyxact significantly reduced 24-hour UPCR versus placebo at nine months (41.8%, 49.8% and 51.2%, respectively). In addition, all three products are self-administered by SC injection, with atacicept delivered as a 1 mL once-weekly injection, povetacicept as a 0.5 mL injection every four weeks and Voyxact as a 2 mL injection every four weeks. Anti-drug antibodies (ADA) can form in patients treated with biologic medications. In the VISIONARY trial, 34% of evaluable patients treated with Voyxact developed ADA, of whom 23.9% developed ADA with neutralizing activity. ADA led to approximately 40% lower drug exposure and a numerically lower 24-hour UPCR at nine months (24-hour UPCR, 41.6% with ADA versus 52.7% without ADA). The clinical significance of the differences in UPCR reduction based on the presence or absence of ADA is unclear. ADA were observed with povetacicept but did not impact its efficacy or safety. The detection of ADA with atacicept have not been reported.
denecimig (Mim8) SC
Proposed indications
Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A, with or without inhibitors
Clinical overview
Mechanism of action
Denecimig is a factor VIIIa (FVIIIa) mimetic bispecific antibody that mimics the role of FVIIIa by bridging factor IXa and factor X.
Clinical trial(s)
The open-label Phase 3 FRONTIER2 (NCT05053139) trial evaluated prophylaxis with denecimig in 254 patients ages ≥ 12 years with hemophilia A, with or without inhibitors. In the main phase, patients receiving on-demand therapy treatment before the study were randomized 1:1:1 to continue on-demand treatment (arm 1), or switch to denecimig once-weekly (arm 2a) or once-monthly (arm 2b) for 26 weeks. Patients on prior clotting factor concentrate (CFC) prophylaxis before the study were randomly (1:1) switched to denecimig once-weekly (arm 3) or once-monthly (arm 4) for 26 weeks. After completing the main phase, all patients entered a 26-week extension phase in which they received denecimig once weekly or once monthly. In the main phase, all patients who continued on-demand therapy experienced treated bleeds, with an estimated mean annualized bleeding rate (ABR) of 16.09. Among patients who were switched from on-demand to denecimig once-weekly or once-monthly treatment, the estimated ABRs were 0.43 and 0.25, respectively; 86% and 91% of patients had zero treated bleeds during the main phase. For participants previously on CFC prophylaxis, switching to once-- weekly or once-monthly denecimig resulted in estimated mean ABRs of 2.32 and 1.79, respectively, with 67% and 69% of patients experiencing zero treated bleeds. During the extension phase, the estimated mean ABR was 0.67 for patients who received once-weekly denecimig and 0.79 for those receiving monthly dosing. Notably, 88% of patients who received weekly doses and 70% who received monthly doses had zero treated bleeds. Denecimig was well tolerated. Injection site reactions (ISR) occurred in 12% of patients receiving weekly dosing and 9% of those on monthly dosing.
The two-part, non-randomized, open-label, Phase 3 FRONTIER3 (NCT05306418) trial evaluated denecimig prophylaxis in 70 children 1–11 years old with severe hemophilia A, with or without inhibitors. In part one, all patients received denicimig once weekly for 26 weeks. Interim results showed an estimated mean ABR of 0.53 with once-weekly denecimig and 74.3% of children had zero treated bleeds through week 26. In part two, patients could either continue denecimig once weekly or switch to denecimig once monthly for an additional 26 weeks; results have not been reported for part two.
The open-label, Phase 3b, FRONTIER5 (NCT05878938) trial assessed the safety of directly switching from emicizumab-kxwh (Hemlibra) prophylaxis to denecimig prophylaxis once-weekly, once every two weeks or once-monthly using a prefilled pen-injector in 61 patients ≥ 12 years of age with hemophilia A, with or without inhibitors. The study showed that patients could safely switch from Hemlibra to denecimig without a washout period. No thromboembolic events, hypersensitivity reactions, or TEAEs leading to discontinuation were observed. Most patients (97%) preferred the denecimig pen-injector over their previous Hemlibra administration method. Injection site reactions were reported by 19.7% of patients who received denecimig (1.5% of injections) and were mild in severity. No exaggerated thrombin peak height response was observed.
Dosage and administration
In the clinical trials, denecimig was administered SC as weekly or monthly doses using a prefilled, single-use pen.
Place in therapy
Hemophilia A is a congenital X-linked bleeding disorder that affects one in 5,500 male births in the United States. It is characterized by a deficiency of coagulation factor VIII, which leads to recurrent spontaneous bleeding into muscles and joints that can ultimately result in debilitating arthropathy.
The primary treatment for severe hemophilia is factor replacement therapy using plasma-derived or recombinant clotting factor concentrates. However, approximately 35% of patients with hemophilia A develop neutralizing antibodies, or inhibitors, against these factor products, making treatment more challenging. Bypassing agents (BPAs) provide alternative clotting factors that circumvent the factor inhibitor, enabling more normal clot formation. BPAs, such as recombinant activated factor VIIa (e.g., Novoseven RT, Sevenfact) and activated prothrombin complex concentrates (e.g., Feiba), are the SOC for reducing bleeding and long-term complications in patients with inhibitors. Hemostatic efficacy rates of about 80% have been reported with BPAs but may vary and change over time.
Non-factor products have improved hemophilia management, including in patients with factor inhibitors, and can reduce treatment burden and improve QOL. Available options include the bispecific FIXa/FX antibody emicizumab-kxwh (Hemlibra), which mimics FVIIIa cofactor. Hemlibra is indicated for routine prophylaxis for patients of all ages with hemophilia A, with or without inhibitors, and is self-administered SC every one, two or four weeks. In addition, two newer agents – the anti-tissue factor pathway inhibitor marstacimab-hncq (Hympavzi) and the antithrombin-directed small interfering RNA agent fitusiran (Qfitlia) – are approved for routine prophylaxis to reduce the frequency of bleeding episodes in patients ages ≥ 12 years with hemophilia A or B without inhibitors. Both products can be self-administered via SC injection using an autoinjector, with Hympavzi given once weekly and Qfitlia administered every two months.
If approved, denecimig would be the first FVIIIa mimetic offering flexible dosing―once weekly or once monthly― delivered via a prefilled single-use pen, for individuals living with hemophilia A, with or without inhibitors.
FDA approval timeline
September 2026
FDA designations: Orphan Drug
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $0 |
| 2027 | $159 |
| 2028 | $286 |
| 2029 | $412 |
| 2030 | $507 |
gedatolisib IV
Proposed indications
Hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-), PIK3CA (phosphatidylinositol-3-kinase catalytic subunit alpha) wild-type advanced breast cancer (ABC)
Clinical overview
Mechanism of actionGedatolisib is a multi-target PI3K/AKT/mTOR (PAM) inhibitor that targets all four Class I PI3K isoforms and mTOR to achieve broad inhibition of the PAM pathway.
Clinical trial(s)
The open-label, Phase 3 VIKTORIA-1 trial (NCT05501886) evaluated gedatolisib-based therapy in adults with HR+/HER2- ABC following progression on or after CDK4/6 and aromatase inhibitor therapy. A total of 392 patients who do not have confirmed PI3KCA mutations, also known as PI3KCA wild-type, were randomized to receive gedatolisib + palbociclib + fulvestrant (gedatolisib triplet), gedatolisib + fulvestrant (gedatolisib doublet), or fulvestrant alone. Patients assigned fulvestrant monotherapy could switch to gedatolisib-triplet or -doublet therapy if disease progression was detected. At a median follow-up of 10.1 months, the primary endpoint of median PFS was two months with fulvestrant monotherapy compared to 9.3 months with gedatolisib-triplet therapy (hazard ratio [HR], 0.24 versus fulvestrant; p<0.001) and 7.4 months with gedatolisib-doublet therapy (HR, 0.33 versus fulvestrant; p<0.001). In addition, the ORR was 31.5% with gedatolisib-triplet, including one complete response, 28.3% with gedatolisib-doublet, and 1% with fulvestrant monotherapy. The median OS had not been reached in the gedatolisib groups and was 18.5 months with fulvestrant monotherapy (HR, 0.74). The median DOR was 17.5 and 12 months with gedatolisib triplet and doublet therapies, respectively, and could not be calculated with fulvestrant monotherapy. The most common TEAEs (> 20%) with gedatolisib-triple and -doublet therapy were stomatitis (69.2%, 59.6%), neutropenia (65.4%, 1.5%), nausea (43.8%, 43.1%%), rash (27.7%, 32.3%), vomiting (27.7%, 23.1%), and fatigue (22.3%, 20.8%). Hyperglycemia was reported in 9.2% and 12.3% of patients in the gedatolisib-triplet and -doublet groups, respectively, with grade 3 events reported in 2.3% of patients in each group.
Dosage and administration
In the VIKTORIA-1 trial, gedatolisib 180 mg was administered IV once weekly for 3 weeks (on days 1, 8 and 15) followed by one week off
Place in therapy
Breast cancer is the most frequently diagnosed cancer in women in the United States and is the second leading cause of cancer-related deaths, following lung cancer. It is estimated that 321,910 new cases of invasive breast cancer will be diagnosed in women in the United States in 2026, and about 42,140 female deaths will occur due to the disease.
The recommended first-line treatment for HR+/HER2- ABC is a combination of an endocrine agent (aromatase inhibitor) and a CDK4/6 inhibitor (e.g., ribociclib [Kisqali], abemaciclib [Verzenio], palbociclib [Ibrance]). However, resistance typically develops. After this first-line therapy, patients may be treated with single agent fulvestrant, endocrine therapy plus everolimus, or a targeted therapy if a corresponding mutation is found.
Resistance to endocrine therapy plus a CDK4/6 inhibitor likely involves the PAM pathway that plays a role in tumor cell proliferation. Unlike PI3K- or AKT-directed therapies, such as alpelisib (Piqray), inavolisib (Itovebi), and capivasertib (Truqap), that are approved in patients with PIK3CA or PTEN mutations, gedatolisib is being developed for patients with and without PIK3CA mutations. Gedatolisib targets all four Class I PI3K isoforms, including PIK3CA, as well as mTOR, to provide broad inhibition of the PI3K pathway in PIK3CA wild-type disease and may help overcome resistance to both CDK4/6 inhibitors and endocrine therapy. The VIKTORIA-1 trial demonstrated significant improvement in PFS when gedatolisib (with or without palbociclib) was added to fulvestrant therapy in patients with HR+/HER2- ABC. If approved, gedatolisib has the potential to be a preferred option in second-line treatment of HR+/HER2- PIK3CA wild-type ABC. It will target the PAM pathway more broadly than current inhibitors and will be the first to be used without requiring a documented mutation in this pathway. Notably, the incidence of hyperglycemia was relatively low with gedatolisib plus fulvestrant compared to other agents, such as Piqray, which reported hyperglycemia in 63.7% of patients in the SOLAR-1 trial who received Piqray plus fulvestrant. The VIKTORIA-1 trial also studies the use of gedatolisib in patients with confirmed PIK3CA mutations, with topline data for this phase (Study 2) expected in the first half of 2026.
FDA approval timeline
July 17, 2026
FDA designations: Breakthrough Therapy, Fast Track, Priority Review, Real-Time Oncology Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $50 |
| 2027 | $379 |
| 2028 | $687 |
| 2029 | $968 |
| 2030 | $1,217 |
molgramostim inhaled
Manufacturer: Savara
Proposed indications
Autoimmune pulmonary alveolar proteinosis (aPAP)
Clinical overview
Mechanism of action
Molgramostim is a non-glycosylated form of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF). It activates macrophages in the lung alveoli restoring their ability to clear surfactant.
Clinical trial(s)
The Phase 3, double-blind, placebo-controlled IMPALA-2 trial (NCT04544293) evaluated molgramostim in 164 adults with aPAP. Throughout the study, pulmonary gas transfer was measured by diffusing capacity for carbon monoxide adjusted for hemoglobin concentration (DLCO%). At baseline, median DLCO% was 55%. The study reported a significantly greater LSM change in DLCO% from baseline at week 24 (primary endpoint) with molgramostim compared to placebo (LSM difference, 6 percentage points; p=0.0001). The magnitude of response was consistent across DLco% severity subgroups (DLCO%, ≤ 50% and > 50%) and was maintained at 48 weeks (LSM difference, 6.9 percentage points; p=0.0008). In addition, molgramostim produced significant improvements in the respiratory health-related QOL, as measured by St. Georges Respiratory Questionnaire-Total (SGRQ-T), in the overall population at week 24 (LSM differences: -6.6; p=0.0072), but significance was not maintained at week 48 (LSM differences: SGRQ-T, -4.9; p=0.1046). Notably, the magnitude of molgramostim’s effect on SGRQ-T was greater at 48 weeks in patients with a DLco% of > 50% than in the overall population. As an exploratory assessment, molgramostim reduced surfactant burden as measured by Ground-glass opacity (GGO) score on CT scans at week 24 (-2.1 versus -1.1 with placebo). In addition, fewer patients required whole lung lavage (WLL) as rescue therapy over the 48-week period with molgramostim compared to placebo (7.4% versus 13.3%, respectively). Molgramostim was generally well tolerated and 98% of patients completed the double-blind treatment period and enrolled in the open-label period.
Dosage and administration
In the IMPALA-2 trial, molgramostim 300 µg solution was administered via nebulizer once daily.
Place in therapy
aPAP is a rare, chronic lung disease characterized by the abnormal accumulation of surfactant within the alveoli. Surfactant is a naturally occurring substance in the alveoli that prevents the lungs from collapsing. Under normal conditions, GM-CSFs stimulate alveolar macrophages to clear excess surfactant. In aPAP, however, autoantibodies neutralize GM-CSF, impairing surfactant clearance and leading to respiratory distress, hypoxemia and increased risk of infection. In the United States, approximately seven per million people are diagnosed with aPAP. Although it can occur at any age, diagnosis most often is made between the fourth and sixth decades of life. Notably, an estimated 50% to 80% of patients with aPAP are current or former cigarette smokers.
The clinical course of aPAP can follow one of three patterns: stable disease, progressive deterioration and spontaneous resolution; high rates of spontaneous remission reported with mild disease. For patients who are asymptomatic or mildly symptomatic and do not have resting hypoxemia, immediate treatment may not be necessary; instead, they may be monitored for change in clinical status, including spontaneous remission. In patients with moderate symptoms and/or who have mild hypoxemia at rest, off-label daily inhaled GM-CSF (e.g., sargramostim [Leukine]) may be considered. Evidence suggests that nebulized GM-CSF can modestly improve lung function and facilitate clearance of antibody complex from the lungs; SC-administered GM-CSF is less well-studied. WLL is recommended in patients with moderate to severe symptoms and hypoxemia.
If approved, molgramostim would be the first medication indicated to treat aPAP. In clinical trials, treatment with nebulized molgramostim led to improvements in pulmonary gas transfer and health-related QOL in patients with aPAP.
FDA approval timeline
FDA designations: Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $10 |
| 2027 | $58 |
| 2028 | $123 |
| 2029 | $225 |
| 2030 | $323 |
pariglasgene brecaparvovec (DTX401) IV
Proposed indications
Glycogen storage disease type Ia (GSDIa)
Clinical overview
Mechanism of actionDTX401 is an adeno-associated virus vector type 8 (AAV8) gene therapy designed to deliver stable expression and activity of glucose-6-phosphatase (G6Pase).
Clinical trial(s)
A double-blind, Phase 3, cross-over trial (GlucoGene, NCT05139316) evaluated DTX401 in 46 patients 8 years of age and older with confirmed GSD1a who were receiving guideline recommended therapeutic cornstarch. Patients were randomized 1:1 to DTX401 or placebo. After week 48, eligible patients who received placebo were switched to DTX401 and those who initially received DTX401 switched to placebo; all patients were followed for an additional 96 weeks. At 48 weeks, the study met its primary endpoint reporting significant reduction in cornstarch consumption from baseline in patients who received DTX401 (n=20) compared to those who received placebo (n=24) (41% versus 10%, respectively; p < 0.0001). At 96 weeks, both the initial DTX401 group (n=20) and crossover group (n=19) achieved a mean reduction in daily cornstarch intake of 61% from baseline. In addition, at 96 weeks, approximately two-thirds of participants across both groups eliminated at least one nighttime cornstarch dose, while maintaining low incidences of hypoglycemia. Overall, 83% and 95% of patients in the initial DTX401 group and crossover group reported improvement in disease burden as measured by Patient Global Impression of Change (PGIC). DTX401 was well tolerated; expected hepatic reactions were managed with prophylactic corticosteroids, and no cases of dorsal root ganglion toxicity, malignancy, or thrombotic microangiopathy were reported.
Dosage and administration
In the clinical trials, DTX401 was administered as a single IV infusion at a dose of 1 x 10¹³ genome copies (GC)/kg dose measured by droplet digital polymerase chain reaction (ddPCR).
Place in therapy
GSDIa is a rare and life-threatening genetic disorder due to an inborn error of carbohydrate metabolism. It occurs in about one in 100,000 live births worldwide, with roughly 25% of cases in the United States and Europe. Mutations in the glucose-6-phosphatase catalytic subunit 1 (G6PC1) gene lead to deficiency of the G6Pase enzyme, preventing the release of stored glycogen or endogenous glucose production. As a result, glycogen accumulates in organs, such as the liver, kidneys, and small intestine, impairing their function. Symptoms of GSD1a typically appear between 3–6 months of age, as feeding intervals lengthen. Infants may present with episodes of severe hypoglycemia, feeding difficulties, failure to thrive, hypotonia, and developmental delays. Other characteristic features include lactic acidosis, hyperlipidemia, hypoglycemic seizures, and hepatomegaly.
There are no FDA-approved pharmacologic therapies for GSD1a. To maintain glucose levels, patients must avoid fasting and take prescribed cornstarch doses every few hours throughout the day and night. Because people with GSD1a cannot metabolize fructose or galactose, their diet must exclude fruits, dairy products, honey, molasses, maple syrup, and foods containing added sugars or sorbitol.
If approved, DTX401 will be the first pharmacologic treatment to address the underlying cause of GSD1a.
FDA approval timeline
Aug. 23, 2026
FDA designations: Fast Track, Orphan Drug, Priority Review, Rare Pediatric Disease, Regenerative Medicine Advanced Therapy
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $28 |
| 2027 | $67 |
| 2028 | $106 |
| 2029 | $147 |
| 2030 | $179 |
veligrotug IV
Manufacturer: Viridian
Proposed indications
Thyroid eye disease (TED)
Clinical overview
Mechanism of action
Veligrotug is an anti-insulin-like growth factor-1 receptor (IGF-1R) antibody
Clinical trials
Two randomized, double-blind, placebo-controlled, Phase 3 trials evaluated veligrotug in adults with moderate-to-severe active TED (THRIVE; NCT05176639; n=113) or chronic TED (THRIVE-2; NCT06021054; n=188). The primary endpoint in each trial was the rate of proptosis (abnormal bulging of eye) response, defined as reduction of proptosis of ≥ 2 mm from baseline (without a corresponding increase of ≥ 2 mm in the fellow eye) as measured by exophthalmometer. At week 15, both trials demonstrated statistically significant improvements in responder rate with veligrotug compared to placebo (THRIVE: 70% versus 5% [p<0.0001]; THRIVE-2: 56% versus 8% [p<0.0001]) and proptosis mean change from baseline (THRIVE: -2.89 versus -0.48 mm [p<0.0001]; THRIVE-2: -2.34 versus -0.46 mm [p<0.0001]). Significant improvements were also observed in key secondary endpoints, including diplopia response and Clinical Activity Score (CAS) in the veligrotug group. Clinical benefit in all measures was seen as early as week three and were maintained through week 52. Veligrotug was generally well tolerated. In THRIVE-2, the most common TEAEs (≥10%) with veligrotug were muscle spasms, hearing impairment, hyperglycemia, and menstrual disorders.
Dosage and administration
In the THRIVE and THRIVE-2 trials, veligrotug 10 mg/kg was administered via IV infusion over 30 minutes every three weeks for five doses.
Place in therapy
TED is most often associated with Graves’ disease (GD), an autoimmune condition characterized by hyperthyroidism; up to 40% of patients with GD develop TED, typically within two years of their GD diagnosis. Moderate-to-severe and sight-threatening TED are reported in approximately 6% and 0.5%, respectively, of patients with GD. Clinical signs of TED include orbital inflammation and fibrosis, which can result in permanent facial disfigurement such as proptosis, periorbital edema, strabismus, diplopia (double vision), and optic nerve damage.
Treatment approaches for TED include management of hyperthyroidism. In GD, hyperthyroidism is primarily treated with antithyroid medication or with thyroxine replacement therapy following either radioactive iodine or surgical thyroid ablation. Patients should be counseled to stop cigarette smoking, as it increases the risk for TED and worsens existing symptoms. Most people with TED have mild disease and can be managed symptomatically to address ocular dryness, pain, irritation, and light sensitivity. Some evidence suggests that short-term dietary selenium supplementation (100 μg twice daily for six months) may improve symptoms of mild TED. For moderate-to-severe active disease, systemic glucocorticoids may be used to reduce inflammation and periorbital edema. Treatment with the IGF-1R inhibitor teprotumumab-trbw (Tepezza) is preferred in patients with soft tissue involvement, proptosis or diplopia. If both Tepezza and glucocorticoid therapy are contraindicated, are not tolerated, or produce an inadequate response, off-label use of rituximab (Rituxan, biosimilars) or tocilizumab (Actemra, biosimilars) has been reported. In severe cases, orbital decompression surgery may be considered to improve proptosis, diplopia, and visual acuity.
In TED, the thyroid-stimulating hormone receptor (TSHR) on orbital fibroblasts forms a functional complex with IGF-1R, leading to enlargement of extraocular muscle and orbital connective and adipose tissue. Inhibition of the IGF-1 receptor with agents such as veligrotug and Tepezza results in apoptosis of orbital fibroblasts and adipocytes, as well as a subsequent reduction in proptosis. If approved, veligrotug will be the second IGF-1R inhibitor approved by the FDA and will compete with Tepezza to treat TED. Safety profiles are similar between the agents; however, veligrotug may offer a more convenient dosing regimen, requiring a total of five infusions administered over 30 minutes each compared to Tepezza’s eight infusions each administered over 60-90 minutes. Both therapies are given every three weeks.
Roche plans to file an sNDA for their IL-6 inhibitor satralizumab-mwge (Enspryng) for TED in 2026. Top-line results from a Phase 3 trial (NCT06248619) of teprotumumab-trbw administered by SC injection via an on-body injector (Tepezza OBI) reported comparable efficacy to IV-administered Tepezza in patients with moderate-to-severe TED. Viridian is also evaluating elegrobart, a SC-administered anti-IGF-1R antibody for the treatment of active and chronic TED. Top-line data from the Phase 3 REVEAL-1 and REVEAL-2 trials are anticipated in the first half of 2026.
FDA approval timeline
FDA designations: Breakthrough Therapy, Priority Review
Financial forecast (reported in millions)
| Year | Projected yearly U.S. sales |
|---|---|
| 2026 | $51 |
| 2027 | $261 |
| 2028 | $486 |
| 2029 | $511 |
| 2030 | $500 |
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