Galsulfase, also known as recombinant human N-acetylgalactosamine-4-sulfatase, is a medication used in enzyme replacement therapy for the treatment of mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome. MPS VI is a rare genetic disorder caused by deficiency of the enzyme arylsulfatase B (ASB), which leads to the accumulation of glycosaminoglycans (GAGs) within cells, leading to various symptoms and organ damage.

Galsulfase works by replacing the deficient enzyme ASB in patients with MPS VI. By providing the missing enzyme, galsulfase helps to break down the accumulated GAGs, reducing their buildup in tissues and organs and thereby slowing the progression of the disease.

Galsulfase is typically administered through intravenous infusion and is given on a regular basis, often weekly, under the supervision of a healthcare professional. Treatment with galsulfase aims to alleviate symptoms, improve quality of life, and potentially slow the progression of MPS VI, although it does not cure the underlying genetic disorder.

Naglazyme (Galsulfase) is a recombinant human N-acetylgalactosamine-4 sulfatase. It is used as an enzyme replacement therapy available as an injection for the treatment of mucopolysacharidosis.

Naglazyme (Galsulfase) Uses:

• Mucopolysaccharidosis VI:
  • It is used as a Replacement therapy in patients with mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome) to improve walking and stair-climbing capacity.

Naglazyme (Galsulfase) Dose in Adults

Note: it should be premeditated 30 to 60 minutes prior to start the infusion with antihistamines with/without antipyretics.

Naglazyme (Galsulfase) Dose in the treatment of Mucopolysaccharidosis VI (MPS VI):

• In treating Mucopolysaccharidosis VI (MPS VI), the typical dose of Galsulfase given through an IV is 1 milligram per kilogram of body weight.
• This dose is usually administered once a week.

Naglazyme (Galsulfase) Dose in Children

Naglazyme (Galsulfase) Dose in the treatment of Mucopolysaccharidosis (MPS) VI:

In treating Mucopolysaccharidosis VI (MPS VI) with Galsulfase, the recommended doses are as follows:

• For infants (limited data available): Intravenously (IV), the dose is either 1 or 2 milligrams per kilogram of body weight per dose, given once weekly. This dosing has been used in infants aged 3 to 12 months, with similar safety results to older patients.
• For children and adolescents: The recommended dose is 1 milligram per kilogram of body weight per dose, administered intravenously once weekly.

Note: It's advised to premedicate with antihistamines with or without antipyretics 30 to 60 minutes prior to the infusion.

Pregnancy Risk Factor B

• Galsulfase is categorized as Pregnancy Risk Factor B, indicating that adverse events have not been observed in animal reproduction studies.
• However, there's a pregnancy registry available for women who may be exposed to galsulfase during pregnancy for the treatment of MPS VI.
• This registry allows healthcare providers to collect information on pregnancies exposed to galsulfase and monitor outcomes.

Galsulfase use during breastfeeding:

• Galsulfase's excretion into breast milk is not known.
• Therefore, caution is recommended if administered to breastfeeding women.
• The pregnancy registry also collects data from women who are breastfeeding while using galsulfase for the treatment of MPS VI.
• This allows healthcare providers to gather information on breastfeeding women's experiences with the medication and monitor any potential effects on infants.

Naglazyme Dose in Kidney Disease:

• According to the manufacturer's labeling, there are no specific dosage adjustments recommended for individuals with renal impairment when using galsulfase for the treatment of Mucopolysaccharidosis VI (MPS VI).
• This implies that the standard dosage is typically used regardless of renal function.

Naglazyme Dose in Liver disease:

• According to the manufacturer's labeling, there are no specific dosage adjustments recommended for individuals with hepatic impairment when using galsulfase for the treatment of Mucopolysaccharidosis VI (MPS VI).
• This suggests that the standard dosage is typically used irrespective of hepatic function.

---

Note:

• The Percentages reported are from a placebo-controlled study (39 patients, 19 on galsulfase).
• It also include the adverse effects that are noted during other clinical studies.

Side effects of Naglazyme (Galsulfase):

• Cardiovascular:
  • Chest Pain
  • Hypertension
• Central Nervous System:
  • Pain
  • Chills
  • Absent Reflexes
  • Malaise
  • Headache
• Dermatologic:
  • Skin Rash
  • Pruritus
  • Urticaria
• Gastrointestinal:
  • Abdominal Pain
  • Gastroenteritis
  • Nausea
  • Vomiting
• Hypersensitivity:
  • Angioedema
• Neuromuscular & Skeletal:
  • Arthralgia
• Ophthalmic:
  • Conjunctivitis
  • Corneal Opacity
• Otic:
  • Otalgia
  • Auditory Impairment
• Respiratory:
  • Dyspnea
  • Pharyngitis
  • Nasal Congestion
  • Apnea
  • Laryngeal Edema
  • Respiratory Distress
• Miscellaneous:
  • Antibody Development
  • Infusion Related Reaction
  • Umbilical Hernia
  • Fever

Contraindications to Naglazyme (Galsulfase):

• According to the manufacturer's labeling, there are no contraindications listed for the use of galsulfase in the treatment of Mucopolysaccharidosis VI (MPS VI).
• This means that there are no specific circumstances in which the use of galsulfase is strictly prohibited based on medical or safety reasons.

Warnings and precautions

Hypersensitivity

• Some people may have severe allergic reactions to galsulfase, which can include symptoms like trouble breathing, swelling of the throat, shock, or low blood pressure.
• These reactions can happen during or up to 24 hours after receiving the medication.
• It's essential to have immediate treatment available during the infusion in case of a reaction.
• If signs of a severe allergic reaction occur, treatment should be stopped right away.
• If there's a need to restart treatment later, it should be done with caution.

Reactions that are immune-mediated

• Some patients may experience severe immune-mediated reactions, such as membranous glomerulonephritis, as a result of treatment with galsulfase.
• It's important to closely monitor patients for any signs or symptoms of these reactions.
• If such symptoms occur, discontinuation of treatment should be considered.
• If treatment needs to be resumed later, it should be done cautiously, with close supervision.
• While some patients have been successfully re-challenged with galsulfase, it's crucial to approach this with caution.

Infusion reactions

• Infusion-related reactions are commonly reported with galsulfase treatment and can range from mild to severe.
• Serious reactions may include swelling of the throat, difficulty breathing, chest pain, rash, fever, and nausea, among others.
• These reactions can occur during or after the infusion, sometimes even after several weeks of treatment.
• Patients are typically given antihistamines and/or antipyretics before the infusion to help prevent these reactions.
• If a reaction occurs, the infusion may be slowed or temporarily stopped, and additional medications like corticosteroids may be given.
• Most patients are able to complete their infusions with these interventions.
• For subsequent infusions, precautions such as slower administration rates and additional medications may be taken to prevent further reactions.
• If a severe reaction happens, treatment should be stopped immediately, and the decision to restart treatment should be carefully considered based on the risks and benefits.

Acute febrile/respiratory disease:

• If a patient has an acute fever or respiratory illness, it's advisable to consider postponing their galsulfase treatment.
• This precaution is taken to ensure the patient's safety and to minimize any potential complications or exacerbation of their existing condition.

Fluid overload is a risk to patients

• Galsulfase should be used cautiously in patients who are at risk of fluid overload, such as those weighing less than or equal to 20 kilograms, individuals with underlying respiratory disease, or those with compromised heart and lung function.
• This caution is necessary because galsulfase treatment may potentially lead to heart failure in such patients.
• Close monitoring of these individuals is essential to detect any signs or symptoms of fluid overload or heart failure promptly.

Sleep Apnea

• Patients receiving galsulfase treatment should be monitored for signs and symptoms of spinal or cervical cord compression (SCC), as worsening or new-onset SCC has been reported.
• SCC, which can lead to myelopathy, is a serious complication associated with MPS VI.
• Signs and symptoms of SCC may include back pain, paralysis in the limbs below the level of compression, and urinary or fecal incontinence.
• If any of these symptoms occur, patients should receive appropriate clinical care promptly.

Compression of cervical/spinal cords

• In patients with sleep apnea, using galsulfase requires caution because sleep apnea is common among those with MPS VI.
• Pre-treating with antihistamines may heighten the risk of apneic episodes in these individuals.
• Before beginning treatment, it's crucial to assess the airway to ensure it stays clear during the infusion.
• Also, have measures in place to manage apnea during the infusion and when using antihistamine premedication.

Monitoring parameters:

Monitor for Infusion/Hypersensitivity Reactions:

• Keep an eye out for any reactions during or after the infusion.
• Be alert for symptoms like difficulty breathing, swelling, chest pain, rash, fever, or nausea.
• If a reaction occurs, treatment may need to be slowed or temporarily stopped, and additional medications may be given.
• Most patients can continue their treatment with proper management of reactions.

Signs and Symptoms of Spinal/Cervical Cord Compression:

• Watch for signs such as back pain, paralysis in limbs below the compression level, or urinary and fecal incontinence.
• Spinal or cervical cord compression can worsen or develop as a serious complication of MPS VI.
• Promptly provide appropriate clinical care if any symptoms of spinal/cervical cord compression are observed.

How to administer Naglazyme (Galsulfase)?

Infusion Guidelines:

• Administer galsulfase intravenously (IV) over at least 4 hours.
• For infusions totaling 250 mL, start at a rate of 6 mL/hour for the first hour. If tolerated, increase the rate to 80 mL/hour for the remaining 3 hours.
• If infusion reactions occur, extend the infusion time up to 20 hours.
• For patients needing fluid restriction, use 100 mL solutions and adjust the rate to infuse over at least 4 hours.
• Use an infusion pump and PVC (low protein-binding) infusion set with an in-line low protein-binding 0.2 micrometer filter.

Premedication:

• Administer antihistamines (with or without antipyretics) 30 to 60 minutes before the infusion.
• If infusion-related reactions occur, reduce the infusion rate or temporarily stop the infusion.
• Immediately discontinue treatment if a severe reaction occurs.

Special Considerations:

• Patients needing supplemental oxygen or continuous positive airway pressure (CPAP) during sleep should have these treatments readily available in case of infusion-related or antihistamine-induced reactions.

Mechanism of action of Naglazyme (Galsulfase):

• Galsulfase is a man-made version of the enzyme N-acetylgalactosamine 4-sulfatase, created using Chinese hamster cells.
• When there's not enough of this enzyme in the body, a substance called glycosaminoglycan dermatan sulfate builds up in tissues, leading to a range of health problems.
• These include slowed growth, bone deformities, breathing difficulties due to airway blockages, cloudy corneas, heart issues, and distinctive facial features.
• Giving patients galsulfase helps replace the missing enzyme, which has been shown to improve mobility and physical abilities, like walking and climbing stairs, in those affected by the condition.

Patient Population and Data:

• The data provided are based on a mixed patient population, including children aged 5 years and older and adults under 29 years of age.

Distribution:

• Week 1: The volume of distribution (V) is 103 milliliters per kilogram (mL/kg) with a range of 56 to 323 mL/kg.
• Week 24: The volume of distribution (V) is 69 mL/kg with a range of 59 to 2,799 mL/kg.

Half-Life Elimination:

• Week 1: The median half-life elimination is 9 minutes with a range of 6 to 21 minutes.
• Week 24: The median half-life elimination is 26 minutes with a range of 8 to 40 minutes.

These values provide insight into how the medication is distributed in the body and how quickly it is eliminated over time.

International Brands of Galsulfase:

• Naglazyme
• Naglazyzme

Galsulfase Brand Names in Pakistan:

You can check for availability and prices by clicking the WhatsApp link here: https://wa.me/message/HV6M3JGTA6RJB1

Galsulfase, also known as recombinant human N-acetylgalactosamine-4-sulfatase, is a medication used in enzyme replacement therapy for the treatment of mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome. MPS VI is a rare genetic disorder caused by deficiency of the enzyme arylsulfatase B (ASB), which leads to the accumulation of glycosaminoglycans (GAGs) within cells, leading to various symptoms and organ damage.

Galsulfase works by replacing the deficient enzyme ASB in patients with MPS VI. By providing the missing enzyme, galsulfase helps to break down the accumulated GAGs, reducing their buildup in tissues and organs and thereby slowing the progression of the disease.

Galsulfase is typically administered through intravenous infusion and is given on a regular basis, often weekly, under the supervision of a healthcare professional. Treatment with galsulfase aims to alleviate symptoms, improve quality of life, and potentially slow the progression of MPS VI, although it does not cure the underlying genetic disorder.

Naglazyme (Galsulfase) is a recombinant human N-acetylgalactosamine-4 sulfatase. It is used as an enzyme replacement therapy available as an injection for the treatment of mucopolysacharidosis.

Naglazyme (Galsulfase) Uses:

• Mucopolysaccharidosis VI:
  • It is used as a Replacement therapy in patients with mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome) to improve walking and stair-climbing capacity.

Naglazyme (Galsulfase) Dose in Adults

Note: it should be premeditated 30 to 60 minutes prior to start the infusion with antihistamines with/without antipyretics.

Naglazyme (Galsulfase) Dose in the treatment of Mucopolysaccharidosis VI (MPS VI):

• In treating Mucopolysaccharidosis VI (MPS VI), the typical dose of Galsulfase given through an IV is 1 milligram per kilogram of body weight.
• This dose is usually administered once a week.

Naglazyme (Galsulfase) Dose in Children

Naglazyme (Galsulfase) Dose in the treatment of Mucopolysaccharidosis (MPS) VI:

In treating Mucopolysaccharidosis VI (MPS VI) with Galsulfase, the recommended doses are as follows:

• For infants (limited data available): Intravenously (IV), the dose is either 1 or 2 milligrams per kilogram of body weight per dose, given once weekly. This dosing has been used in infants aged 3 to 12 months, with similar safety results to older patients.
• For children and adolescents: The recommended dose is 1 milligram per kilogram of body weight per dose, administered intravenously once weekly.

Note: It's advised to premedicate with antihistamines with or without antipyretics 30 to 60 minutes prior to the infusion.

Pregnancy Risk Factor B

• Galsulfase is categorized as Pregnancy Risk Factor B, indicating that adverse events have not been observed in animal reproduction studies.
• However, there's a pregnancy registry available for women who may be exposed to galsulfase during pregnancy for the treatment of MPS VI.
• This registry allows healthcare providers to collect information on pregnancies exposed to galsulfase and monitor outcomes.

Galsulfase use during breastfeeding:

• Galsulfase's excretion into breast milk is not known.
• Therefore, caution is recommended if administered to breastfeeding women.
• The pregnancy registry also collects data from women who are breastfeeding while using galsulfase for the treatment of MPS VI.
• This allows healthcare providers to gather information on breastfeeding women's experiences with the medication and monitor any potential effects on infants.

Naglazyme Dose in Kidney Disease:

• According to the manufacturer's labeling, there are no specific dosage adjustments recommended for individuals with renal impairment when using galsulfase for the treatment of Mucopolysaccharidosis VI (MPS VI).
• This implies that the standard dosage is typically used regardless of renal function.

Naglazyme Dose in Liver disease:

• According to the manufacturer's labeling, there are no specific dosage adjustments recommended for individuals with hepatic impairment when using galsulfase for the treatment of Mucopolysaccharidosis VI (MPS VI).
• This suggests that the standard dosage is typically used irrespective of hepatic function.

---

Note:

• The Percentages reported are from a placebo-controlled study (39 patients, 19 on galsulfase).
• It also include the adverse effects that are noted during other clinical studies.

Side effects of Naglazyme (Galsulfase):

• Cardiovascular:
  • Chest Pain
  • Hypertension
• Central Nervous System:
  • Pain
  • Chills
  • Absent Reflexes
  • Malaise
  • Headache
• Dermatologic:
  • Skin Rash
  • Pruritus
  • Urticaria
• Gastrointestinal:
  • Abdominal Pain
  • Gastroenteritis
  • Nausea
  • Vomiting
• Hypersensitivity:
  • Angioedema
• Neuromuscular & Skeletal:
  • Arthralgia
• Ophthalmic:
  • Conjunctivitis
  • Corneal Opacity
• Otic:
  • Otalgia
  • Auditory Impairment
• Respiratory:
  • Dyspnea
  • Pharyngitis
  • Nasal Congestion
  • Apnea
  • Laryngeal Edema
  • Respiratory Distress
• Miscellaneous:
  • Antibody Development
  • Infusion Related Reaction
  • Umbilical Hernia
  • Fever

Contraindications to Naglazyme (Galsulfase):

• According to the manufacturer's labeling, there are no contraindications listed for the use of galsulfase in the treatment of Mucopolysaccharidosis VI (MPS VI).
• This means that there are no specific circumstances in which the use of galsulfase is strictly prohibited based on medical or safety reasons.

Warnings and precautions

Hypersensitivity

• Some people may have severe allergic reactions to galsulfase, which can include symptoms like trouble breathing, swelling of the throat, shock, or low blood pressure.
• These reactions can happen during or up to 24 hours after receiving the medication.
• It's essential to have immediate treatment available during the infusion in case of a reaction.
• If signs of a severe allergic reaction occur, treatment should be stopped right away.
• If there's a need to restart treatment later, it should be done with caution.

Reactions that are immune-mediated

• Some patients may experience severe immune-mediated reactions, such as membranous glomerulonephritis, as a result of treatment with galsulfase.
• It's important to closely monitor patients for any signs or symptoms of these reactions.
• If such symptoms occur, discontinuation of treatment should be considered.
• If treatment needs to be resumed later, it should be done cautiously, with close supervision.
• While some patients have been successfully re-challenged with galsulfase, it's crucial to approach this with caution.

Infusion reactions

• Infusion-related reactions are commonly reported with galsulfase treatment and can range from mild to severe.
• Serious reactions may include swelling of the throat, difficulty breathing, chest pain, rash, fever, and nausea, among others.
• These reactions can occur during or after the infusion, sometimes even after several weeks of treatment.
• Patients are typically given antihistamines and/or antipyretics before the infusion to help prevent these reactions.
• If a reaction occurs, the infusion may be slowed or temporarily stopped, and additional medications like corticosteroids may be given.
• Most patients are able to complete their infusions with these interventions.
• For subsequent infusions, precautions such as slower administration rates and additional medications may be taken to prevent further reactions.
• If a severe reaction happens, treatment should be stopped immediately, and the decision to restart treatment should be carefully considered based on the risks and benefits.

Acute febrile/respiratory disease:

• If a patient has an acute fever or respiratory illness, it's advisable to consider postponing their galsulfase treatment.
• This precaution is taken to ensure the patient's safety and to minimize any potential complications or exacerbation of their existing condition.

Fluid overload is a risk to patients

• Galsulfase should be used cautiously in patients who are at risk of fluid overload, such as those weighing less than or equal to 20 kilograms, individuals with underlying respiratory disease, or those with compromised heart and lung function.
• This caution is necessary because galsulfase treatment may potentially lead to heart failure in such patients.
• Close monitoring of these individuals is essential to detect any signs or symptoms of fluid overload or heart failure promptly.

Sleep Apnea

• Patients receiving galsulfase treatment should be monitored for signs and symptoms of spinal or cervical cord compression (SCC), as worsening or new-onset SCC has been reported.
• SCC, which can lead to myelopathy, is a serious complication associated with MPS VI.
• Signs and symptoms of SCC may include back pain, paralysis in the limbs below the level of compression, and urinary or fecal incontinence.
• If any of these symptoms occur, patients should receive appropriate clinical care promptly.

Compression of cervical/spinal cords

• In patients with sleep apnea, using galsulfase requires caution because sleep apnea is common among those with MPS VI.
• Pre-treating with antihistamines may heighten the risk of apneic episodes in these individuals.
• Before beginning treatment, it's crucial to assess the airway to ensure it stays clear during the infusion.
• Also, have measures in place to manage apnea during the infusion and when using antihistamine premedication.

Monitoring parameters:

Monitor for Infusion/Hypersensitivity Reactions:

• Keep an eye out for any reactions during or after the infusion.
• Be alert for symptoms like difficulty breathing, swelling, chest pain, rash, fever, or nausea.
• If a reaction occurs, treatment may need to be slowed or temporarily stopped, and additional medications may be given.
• Most patients can continue their treatment with proper management of reactions.

Signs and Symptoms of Spinal/Cervical Cord Compression:

• Watch for signs such as back pain, paralysis in limbs below the compression level, or urinary and fecal incontinence.
• Spinal or cervical cord compression can worsen or develop as a serious complication of MPS VI.
• Promptly provide appropriate clinical care if any symptoms of spinal/cervical cord compression are observed.

How to administer Naglazyme (Galsulfase)?

Infusion Guidelines:

• Administer galsulfase intravenously (IV) over at least 4 hours.
• For infusions totaling 250 mL, start at a rate of 6 mL/hour for the first hour. If tolerated, increase the rate to 80 mL/hour for the remaining 3 hours.
• If infusion reactions occur, extend the infusion time up to 20 hours.
• For patients needing fluid restriction, use 100 mL solutions and adjust the rate to infuse over at least 4 hours.
• Use an infusion pump and PVC (low protein-binding) infusion set with an in-line low protein-binding 0.2 micrometer filter.

Premedication:

• Administer antihistamines (with or without antipyretics) 30 to 60 minutes before the infusion.
• If infusion-related reactions occur, reduce the infusion rate or temporarily stop the infusion.
• Immediately discontinue treatment if a severe reaction occurs.

Special Considerations:

• Patients needing supplemental oxygen or continuous positive airway pressure (CPAP) during sleep should have these treatments readily available in case of infusion-related or antihistamine-induced reactions.

Mechanism of action of Naglazyme (Galsulfase):

• Galsulfase is a man-made version of the enzyme N-acetylgalactosamine 4-sulfatase, created using Chinese hamster cells.
• When there's not enough of this enzyme in the body, a substance called glycosaminoglycan dermatan sulfate builds up in tissues, leading to a range of health problems.
• These include slowed growth, bone deformities, breathing difficulties due to airway blockages, cloudy corneas, heart issues, and distinctive facial features.
• Giving patients galsulfase helps replace the missing enzyme, which has been shown to improve mobility and physical abilities, like walking and climbing stairs, in those affected by the condition.

Patient Population and Data:

• The data provided are based on a mixed patient population, including children aged 5 years and older and adults under 29 years of age.

Distribution:

• Week 1: The volume of distribution (V) is 103 milliliters per kilogram (mL/kg) with a range of 56 to 323 mL/kg.
• Week 24: The volume of distribution (V) is 69 mL/kg with a range of 59 to 2,799 mL/kg.

Half-Life Elimination:

• Week 1: The median half-life elimination is 9 minutes with a range of 6 to 21 minutes.
• Week 24: The median half-life elimination is 26 minutes with a range of 8 to 40 minutes.

These values provide insight into how the medication is distributed in the body and how quickly it is eliminated over time.

International Brands of Galsulfase:

• Naglazyme
• Naglazyzme

Galsulfase Brand Names in Pakistan:

Not available.