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Zidovudine

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Zidovudine

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Method of action: Antivirals For Systemic Use

Medically reviewed

Laura Arias

Last updated on 22/12/2025

This page provides general, reference-level information compiled from official medical sources. It is not a substitute for professional medical advice, diagnosis, or treatment. For decisions about your health, please consult a qualified healthcare professional.

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Overview of Zidovudine

What is Zidovudine? Quick Facts and Overview

Property Description
Active Ingredient Zidovudine (AZT)
Pharmacological Class Antiretroviral agent, Nucleoside Reverse Transcriptase Inhibitor (NRTI)
Origin Synthetic, Nucleoside analog
Forms Tablet, Capsule, Oral Solution, IV Injection
General Purpose To limit the replication of the Human Immunodeficiency Virus (HIV)

What Type of Medicine is Zidovudine (AZT)?

Zidovudine is an antiretroviral agent classified as a Nucleoside Reverse Transcriptase Inhibitor (NRTI). The drug is the official non-proprietary name for the synthetic chemical compound historically known as Azidothymidine or AZT. This active ingredient is a synthetic nucleoside analog, designed to chemically mimic the natural building blocks of genetic material. Zidovudine’s classification as an NRTI is recognized by global health authorities and it is included on the WHO Model List of Essential Medicines. The medicine’s composition consists solely of the active substance Zidovudine combined with necessary pharmaceutical excipients or an aqueous solution, depending on the final preparation.


What is the General Purpose of Zidovudine?

The general purpose of Zidovudine is to limit the spread and slow the progression of the Human Immunodeficiency Virus (HIV) within the body. It achieves this by directly interfering with a critical enzyme the virus uses to copy its genetic information, a core action known as reverse transcriptase inhibition. Zidovudine is used to restrict the production of new virus particles, which is critical for managing chronic viral infections. By helping to reduce the overall viral load—the amount of active virus—Zidovudine is intended to support and maintain the function of the patient’s immune system.


In What Forms is Zidovudine Available?

Zidovudine is manufactured as a single-entity product, which is a key distinguishing factor, though it is almost universally prescribed as part of a multi-drug regimen. It is available in multiple physical forms for systemic delivery: the solid tablet and capsule, a suitable oral solution for liquid ingestion, and a preparation for intravenous (IV) injection. The oral solution form is particularly important, as it facilitates administration to pediatric patient groups who may be unable to swallow tablets. The availability of both oral and intravenous administration routes ensures essential flexibility, which is a significant factor in managing treatment needs across diverse patient populations.

Regulatory References

  1. WHO Essential Medicines List for Zidovudine
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What side effects are possible with Zidovudine?

Possible Side Effects and Safety Information

The official safety information for Zidovudine classifies adverse reactions according to frequency and affected body systems. The most critical safety information is highlighted by regulatory authorities due to the potential for serious adverse reactions.


Serious Adverse Reactions

Regulatory documents emphasize the potential for severe, clinically significant adverse events:

  • Severe Hematologic Toxicity: This includes severe anemia and neutropenia, which may require dose adjustment or, in the case of anemia, blood transfusion. This risk is noted to be higher in patients with advanced HIV-1 disease.
  • Lactic Acidosis and Hepatotoxicity: Severe lactic acidosis (an excess of lactic acid in the blood) and severe hepatomegaly with steatosis (enlargement of the liver with fat accumulation) have been reported and can be fatal.
  • Symptomatic Myopathy: Muscle weakness and pain associated with prolonged use of Zidovudine.

Common Adverse Reactions and Safety Patterns

In adult clinical trials, reactions classified as having an incidence of 15% or greater include nausea, headache, malaise, vomiting, and anorexia. These effects primarily involve the nervous and gastrointestinal systems.

Safety notes also indicate time-related patterns. For instance, neutropenia typically occurs later in treatment, often around 6 to 8 weeks after initiation. The medicine is contraindicated in individuals with known hypersensitivity to the compound. Additionally, Zidovudine should not be administered alongside any other Zidovudine-containing products.

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Overdose and Emergency Response

Zidovudine Overdose and When to Seek Help

The documented profile of Zidovudine overdose is derived exclusively from regulatory labeling and case reports. Overdose manifestations typically involve gastrointestinal disturbances, such as nausea and vomiting, and central nervous system effects, including headache, drowsiness, and fatigue. More specific neurological signs, such as ataxia and ocular nystagmus, have also been reported.

Overdose carries the risk of severe systemic toxicity, which includes complications known to be associated with Zidovudine, such as potentially fatal lactic acidosis and severe hematologic toxicity (anemia and neutropenia). Acute events like collapse or seizure necessitate immediate action. Specific findings, including transient metabolic acidosis and persistent neutropenia, have been noted in overdose cases involving neonates and infants.

In the event of suspected overdose, it is mandated that patients seek immediate medical attention. Authorities require calling emergency services if the affected individual has collapsed, experienced a seizure, or cannot be awakened. There is no known specific antidote for Zidovudine. Management consists of discontinuing the medication and providing supportive and symptomatic treatment, often requiring hospital monitoring of clinical status and blood counts.

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Therapeutic Uses of Zidovudine

What Zidovudine Treats: Main Uses and Benefits

Zidovudine is commonly used to address the systemic conditions caused by the Human Immunodeficiency Virus (HIV) and is relevant across two key therapeutic areas: the long-term treatment of infection and the mitigation of viral transmission. This use is applied across different therapeutic contexts.

The primary therapeutic benefit involves easing the overall symptom load by supporting immune system function and assisting in the long-term management of the disease. This action helps maintain a sense of stability when symptoms are more noticeable. The key uses for Zidovudine include treating established HIV-1 infection, assisting in the preservation of immune health, and is relevant for easing the overall symptomatic burden by addressing the potential risk of mother-to-child transmission.

“It is applied across domains where additional symptomatic support is needed, contributing to improved comfort during periods of heightened symptoms.”


Quick Fact: Support for HIV-Related Immune Health
Zidovudine is considered relevant for supporting patient health by aiding in the maintenance of functional stability within the immune system and helping to lower the potential risk of vertical transmission in high-risk scenarios.
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Eligibility and Restrictions for Use

Zidovudine's population eligibility is strictly defined by regulatory authorities based on specific clinical parameters and age thresholds. It is officially approved for adults and pediatric patients aged four weeks and older for the treatment of HIV-1 infection, and is indicated for pregnant women and neonates for preventing maternal-fetal transmission.


Absolute Contraindications

Zidovudine must not be used (is contraindicated) in individuals who have a known hypersensitivity to the medicine or its components. Absolute non-eligibility is also defined by pre-treatment blood counts: patients with severe anemia (haemoglobin less than 7.5 g/dL) or severe neutropenia (neutrophil counts less than 0.75 imes 10^9/ L) are formally excluded from use.


Restrictions and Limitations

Use is restricted and requires special consideration in several populations. Patients with severe renal impairment require a regulatory dosage adjustment, and use is not recommended for those with moderate to severe liver disease due to insufficient data for reliable dosing. Caution is also advised for patients with pre-existing bone marrow compromise or co-infection with chronic Hepatitis B or C. Furthermore, standard use is not established in older adults (over 65 years) because pharmacokinetic data is lacking in this population.

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What should I know about interactions with other medicines?

Zidovudine Interactions with other medicines and products

Official regulatory documentation structures Zidovudine's interaction profile around two primary concerns: avoiding antagonism of its antiviral effect and managing potential increased exposure in the body. Certain combinations are formally restricted or prohibited to mitigate these risks.


Documented Interaction Restrictions

Category Interacting Substance Restriction Status (Official Labeling)
Antagonism (Avoid) Stavudine, Doxorubicin Co-administration should be avoided due to documented in vitro antagonism of antiviral activity.
Prohibited Other Zidovudine-Containing Products Must not be administered concomitantly.
Not Advised Ribavirin Co-administration is not advised due to the risk of exacerbating anemia.
Herbal Product St. John's Wort Use is prohibited as a concomitant medication.

Exposure and Toxicity Interactions

Substances that inhibit Zidovudine's clearance can lead to increased plasma concentrations (exposure). Medicines such as Probenecid and Trimethoprim are documented to reduce its elimination by inhibiting renal excretion or metabolism (glucuronidation). Co-administration with other bone marrow suppressive or cytotoxic agents may lead to additive hematologic toxicity. Caution is also noted for patients with renal or hepatic impairment as these conditions inherently slow Zidovudine’s removal from the body, potentially increasing its effects. Food does not typically necessitate separation; Zidovudine may be taken with or without food.

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Mechanism of Action

How Zidovudine Works

Enzyme-Targeted Molecular Blockade

The core mechanism of Zidovudine relies on its function as a prodrug, which is transformed by host cellular kinases into the active molecule, Zidovudine triphosphate (ZTV-TP). ZTV-TP then acts as a substrate mimic to competitively inhibit the viral enzyme Reverse Transcriptase (RT). This interaction results in the immediate and irreversible DNA chain termination, which prevents the Human Immunodeficiency Virus (HIV) from converting its genetic material into proviral DNA.


Systemic Suppression of Viral Replication

By preventing the successful synthesis of proviral DNA, the drug fundamentally blocks a critical early step in the HIV replication cycle. This cascade leads to a systemic suppression of new infectious particle production. The resulting physiological effect is a measurable decrease in the viral load throughout the body, which, in turn, results in the maintenance of CD4+ T-cell population.


Mechanism Constraints and Viral Adaptability

The mechanism is constrained by two primary factors: the required efficiency of the host's cellular phosphorylation process for activation, and the virus's ability to develop resistance. Specific Reverse Transcriptase mutations can weaken the drug's binding affinity or enhance the enzyme's capacity to excise the incorporated chain terminator, thereby compromising the intended molecular blockade and permitting continued proviral DNA synthesis.

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Dosage and Administration Information

How Zidovudine is Used: Official Administration Guidelines

Zidovudine (AZT) is administered according to precise protocols that define its routes, standard dosing, and frequency. The medicine is primarily used via the oral route for long-term daily management in adults and children, but it is also available as a Concentrate for Intravenous (IV) Infusion for specific short-term uses.

Oral administration is most commonly structured as a total dose of 600 mg daily, typically divided into 300 mg taken twice daily or 200 mg taken three times daily, and may be administered without regard to food.


Specialized Administration Contexts

Context Route and Dose Principle Duration Pattern
Intrapartum Administration IV Infusion involving a 2 mg/kg loading dose followed by a 1 mg/kg/hour continuous infusion. Used until the umbilical cord is clamped.
Neonatal Administration 2 mg/kg orally every 6 hours or 1.5 mg/kg IV every 6 hours. Continues from birth through 6 weeks of age.
Renal Impairment Oral dose is reduced to 100 mg every 6 to 8 hours for severe impairment. Single-entity products allow for this adjustment.

Preparation and Procedural Rules

The IV concentrate requires mandatory dilution in 5% Dextrose Injection to a final concentration no greater than 4 mg/mL before use. This diluted solution must then be administered via controlled infusion over one hour for adults, as rapid injection is restricted. For patients unable to swallow tablets, the oral solution or syrup form is the designated alternative, providing necessary dose flexibility for various populations.

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Recent Clinical Evidence

Research evidence / Overview of studies for Zidovudine

The clinical record for Zidovudine is based on findings from a wide range of studies, including early placebo-controlled trials, large combination therapy studies, and long-term observational research. The evidence contributes to understanding symptom patterns and is applied in the regulatory evaluation of this antiretroviral drug. This overview describes the types of research conducted, the outcomes monitored, and the acknowledged limitations in the evidence base.


Evidence for use in Established HIV-1 Infection

This section will summarize the types of research, including Randomized Controlled Trials (RCTs), comparative studies, and observational cohorts, that evaluated Zidovudine was studied for use as a core component of combination antiretroviral therapy (cART) in research populations diagnosed with HIV-1 infection. Early research examined markers related to systemic or functional imbalance, such as changes in viral load measurements and T-lymphocyte cell counts.

More recent research explores Zidovudine as part of a multi-drug regimen. Research describes the findings of these combination trials, which measured outcomes related to systemic or functional imbalance when Zidovudine was included in the regimen.

Research exploring Mother-to-Child Transmission

Research related to perinatal transmission centers on the structure of the definitive placebo-controlled RCT, historically known as ACTG 076, and subsequent comparative trials. This research measured an outcome related to HIV-1 transmission in the infant following a specific three-part regimen (antenatal, intra-partum, and post-natal).

The original research design was unable to isolate the independent contribution of the individual phases of the three-part regimen to the reported outcomes, representing a research limitation. Research describes the distribution of transmission rates when the Zidovudine regimen was compared to other combination antiretroviral regimens.

Research Landscape and Limitations

The research record for Zidovudine includes known limitations. Long-term studies are ongoing to track potential outcomes in children exposed to the drug during the perinatal transmission regimen. These studies help show what has been observed so far regarding the long-term patterns of immunologic or virologic markers within the observed populations. Additionally, dedicated data characterizing outcomes in certain subgroups, such as older adults, remain insufficient.

Research describes group patterns, not personal outcomes. Evidence highlights what is known and what is still uncertain.

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Frequently Asked Questions (FAQ)

Common questions about Zidovudine (FAQ)

Q: Is Zidovudine still used for HIV treatment today?

A: According to official product information, Zidovudine remains a core component of combination antiretroviral therapy (cART). It is typically used in multi-drug regimens for the management of HIV-1 infection.

Q: Can Zidovudine be taken by itself or does it need to be combined with other drugs?

A: Regulatory documents indicate that although Zidovudine is available as a single-entity product, it is almost universally prescribed as part of a combination regimen. Its use in combination regimens is the established approach for managing chronic viral infections.

Q: Is Zidovudine used to prevent HIV transmission from mother to baby?

A: Yes, Zidovudine is specifically indicated for use in pregnant women and neonates (newborns) to reduce the risk of maternal-fetal HIV-1 transmission. This use is documented in regulatory indications and is supported by clinical research.

Q: Is it true that Zidovudine can cause a low red blood cell count (anemia)?

A: Official product labeling notes the potential for hematologic toxicity, which includes severe anemia (a low red blood cell count) and neutropenia (a low white blood cell count). This risk is particularly noted in individuals with advanced HIV-1 disease.

Q: What are the most common side effects people experience with Zidovudine?

A: Clinical trial data from regulatory submissions show that the most common adverse reactions reported by adult patients include headache, nausea, vomiting, and malaise (a general feeling of discomfort). These effects were reported with a high frequency in studies.

Q: What is the connection between Zidovudine and muscle pain or weakness (myopathy)?

A: The official product labeling indicates that prolonged use of Zidovudine has been associated with symptomatic myopathy. This is a condition characterized by muscle pain and weakness.

Q: What is lactic acidosis and is it a possible side effect of Zidovudine?

A: Lactic acidosis is described in regulatory warnings as a severe buildup of lactic acid in the blood. Like other nucleoside analogues, Zidovudine has been associated with reports of this serious condition, which can sometimes occur alongside liver issues.

Q: Can Zidovudine treatment affect the liver?

A: Yes, regulatory warnings note the potential for Zidovudine use to be associated with liver issues. This includes severe hepatomegaly (enlargement of the liver) with steatosis (fat accumulation), which is noted as a serious adverse event.

Q: Does Zidovudine interact with common over-the-counter pain relievers?

A: Official labeling advises that the body's ability to clear Zidovudine may be reduced by medications that inhibit glucuronidation. This can potentially increase the concentration of Zidovudine in the body, and specific interactions may require caution.

Q: What are the special considerations for people with kidney problems who take Zidovudine?

A: Regulatory information states that patients with severe renal (kidney) impairment require a dosage adjustment. This is because impaired kidney function can decrease the body's clearance of Zidovudine, potentially affecting its exposure.

Q: Can people with pre-existing liver conditions use Zidovudine?

A: The official product information notes that the use of Zidovudine is not recommended for patients who have moderate or severe liver disease. This is due to a lack of sufficient data to reliably determine dosing in this specific population.

Q: What research evidence supports the use of Zidovudine in preventing perinatal HIV transmission?

A: The evidence supporting this use stems from a definitive placebo-controlled clinical trial (ACTG 076). This study demonstrated that a specific regimen of Zidovudine significantly reduced the rate of HIV-1 transmission from an infected mother to her infant.

Q: Why is Zidovudine sometimes given by injection (IV) instead of by mouth?

A: Official administration guidelines confirm that the intravenous (IV) formulation is used in specific, time-critical settings. This is primarily for administration during labor and delivery (intrapartum use) or for patients who cannot tolerate taking the medicine by mouth temporarily.

Q: Does Zidovudine interact with herbal remedies or supplements?

A: Regulatory documents explicitly prohibit the concomitant use of the herbal product St. John's Wort with Zidovudine. This restriction is in place because the herbal product may lead to undesirable changes in the metabolism of the drug.

Q: Do patients need special monitoring or blood tests while taking Zidovudine?

A: Yes, official warnings strongly recommend frequent monitoring of blood counts for patients taking Zidovudine. This is essential to detect the potential and timely development of severe anemia or neutropenia, which are key safety concerns.

Q: How is Zidovudine different from newer HIV medications?

A: Zidovudine is classified as a Nucleoside Reverse Transcriptase Inhibitor (NRTI), which is one of the original classes of antiretroviral agents. It is used as a foundational component in combination regimens to inhibit the HIV replication cycle.

Q: Is Zidovudine used in cases of potential HIV exposure, such as after a needlestick injury?

A: Official guidelines confirm that Zidovudine, as part of a combination regimen, is an option for use in certain situations of occupational or non-occupational post-exposure prophylaxis (PEP). This is an established strategy to potentially prevent infection after exposure.

Q: Does Zidovudine cause headaches, and if so, how common is this side effect?

A: Headache is confirmed in official documentation as a common side effect of Zidovudine. Clinical trial data indicates that headaches were reported with an incidence of 15% or greater in adult patients.

Q: Is nausea or vomiting a frequent side effect of Zidovudine?

A: Yes, official safety information reports that both nausea and vomiting are among the most common adverse reactions. They were reported with an incidence of 15% or greater in clinical studies involving adults.

Q: How often does Zidovudine cause a loss of body fat (lipoatrophy) in the face or limbs?

A: Loss of body fat, a condition known as lipoatrophy, has been reported in patients receiving combination antiretroviral therapy. Official warnings note that this change in fat distribution is a potential risk associated with the use of nucleoside analogues like Zidovudine.

Q: Are changes in the color of fingernails or toenails a known side effect of Zidovudine?

A: Official documents describe that changes in skin and nail pigmentation have been observed in some patients taking the medication. These can appear as bluish-brown colored bands specifically on the nails.

Q: Can Zidovudine cause blood disorders beyond anemia, such as neutropenia?

A: Yes, Zidovudine is confirmed in regulatory warnings to be associated with hematologic toxicity. This includes both severe anemia (low red blood cells) and neutropenia (a lower than normal number of white blood cells).

Q: What are the signs of a blood disorder related to Zidovudine that people should be aware of?

A: Patients should be aware of possible signs of a severe blood disorder. These symptoms may include unusual tiredness or weakness, pale skin (signs of anemia), or fever and chills (signs of neutropenia and potential infection).

Q: Is it common to feel unusually tired or weak when taking Zidovudine?

A: The feeling of malaise, which is a general sense of discomfort or unease, and fatigue are listed in official documentation as common side effects. These feelings of unusual tiredness or weakness may occur during treatment.

Q: Are there any long-term side effects associated with Zidovudine use?

A: Official safety information notes that prolonged use of Zidovudine has been associated with specific serious adverse events. The key noted long-term risk includes symptomatic myopathy, a muscle disease.

Q: Can Zidovudine treatment lead to changes in mood or cause trouble sleeping?

A: Official product information lists insomnia (trouble sleeping) as a common adverse reaction. Less frequent central nervous system effects may involve mood or mental changes.

Q: What is 'Immune Reconstitution Inflammatory Syndrome' (IRIS) and is it connected to Zidovudine?

A: Changes in the immune system, formally known as Immune Reconstitution Inflammatory Syndrome (IRIS), have been reported in patients treated with combination antiretroviral therapy. Zidovudine is a component of these regimens.

Q: Does taking Zidovudine affect a person's ability to drive or operate machinery?

A: Official documentation advises that patients should be aware of certain adverse effects. These include possible headaches, dizziness, and somnolence (sleepiness) which could potentially impact the ability to safely drive or operate machinery.

Q: If a person feels better, can they stop taking Zidovudine?

A: Patient counseling information states that individuals should not stop antiretroviral therapy without the recommendation of their health care provider. Discontinuing treatment can lead to the return and possible progression of the HIV infection.

Q: How quickly does Zidovudine start to work after beginning treatment?

A: Clinical studies show that treatment with Zidovudine, as part of a combination regimen, begins to suppress viral replication shortly after initiation. This action typically leads to measurable decreases in the viral load over the initial weeks of starting therapy.

Q: Does Zidovudine relate to the term 'HAART' (Highly Active Antiretroviral Therapy)?

A: HAART is a historical term used to describe modern combination antiretroviral therapy (cART). Zidovudine is recognized as one of the original and core components used in many of these multi-drug regimens.

Q: What happens in the body to cause the potential for Zidovudine to be toxic to muscles?

A: Official information explains that the active form of Zidovudine can interfere with the enzyme DNA polymerase gamma (gamma). Inhibition of this enzyme may disrupt the formation of mitochondrial DNA, which is the underlying mechanism linked to the potential for muscle toxicity.

Q: Can Zidovudine treatment cause problems with body fat distribution?

A: Official reports confirm that changes in body fat distribution have been reported in patients on combination antiretroviral therapy. This can involve both the loss of body fat (lipoatrophy) and the accumulation of fat (lipohypertrophy).

Q: Does Zidovudine help in treating Hepatitis C if a person has both HIV and Hepatitis C?

A: Zidovudine is not indicated for the treatment of Hepatitis C. However, warnings note that co-infected patients receiving Zidovudine along with certain Hepatitis C medications have reported an increased risk of hepatic (liver) decompensation.

Q: Is Zidovudine known to affect bone density or cause bone pain?

A: Official documents confirm that bone pain has been reported as an adverse reaction. While the medicine is not primarily associated with major skeletal outcomes, musculoskeletal effects have been noted.

Q: Does Zidovudine interact with alcohol?

A: While regulatory labeling may not explicitly detail the interaction with alcohol, it warns about other substances that can increase the risk of certain side effects. Patients are generally advised to discuss alcohol use with their healthcare provider.

Q: Is Zidovudine considered safe to use during the first trimester of pregnancy?

A: Based on available human studies and according to national guidelines, first-trimester exposure to Zidovudine is not associated with an increased risk of congenital anomalies.

Q: How does Zidovudine cross the placenta to reach the baby during pregnancy?

A: Regulatory information indicates that Zidovudine has been shown to rapidly cross the human placenta. This results in the medicine being present in the fetus, particularly during administration leading up to delivery.

Q: If a pregnant woman takes Zidovudine, will her baby still need medication after birth?

A: Yes, for the prevention of perinatal transmission, the recommended treatment regimen for the infant starts at birth and typically continues for up to six weeks. This is standard procedure, regardless of the mother’s treatment history during pregnancy.

Q: Is it possible for Zidovudine to be passed to an infant through breast milk?

A: Official reports indicate that Zidovudine is excreted into human breast milk. Although most infants' blood levels are undetectable, the drug is known to be present in the milk.

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How should Zidovudine be stored and disposed of?

How to Store and Dispose of Zidovudine

The storage of zidovudine must strictly adhere to regulatory requirements, which vary by formulation.

Storage Requirements

Zidovudine tablets, capsules, and oral solution must be stored at controlled room temperature, typically between 59°F and 77°F (15°C to 25°C). The medication must be kept in its original container and the container must remain tightly closed. The oral solution must be protected from freezing, and storage should avoid excess heat and moisture.

Formulation Constraint Requirement
Temperature Store between 59°F and 77°F (15°C to 25°C).
Environmental Protect from moisture; the oral solution must not freeze.
Container Rule Keep in the original, tightly closed container.
Stability Note (Injection) Diluted solution must be used within 8 hours at room temperature.

Child Safety and Disposal

All forms of zidovudine must be stored out of the sight and reach of children using a locked safety cap. Expired or unused medication must be disposed of by following official governmental guidelines for safe drug disposal.

Attention! Always consult to a doctor or pharmacist before using pills or medicines.

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