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darbepoetin alfa (Darbecure)

✓ Approved

Emcure Pharmaceuticals · EPOR · 重组蛋白

什么是 darbepoetin alfa？

darbepoetin alfa 是一种重组蛋白，由Emcure Pharmaceuticals研发。该药已获批，用于治疗相关适应症，给药途径：Injectable (Others)、Intravenous (IV)。

药物档案

商品名Darbecure
公司Emcure Pharmaceuticals
药物类别重组蛋白, 多肽类
分子靶点EPOR
给药途径Injectable (Others), Intravenous (IV)
状态Approved
来源： ClinicalTrials.gov, Emcure Pharmaceuticals

作用机制

分子靶点

darbepoetin alfa 作用于 1 个分子靶点：

EPORerythropoietin receptor (EPO-R)
需要更深入的分析？Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

darbepoetin alfa 针对 2 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Blood and lymphatic system disordersAnaemia✓ Approved
Blood and lymphatic system disordersNephrogenic anaemia✓ Approved

相关研究文献

PubMedJournal of virology2026-07-27

Rapid and robust immune response boosting with potent, next-generation adjuvant in viral vector-primed primates.

King Hannah A D HAD, Subra Caroline C, Tourtellott Emily E, Swafford Isabella I et al.

To identify strategies for augmenting vaccine immunogenicity, we compared a pox-protein prime-boost regimen comprising recombinant modified vaccinia virus Ankara and multimeric HIV-1 Env gp145, adjuvanted with Army Liposomal Formulation (ALF) either adsorbed to aluminum salt (ALFA) or formulated with the QS-21 saponin (ALFQ), in rhesus macaques. ALFQ promoted greater magnitude and more durable humoral and cellular immune responses than ALFA, which exhibits similar immunogenicity to aluminum-based adjuvants. Peak Env-specific CD4+ T cell responses assessed by intracellular cytokine staining were 10-fold greater with ALFQ, and CD8+ T cell responses were unexpectedly robust, averaging greater than 1%. ALFQ induced higher levels of several immunostimulatory cytokines in plasma, which correlated with adaptive immune responses. However, vaccination did not protect against heterologous intrarectal challenge with transmitted/founder SHIV-CH505. We provide evidence that CH505 Env may maintain a relatively closed conformation, rendering it less susceptible to targeting by Fc-mediated antibody functions. Overall, ALFQ is a promising adjuvant to improve antibody and T cell response magnitude.IMPORTANCEAn effective and durable vaccine preventing HIV-1 acquisition is urgently needed to end the HIV-1 pandemic. To date, of the nine vaccine efficacy trials conducted in humans, only the RV144 vaccine trial demonstrated efficacy in reducing infections, although immune responses waned rapidly. We evaluated a modified HIV-1 vaccine regimen incorporating next-generation adjuvants to improve immune responses and vaccine efficacy in a gold standard, pre-clinical primate model. Adjuvanted protein boosting markedly increased antibody, T cell, and pro-inflammatory response magnitude. These data, combined with the adjuvant's strong safety and immunogenicity track record in clinical trials, indicate that novel adjuvants represent a promising strategy for improving immune responses to protein immunogens. Future vaccine regimens against HIV-1 and other pathogens for which eliciting robust immunity has been difficult may benefit from incorporating these or related next-generation adjuvants.

PMID 42505121
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PubMedJIMD reports2026-07-27

Early Versus Late Enzyme Replacement Therapy in Siblings With Morquio A Syndrome: Insights Into Therapeutic Timing.

Choi Shinjie S, Kim Hyoungmin H, Cho Tae-Joon TJ, Ko Jung Min JM

Enzyme replacement therapy (ERT) with elosulfase alfa is the only approved treatment for mucopolysaccharidosis type IVA. This case report delineates the 5-year outcomes of ERT in two Korean siblings with mucopolysaccharidosis type IVA, with the younger sibling initiating treatment at 0.8 years of age and the older one at 5.4 years. Both patients exhibited a progressive decline in their height standard deviation scores, with trajectories paralleling the natural history curves of the disease. At 5.2 years of age, persistent skeletal dysplasia was evident in both siblings. However, the younger sibling demonstrated attenuated disease severity, lacking cervical myelopathy or spinal stenosis requiring C1 laminoplasty. Cardiorespiratory assessment revealed normalized left ventricular mass index z-scores, stable ejection fractions, and the absence of valvular pathology. Overall, these findings suggest that early ERT attenuates severe spinal and upper body manifestations but does not prevent lower limb skeletal progression, highlighting the need for early therapeutic initiation along with orthopedic intervention to preserve cardiorespiratory parameters and functional independence.

PMID 42504165
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PubMedJournal for immunotherapy of cancer2026-07-25

Durable responses to triplet immunotherapy targeting TGF-β, PD-L1, and tumor antigen, with an IL-15 receptor superagonist in mismatch repair proficient castration-resistant prostate cancer.

Redman Jason Mark JM, Madan Ravi A RA, Donahue Renee N RN, Toney Nicole J NJ et al.

Immune checkpoint blockade is minimally active in unselected castration-resistant prostate cancer (CRPC) and does not reproducibly yield durable decreases in prostate-specific antigen (PSA) levels. The Quick Efficacy Seeking Trial was designed to employ a combination of agents to initiate an immune response (with BN-Brachyury vaccine), potentiate that response (with nogapendekin-alfa inbakicept (NAI), an interleukin (IL)-15 receptor superagonist), and reduce or eliminate immunosuppressive entities in the tumor microenvironment (with bintrafusp alfa, a dual inhibitor of programmed death-ligand 1 and transforming growth factor beta). Epacadostat (an indoleamine 2,3-dioxygenase (IDO) inhibitor) was also employed in one cohort to reduce immune suppression induced by IDO's conversion of tryptophan to kynurenine. Patients with CRPC enrolled sequentially to receive vaccine + bintrafusp alfa (Arm 2.1), vaccine + bintrafusp alfa + NAI (Arm 2.2), and vaccine + bintrafusp alfa + NAI + epacadostat (Arm 2.3), with the primary objective to determine response rate. Adverse events in Arms 2.1 and 2.2 were manageable and consistent with the safety profiles of each agent individually, and notable for five individuals developing isolated adrenocorticotropic hormone deficiency. Arm 2.3 was closed early due to skin toxicity. Sustained declines in PSA were seen in 1/13 (8%) patients in Arm 2.1, 7/24 (29%) patients in Arm 2.2, including six with proficient mismatch repair/microsatellite stable tumors, and 0/6 (0%) patients in Arm 2.3. Analyses of peripheral immune profiles provided evidence of a multifaceted antitumor immune response, including IL-15 receptor superagonist NAI-dependent expansion and activation of natural killer cells and CD8+ T cells, increased effector-to-suppressor immune cell ratios, and induction of cytotoxic immune gene programs. NCT03493945.

PMID 42498485
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PubMedHaemophilia : the official journal of the World Federation of Hemophilia2026-07-24

Extended Half-Life Recombinant Factor VIII Conjugated with Modifying Substances Does Not Affect Fibrin Clot Formation or Stability in Haemophilia A Blood Samples.

Shimonishi Naruto N, Nogami Keiji K

Various extended half-life recombinant factor VIII (EHL-FVIII) products have been designed to improve the pharmacokinetic properties of FVIII, allowing prolonged haemostatic coverage and reducing the injection burden in people with haemophilia A. Nevertheless, the influence of direct molecular attachment on fibrin clot formation and stability remains to be investigated. To investigate the stability and architecture of fibrin clots in the presence of various types of EHL-FVIII. Three EHL-FVIII products with direct attachment modifications (damoctocog alfa pegol, efraloctocog alfa, and rurioctocog alfa pegol) and two standard FVIII (turoctocog alfa and rurioctocog alfa) were added to FVIII-deficient whole blood and plasma at various concentrations for functional comparison. Under whole-blood conditions, functional assays were performed using rotational thromboelastometry (ROTEM) and a microchip flow-chamber system (T-TAS). Under plasma-based conditions, fibrin fibres were directly observed by electron microscopy and coagulation function was assessed using clot waveform analysis (CWA). Anticoagulant and fibrinolytic activities were evaluated by CWA with the addition of activated protein C and tissue plasminogen activator, respectively. At equivalent activity levels, none of the assays revealed significant differences among the three EHL products or the two standard products. All contributed comparably to fibrin clot formation and stability, as well as to anticoagulation and fibrinolysis functions. Direct modification by PEGylation or IgG-Fc fusion to impart EHL characteristics preserves the functional properties of native FVIII. People with haemophilia A require treatment with factor VIII (FVIII) to prevent or control bleeding. Some FVIII products are designed to remain active in the body for a longer time, which can reduce the number of injections needed. This extended half-life is achieved by chemically or biologically modifying FVIII, for example by attaching polyethylene glycol (PEG) or the Fc portion of immunoglobulin G. These treatments are known as extended half-life FVIII (EHL-FVIII) products. However, it has not been fully established whether these modifications affect how blood clots form and remain stable. In this study, we compared three EHL-FVIII products with two standard FVIII products using FVIII-deficient blood and plasma. We evaluated clot formation and stability using several laboratory techniques, including whole-blood assays and scanning electron microscopy. We also examined potential differences in anticoagulant and fibrinolytic properties. At comparable FVIII activity levels, we observed no major differences between the EHL-FVIII products and the standard FVIII products in any of the assays performed. These findings suggest that PEGylation or Fc fusion, which are used to extend the half-life of FVIII, do not impair its functional properties related to fibrin clot formation and stability.

PMID 42496038
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PubMedMolecular genetics and metabolism reports2026-07-24

A challenging case of ASMD (acid sphingomyelinase deficiency): A severe interstitial lung disorder in an asplenic patient.

Guimas Arlindo A, Martins Esmeralda E

Acid sphingomyelinase deficiency (ASMD) is a rare lysosomal storage disorder with multisystemic involvement. We report a 68-year-old asplenic man with late-onset ASMD and severe interstitial lung disease, chronic respiratory failure, and markedly reduced diffusion capacity. Treatment with olipudase alfa resulted in significant clinical, functional, and biomarker improvement despite advanced age and disease severity. This case supports the benefit of enzyme replacement therapy in patients with complex, late-presenting ASMD.

PMID 42495085
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PubMedBMJ case reports2026-07-24

Spontaneous CSF rhinorrhoea in a patient with factor VII deficiency: surgical and haematological considerations.

Maniyankode Krishnamohan Goutham G, Chakravarthy Priya P

A middle-aged woman presented with a 4-month history of watery nasal discharge and was diagnosed with spontaneous cerebrospinal fluid (CSF) rhinorrhoea. Routine blood tests revealed a prolonged prothrombin time (PT) leading to further evaluation and the incidental diagnosis of factor VII deficiency. To optimise safety before imaging, the patient received intravenous recombinant factor VII (NovoSeven, Eptacog alfa), after which CT cisternography demonstrated a persistent CSF leak from the cribriform plate. To avoid repeated recombinant factor VII administration, endoscopic endonasal repair of the defect was undertaken immediately, with meticulous intraoperative haemostasis. Her PT and international normalised ratio were closely monitored in the postoperative period. During more than 1 year of follow-up, she remained well with no recurrence. This case underscores the challenges of balancing haemostatic control during surgical intervention in patients with rare bleeding disorders. Notably, no prior reports in the literature describe CSF rhinorrhoea occurring in the setting of factor VII deficiency, making this presentation unique.

PMID 42493220
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