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Pegfilgrastim

Pegfilgrastim is a pegylated, long-acting of , a recombinant (G-CSF), that functions as a leukocyte to stimulate production and reduce the incidence of in patients undergoing myelosuppressive for non-myeloid malignancies. It is administered subcutaneously as a single 6 mg dose per chemotherapy cycle, typically at least 24 hours after chemotherapy administration, offering a convenient alternative to daily dosing required for non-pegylated G-CSF. The involves pegfilgrastim binding to specific G-CSF receptors on the surface of hematopoietic cells and mature , which promotes their , , , and functional , thereby accelerating neutrophil recovery from chemotherapy-induced myelosuppression. The (PEG) conjugation extends its to 15-80 hours compared to filgrastim's shorter duration, enabling self-regulation of serum levels through neutrophil-mediated clearance and reducing the need for multiple injections. This pharmacokinetic profile results in peak serum concentrations 1-2 days after subcutaneous injection, with nonlinear elimination that decreases as the dose increases. Pegfilgrastim was first approved by the U.S. (FDA) on January 31, 2002, under the brand name Neulasta by Amgen Inc., for decreasing the incidence of infection manifested by in patients with non-myeloid malignancies receiving myelosuppressive anticancer drugs associated with a clinically significant incidence of such events. In 2015, its indications were expanded to increase survival in patients acutely exposed to myelosuppressive doses of radiation in the setting of , with a recommended regimen of two 6 mg doses administered one week apart. Pediatric dosing is weight-based for patients under 45 kg, such as 1.5 mg for those weighing 10-20 kg, to ensure appropriate stimulation while minimizing risks like , the most common . Contraindications include hypersensitivity to pegfilgrastim or its components, with warnings for potential serious events such as splenic rupture, , and sickle cell crises in predisposed individuals.

Medical uses

Indications

Pegfilgrastim is primarily indicated to decrease the incidence of infection, as manifested by , in patients with non-myeloid malignancies receiving myelosuppressive anticancer associated with a clinically significant incidence of . This approval is based on phase III clinical trials, including a placebo-controlled study in patients receiving , demonstrating that a single 6 mg dose administered approximately 24 hours after significantly reduces the incidence of (from 17% with to 1% with pegfilgrastim). Trials comparing pegfilgrastim to showed equivalent reduction in the duration of severe (approximately 1.7 days in cycle 1). An additional approved indication is to increase survival in patients acutely exposed to myelosuppressive doses of , as part of the hematopoietic subsyndrome of , supported by efficacy data from animal models and limited human exposure scenarios. Patient selection for prophylactic use in focuses on individuals with non-myeloid cancers, such as , , or , undergoing regimens with a high risk of (greater than 20%), particularly those with risk factors including age 65 years or older, advanced disease stage, or poor . In supportive care settings, pegfilgrastim is used off-label following (HSCT) to accelerate recovery, with phase II and III trials showing it shortens the duration of severe by 1 to 2 days compared to daily , achieving recovery times of 8 to 10 days. Similarly, it is employed in (AML) patients after induction chemotherapy to reduce duration, though not approved for myeloid malignancies; clinical studies, including randomized phase II trials, report 50% to 70% reductions in duration relative to controls in select cohorts. Emerging off-label applications include management of severe chronic , where small studies and case series indicate effective elevation with less frequent dosing than , though monitoring for dosing optimization is required.

Administration and dosage

Pegfilgrastim is administered as a fixed dose of 6 mg via subcutaneous injection once per chemotherapy cycle in adult patients receiving myelosuppressive chemotherapy. This single-dose regimen differs from daily administration of the parent compound filgrastim, reflecting pegfilgrastim's prolonged half-life due to PEGylation. The injection should occur no earlier than 24 hours after completion of and not within the 14-day period before the start of the next cycle to avoid interference with cytotoxic effects on . Common sites for subcutaneous injection include the , upper arm, or thigh, with rotation recommended to minimize local reactions. Alternative formulations include the on-body injector (Neulasta Onpro), a single-use device applied to the skin on the day of , which automatically delivers the 6 mg dose approximately 27 hours later over about 45 minutes. Biosimilars such as Ziextenzo (pegfilgrastim-bmez), Udenyca (pegfilgrastim-cbqv), and others follow equivalent dosing of 6 mg subcutaneously once per cycle, with no differences in administration protocols from the reference product Neulasta. No dose adjustments are required based on body weight in adults, or for renal or hepatic impairment, as pharmacokinetic studies indicate no clinically significant alterations in these populations. In pediatric patients weighing less than 45 kg, weight-based dosing is used (e.g., 1.5 mg for 10-20 kg body weight), but fixed 6 mg dosing applies for those 45 kg or greater. For patients acutely exposed to myelosuppressive doses of in the setting of , the recommended regimen is two 6 subcutaneous doses administered one week apart for adults and pediatric patients weighing 45 or more. For pediatric patients weighing less than 45 , weight-based dosing is used (e.g., 1.5 for 10-20 body weight). The first dose should be administered as soon as possible after exposure. Monitoring of (ANC) is recommended prior to each cycle and periodically thereafter to assess response and guide timing, with a target ANC greater than 1,000/mm³ indicating recovery from . Complete blood counts, including ANC, should be evaluated to detect excessive ( >100 × 10⁹/L), which may warrant dose interruption.

Safety profile

Adverse effects

Pegfilgrastim is associated with several common adverse effects, primarily derived from clinical trials and post-marketing surveillance. The most frequently reported is bone pain, occurring in 31% of patients receiving pegfilgrastim compared to 26% in the placebo group in a pivotal placebo-controlled trial involving breast cancer patients undergoing myelosuppressive chemotherapy. Pain in the extremities was also common, affecting 9% of pegfilgrastim-treated patients versus 4% in placebo recipients. Injection site reactions, including pain, erythema, and swelling, are frequently observed, though specific incidence rates from trials are not quantified and are more commonly noted in post-approval use. Fatigue and nausea have been reported as adverse effects, but they do not consistently show a greater than 5% difference compared to placebo in clinical studies. Serious adverse effects are less common but can be severe. has been identified in post-marketing reports, with an incidence of less than 1%, and requires monitoring via if symptoms arise; splenic rupture, a potentially fatal complication, has also been reported rarely. (ARDS) is a rare but serious event, occurring in isolated cases during post-approval surveillance, and pegfilgrastim should be discontinued upon diagnosis. In patients with sickle cell disorders, pegfilgrastim can precipitate severe or fatal sickle cell crises, warranting discontinuation if this occurs. , which can lead to and , has been reported infrequently in post-marketing experience and necessitates close monitoring with symptomatic supportive care. Severe allergic reactions, including , have been reported rarely in post-marketing data, and require immediate discontinuation of the drug. Alveolar hemorrhage has been reported in post-marketing experience. Management of adverse effects focuses on symptomatic relief and prompt intervention for serious events. is typically managed with analgesics such as non-opioid pain relievers, and non-pharmacologic measures like rest may be employed. For injection site reactions, local care including cool compresses is recommended. In cases of serious adverse effects like ARDS, , , or severe allergic reactions, pegfilgrastim must be permanently discontinued, with supportive treatments initiated as needed. Patients should be monitored for signs of splenic enlargement, particularly those reporting abdominal or shoulder pain.

Contraindications and precautions

Pegfilgrastim is contraindicated in patients with a history of serious allergic reactions to pegfilgrastim, , or components such as (PEG), with reported reactions including . It is not indicated for use in patients with (CML) or myelodysplastic syndromes (MDS), and caution is advised due to the potential for tumor growth stimulation by granulocyte colony-stimulating factors (G-CSFs), with monitoring for MDS/AML progression recommended. Precautions are advised in patients with or trait, as pegfilgrastim may precipitate sickle cell crises, necessitating discontinuation if a crisis occurs. Caution is recommended in individuals with a history of (ARDS), with prompt evaluation and discontinuation if signs such as fever, lung infiltrates, or respiratory distress emerge. Pegfilgrastim should be avoided in patients with active infections until the underlying cause is addressed, as it does not treat the infection itself. Additionally, monitor for rare complications such as aortitis (with symptoms like fever and abdominal or ) or (via ), discontinuing therapy if confirmed. Drug interactions with may potentiate release, leading to , thus requiring close monitoring of () counts during concurrent use. Similarly, corticosteroids can have additive myeloproliferative effects with pegfilgrastim, warranting caution and monitoring. No significant interactions with the system are expected, given pegfilgrastim's biologic nature. Special monitoring includes assessing baseline spleen size, as splenic rupture is a , and regular complete blood counts (CBCs) to avoid excessive (discontinue if exceeds 50 × 10⁹/L). Platelet counts and should also be monitored periodically.

Pharmacology

Mechanism of action

Pegfilgrastim is a pegylated form of , a recombinant (G-CSF), designed to stimulate the production and maturation of neutrophils. It binds specifically to G-CSF receptors on the surface of hematopoietic progenitor cells and mature neutrophils within the . This binding initiates intracellular signaling cascades that promote the proliferation, differentiation, and enhanced survival of neutrophils, thereby increasing their release from the storage pool into the bloodstream. Upon receptor binding, pegfilgrastim induces dimerization of the G-CSF receptor, activating key downstream pathways such as JAK-STAT, which translocates to the to upregulate genes involved in neutrophil development and function. Additional pathways, including PI3K/AKT and MAPK/ERK, contribute to anti-apoptotic effects and functional activation of s, ensuring a rapid and sustained response to chemotherapy-induced myelosuppression. The polyethylene glycol (PEG) conjugation in pegfilgrastim extends its compared to unmodified by reducing renal clearance, while preserving the core receptor binding affinity and of the parent molecule. This modification does not alter the dissociation constant (Kd) for G-CSF receptor binding, equivalent to . Pegfilgrastim exhibits a self-regulating mechanism through -mediated clearance: as circulating counts rise in response to , the is internalized and degraded by these cells via receptor , leading to increased elimination and preventing excessive accumulation. This feedback loop aligns exposure with the degree of , optimizing therapeutic efficacy.

Pegfilgrastim exhibits nonlinear following , primarily due to its self-regulating clearance mediated by neutrophils.
Pegfilgrastim is slowly absorbed after subcutaneous injection, with a time to concentration (Tmax) of 1 to 2 days, which is slower than because of the () moiety that promotes lymphatic uptake. The absolute is lower than that of , estimated at 20% to 50%, while relative bioavailability compared to a 30 μg/kg dose is approximately 80% to 94% at higher doses.
Distribution
The volume of distribution is approximately 170 L, consistent with distribution primarily in the compartment and limited tissue beyond the . No significant differences in distribution are observed based on , , or renal .
Metabolism and elimination
is not metabolized by hepatic enzymes; instead, it undergoes nonspecific following by via receptor . Clearance is predominantly -mediated, resulting in reduced renal excretion compared to , and is self-regulating such that it decreases with rising absolute counts (ANC). The elimination is dose-dependent, ranging from 15 to 80 hours (median approximately 42 hours). are nonlinear, with higher systemic exposure observed at lower counts due to diminished clearance.
Key parameters
The area under the concentration-time curve () increases more than proportionally with dose, reflecting the nonlinear clearance. Apparent serum clearance is approximately 14 mL/hour/kg and is lower at reduced ANC levels (e.g., inversely correlated with neutrophil-mediated elimination). This pattern aligns with the drug's , where clearance feedback is tied to recovery.

Chemistry

Molecular structure

Pegfilgrastim is a pegylated form of , a recombinant (G-CSF), consisting of filgrastim covalently conjugated to a 20 kDa monomethoxypolyethylene glycol (mPEG) molecule at the N-terminal residue via reductive . This modification involves the attachment of the PEG derivative to the amino group of the N-terminal methionine under mildly acidic conditions (pH 5), ensuring site-specific conjugation without altering the receptor-binding interface. The protein core of pegfilgrastim comprises 175 , identical to the sequence of endogenous G-CSF except for the addition of an N-terminal residue required for bacterial expression. The unmodified portion has a molecular weight of approximately 19 kDa, while the addition of the 20 kDa results in a total average molecular weight of about 39 kDa for pegfilgrastim. Pegfilgrastim is produced through technology, with the polypeptide expressed in via a genetically engineered containing the G-CSF . Following purification of the filgrastim, is performed as a step on the isolated protein. Due to its bacterial origin, pegfilgrastim lacks , resulting in a non-glycosylated structure that differs from the naturally occurring glycosylated G-CSF. The of pegfilgrastim increases its from approximately 4 nm to 6 nm, which reduces renal clearance by limiting glomerular while preserving the of the G-CSF moiety. Additionally, this modification shields the protein from immune recognition, thereby decreasing compared to unmodified .

Physical and chemical properties

Pegfilgrastim is formulated as a clear, colorless, preservative-free for subcutaneous , with a concentration of 10 mg/mL (based on protein content) in a 10 mM at 4.0, containing 5% and 0.004% as excipients. This formulation ensures compatibility with pre-filled syringes (typically 6 mg/0.6 mL) and supports direct subcutaneous without dilution. The modification enhances its aqueous , making it highly soluble in water and buffered solutions suitable for parenteral use. The protein exhibits an (pI) of approximately 6.2 (range 6.14–6.22), resulting in a net negative charge at physiological and contributing to its acidic behavior in . Pegfilgrastim is stable when stored refrigerated at 2–8°C, with a of up to 3 years in its original , protected from ; per the FDA , it should not be left at for more than 48 hours, while the product information allows up to 72 hours at ≤30°C, after which it must be discarded if frozen or left at ambient conditions longer than specified to prevent degradation. It is particularly sensitive to mechanical agitation, which can induce foaming and , and to improper storage, leading to chemical degradation pathways such as of or residues and oxidation of or . Analytical characterization of pegfilgrastim relies on techniques such as reversed-phase (RP-HPLC) for purity and oxidation assessment, size-exclusion HPLC (SE-HPLC) for aggregation detection, cation-exchange HPLC (CEX-HPLC) for charge variants, and for site-specific modifications including attachment and degradation products. () spectroscopy confirms the covalent linkage to the N-terminal residue. These methods ensure by verifying structural integrity and stability under various stress conditions.

History

Development

Pegfilgrastim was developed by in the late as a PEGylated form of , the recombinant human (G-CSF) marketed as Neupogen and approved by the FDA in 1991, to extend its circulating half-life and eliminate the need for daily subcutaneous injections during chemotherapy cycles. The motivation stemmed from 's established role in stimulating production to mitigate chemotherapy-induced , but its short half-life necessitated multiple doses per cycle, posing patient inconvenience and compliance challenges. , the covalent attachment of a () chain to the N-terminal of , was selected based on prior research into protein modification techniques to enhance without substantially altering . Research on PEG-filgrastim originated shortly after filgrastim's approval, with initiating PEGylation studies in the early 1990s to build on the molecule's pluripotent hematopoietic effects. A key milestone was the filing of on June 2, 1995, covering methods for preparing PEGylated G-CSF analogs, which laid the foundation for the drug's commercialization. focused on confirming the modified molecule's safety and efficacy in non-human models, serving as a bridge to filgrastim's established profile. In preclinical studies, pegfilgrastim exhibited prolonged stimulation in animal models, including cynomolgus monkeys, where a single dose sustained absolute counts (ANC) for up to 10-14 days compared to filgrastim's shorter duration, due to reduced renal clearance from the moiety. Toxicity assessments in rodents and monkeys revealed dose-dependent hyperplasia as the primary effect, consistent with G-CSF , with no unexpected toxicities or at therapeutic levels; the (NOAEL) was established at 0.25 mg/kg in monkeys. These findings supported advancement to clinical testing, confirming pegfilgrastim's potential for once-per-cycle administration while maintaining filgrastim's stimulatory potency on . Clinical development progressed through Phase I and II trials from 1998 to 2000, evaluating safety, , and in healthy volunteers and cancer patients receiving myelosuppressive . In a multicenter Phase I/II dose-escalation study involving women with treated with and , single doses of pegfilgrastim (30-300 µg/kg) produced dose-proportional increases in ANC, with a 100 µg/kg dose achieving median ANC nadirs and recovery times comparable to daily (5 µg/kg), and a favorable safety profile limited to mild . These trials established the fixed 6 mg dose for further evaluation, demonstrating single-dose efficacy in preventing severe without increased adverse events. Pivotal Phase III trials conducted in 2001 confirmed efficacy. One multicenter trial randomized 310 patients receiving four cycles of plus to a single 100 µg/kg pegfilgrastim injection or daily (5 µg/kg) per cycle. A second trial randomized 157 patients to a single fixed 6 mg pegfilgrastim dose or daily filgrastim. Results from both showed equivalent efficacy, with mean duration of severe of approximately 1.7 days in the pegfilgrastim arms versus 1.6 days for filgrastim, and low incidence (around 3-13%), confirming non-inferiority (p < 0.001 for equivalence). Safety was similar, with reported in 26% of pegfilgrastim patients versus 28% on filgrastim, supporting the molecule's transition to regulatory submission. These milestones culminated in pegfilgrastim's readiness for approval by early 2002.

Regulatory approvals

Pegfilgrastim, marketed as Neulasta by , received initial approval from the U.S. (FDA) on January 31, 2002, for decreasing the incidence of infection, as manifested by , in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs associated with a clinically significant incidence of . This approval was supported by data from two randomized, double-blind, active-controlled III clinical trials involving a total of 467 patients with (157 in one trial and 310 in the other), which demonstrated that pegfilgrastim was noninferior to daily 5 μg/kg doses of in reducing the duration of severe following myelosuppressive . Subsequent label expansions included an indication in 2015 for increasing survival in patients acutely exposed to myelosuppressive doses of , based on data from and supportive clinical evidence. In Europe, the () granted centralized marketing authorization for Neulasta on August 22, 2002, for reducing the duration of and the incidence of following cytotoxic for solid tumors, , and (excluding chronic myeloid leukemia and myelodysplastic syndromes). The authorization was based on similar phase III trial data showing noninferiority to . Pediatric use was later incorporated into the label following completion of a pediatric investigation plan, with evidence supporting its application in children aged 2 months and older receiving . Pegfilgrastim was approved in other regions shortly following initial U.S. and EU authorizations, including in on March 12, 2004, by for indications consistent with FDA and approvals to reduce duration in patients. In , approval was granted by the in 2003 for decreasing incidence in nonmyeloid patients undergoing myelosuppressive . Approvals in occurred later, with Kyowa Kirin receiving authorization for G-Lasta (pegfilgrastim) on September 26, 2014, for similar chemotherapy-induced reduction. The FDA approved the first pegfilgrastim , Fulphila (pegfilgrastim-jmdb) by , on June 4, 2018, based on analytical, nonclinical, and clinical studies demonstrating similarity to Neulasta, including a phase III trial in 218 patients showing comparable duration of severe . Subsequent approvals included Udenyca (pegfilgrastim-cbqv) on November 2, 2018; Ziextenzo (pegfilgrastim-bmez) on November 4, 2019; Nyvepria (pegfilgrastim-apgf) on June 10, 2020; Fylnetra (pegfilgrastim-pbbk) on May 26, 2022; Stimufend (pegfilgrastim-fpgk) on September 1, 2022; and Pyzchiva (pegfilgrastim-apgx) on October 30, 2023, all referencing Neulasta for the same core indications. As of 2025, seven pegfilgrastim biosimilars had received FDA approval, enhancing access and competition in supportive cancer care. Label updates for Neulasta have incorporated postmarketing safety data, including warnings for potential splenic rupture associated with added following reports of cases in 2005, and aortitis added in 2010 based on rare inflammatory vascular events observed in clinical use. Beginning in 2023, certain pegfilgrastim biosimilars received FDA designations for interchangeability with Neulasta, allowing pharmacy-level substitution without prescriber intervention in applicable states.

Society and culture

Brand names and biosimilars

Pegfilgrastim is commercially available under the brand name Neulasta, developed and marketed by , which was approved by the FDA in January 2002 for reducing the incidence of in patients with non-myeloid malignancies receiving myelosuppressive . An on-body variant, Neulasta Onpro, was approved by the FDA in 2014 to provide automated subcutaneous delivery approximately 27 hours after activation. Several biosimilars to Neulasta have been approved by the FDA, demonstrating no clinically meaningful differences from the reference product in terms of safety, purity, and potency. These include:
  • Fulphila (pegfilgrastim-jmdb) by and , approved in June 2018
  • Udenyca (pegfilgrastim-cbqv) by Coherus BioSciences, approved in November 2018
  • Ziextenzo (pegfilgrastim-bmez) by , approved in November 2019
  • Nyvepria (pegfilgrastim-apgf) by , approved in June 2020
  • Fylnetra (pegfilgrastim-pbbk) by , approved in May 2022
  • Stimufend (pegfilgrastim-fpgk) by Fresenius Kabi, approved in September 2022
For example, an on-body injector version of Udenyca (UDENYCA ONBODY) was approved by the FDA in December 2023 for automated delivery. The introduction of these biosimilars has contributed to market competition, leading to an overall reduction in U.S. claim-level costs for pegfilgrastim by approximately 23% across channels following their entry in 2018. Global availability of pegfilgrastim biosimilars varies by region; for example, Rixfil (by ) is approved and marketed in the . Most approved pegfilgrastim products, including the reference and biosimilars, are formulated as 6 mg/0.6 mL solutions in single-dose pre-filled syringes for . Some biosimilars, such as Udenyca, also offer on-body injector options. Some biosimilars incorporate minor formulation variations, such as differences in buffering agents (e.g., citrate versus ), to optimize stability while maintaining . In the United States, pegfilgrastim is classified as a biologic product and is not subject to the scheduling system, as it does not have abuse potential. It is available by prescription only, requiring administration under medical supervision or self-injection following healthcare provider instructions. Part B covers pegfilgrastim for eligible patients undergoing myelosuppressive to reduce the risk of , with coverage typically including outpatient administration and associated costs after deductibles and . The drug received orphan drug designation from the FDA for the treatment of in 2013, providing market exclusivity for that rare indication until its approval in 2015, though prior designations for other rare neutropenic conditions had expired by 2009, limiting ongoing orphan benefits primarily to non-rare uses. Internationally, the (EMA) classifies pegfilgrastim as a prescription-only , restricting its distribution to authorized healthcare settings for cancer patients at risk of . The included pegfilgrastim on its Model List of in 2023 specifically for prophylaxis of in adults receiving myelosuppressive . Pegfilgrastim is prohibited by the (WADA) under the category of growth factors, as it stimulates production and is considered performance-enhancing in sports; its use requires a therapeutic use exemption in competitive athletics and is controlled or banned in many countries adhering to WADA standards. Access to pegfilgrastim is supported by patient assistance programs, such as the Safety Net Foundation, which provides the medication at no cost to qualifying uninsured or underinsured U.S. patients based on income and residency criteria. Copay assistance cards are available for s, enabling commercially insured patients to reduce out-of-pocket expenses to as low as $0 per dose through programs like Oncology Together. In low- and middle-income countries, access remains restricted due to high costs—approximately $3,000 to $5,000 per dose for the originator product prior to availability—often exceeding annual per capita health expenditures and limiting use despite WHO essential status. The original U.S. patents for pegfilgrastim expired in 2015, which facilitated the approval and market entry of biosimilars by removing key barriers. Pediatric exclusivity, granted for studies in younger populations, extended this protection by six months until April 2016, further delaying competition until regulatory pathways for biosimilars matured.

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