Naglazyme

galsulfase

(gal-sul-fase) ,

Naglazyme

(trade name)

Classification

Therapeutic: replacement enzyme
Pharmacologic: enzymes
Pregnancy Category: B

Indications

Mucopolysaccharridosis VI (MPS IV).

Action

Replaces a deficient enzyme in MPS IV. Without replacement, glycoaminoglycans accumulate resulting in cell, organ and tissue dysfunction.

Therapeutic effects

Improved walking and stair climbing.

Pharmacokinetics

Absorption: IV administration results in complete bioavailability.
Distribution: Widely distributed.
Metabolism and Excretion: Unknown.
Half-life: 9 min (after one week of treatment, 26 min (after 24 weeks of treatment).

Time/action profile (inprove exercise parameters)

ROUTEONSETPEAKDURATION
IVunknown24 wkunknown

Contraindications/Precautions

Contraindicated in: None.
Use Cautiously in: Febrile or respiratory illness; Pediatric: Children <5 yr (safety not established); Obstetric / Lactation: Safety not established.

Adverse Reactions/Side Effects

Cardiovascular

  • malaise (most frequent)

Ear, Eye, Nose, Throat

  • conjunctivitis (most frequent)
  • cornela opacification (most frequent)
  • ear pain

Respiratory

  • dyspnea (most frequent)

Cardiovascular

  • chest pain (most frequent)
  • ↑ BP

Dermatologic

  • facial edema (most frequent)

Gastrointestinal

  • gastroenteritis (most frequent)
  • abdominal pain

Musculoskeletal

  • areflexia (most frequent)

Neurologic

  • spinal/cervical cord compression (life-threatening)

Miscellaneous

  • infusion reactions
  • rigors (most frequent)

Interactions

Drug-Drug interaction

None noted.

Route/Dosage

Intravenous (Adults and Children >5 yr) 1 mg/kg once weekly.

Availability

Solution for IV administration (diluted prior to use): 5 mg/5 mL

Nursing implications

Nursing assessment

  • Assess for infusion reactions (fever, chills/rigors, headache, rash, mild to moderate urticaria); may occur in over half of patients. May also cause nausea, vomiting, elevated BP, retrosternal pain, abdominal pain, malaise, and joint pain. Severe reactions include angioneurotic edema, hypotension, dyspnea, bronchospasm, respiratory distress, apnea. May occur as late as week 55 of treatment. Patients should receive antihistamines and antipyretics prior to administration to minimize occurrence. Slowing or temporary interruption of infusion and administration of additional antihistamines, antipyretics, and occasionally corticosteroids usually stop symptoms. Most patients are able to complete infusion. Administer subsequent infusions at a slower rate additional prophylactic antihistamines, and, if reaction is severe, prophylactic corticosteroids. If severe reaction occurs, discontinue infusion immediately and treat symptoms. Consider risk versus benefit before re-administering galsulfase.
  • Assess for sleep apnea, common in patients with MPS VI, prior to therapy. Administration of pretreatment antihistamines may increase risk. Supplemental oxygen and continuous positive airway pressure (CPAP) should be available during infusion.
  • Assess vital signs and respiratory status prior to administration. Administration should be delayed in patients with an acute febrile or respiratory illness.
  • Monitor for signs and symptoms of spinal/cervical cord compression (back pain, paralysis of limbs below level of compression, urinary and fecal incontinence). Manage symptomatically.

Potential Nursing Diagnoses

Activity intolerance (Indications)
Deficient knowledge, related to medication regimen (Patient/Family Teaching)

Implementation

  • Pretreat with antihistamines with or without antipyretics 30–60 min prior to start of infusion.
  • Intravenous Administration
  • Intermittent Infusion: Diluent: 250 mL bag of 0.9% NaClDetermine number of vials to be diluted based on patient weight; round to nearest whole vial. Remove vials from refrigerator and allow to reach room temperature. Do not use after expiration date. Do not allow vials to remain at room temperature for longer than 24 hrs prior to dilution. Do not heat or microwave vials. Solution is clear to slightly opalescent and colorless to pale yellow; a few translucent particles may be present. Do not administer solutions that are discolored or contain particulate matter. Withdraw and discard amount equal to galsulfase from a 250 mL bag of 0.9% NaCl (not necessary if using 100 mL bag in patients susceptible to fluid volume overload). Slowly withdraw calculated volume from galsulfase vials using caution to avoid excessive agitation, may denature and render inactive. Do not use a filter needle. Slowly add galsulfase to 0.9% NaCl; avoid agitation. Gently rotate infusion bag to ensure distribution; do not shake. Discard unused portions. Use diluted solution immediately; storage should not exceed 48 hr from time of preparation to completion of administration. Use PVC containers and administer via PVC infusion set with an in-line low protein binding 0.2 micrometer filter.
  • Rate: Administer at a rate of 6 mL/hr for first hour. If infusion is well tolerated, rate may be increased to 80 mL/hr for next 3 hr. Administer total volume of infusion over no less than 4 hr. If 100 mL bag is used, decrease infusion rate so total volume is infused over at least 4 hrs. Infusion time can be extended for up to 20 hr if infusion reactions occur.
  • Additive Incompatibility: Do not admix or administer with other products.

Patient/Family Teaching

  • Inform patient that a Clinical Surveillance Program has been established to better understand the variability and progression of MPS VI and to evaluate long term effects of galsulfase. Encourage patients to participate; participation is voluntary and may be long term. For information visit www.MPSVI.com/CSP or call 866–906–6100.

Evaluation/Desired Outcomes

  • Improved walking and stair climbing capacity in patients with MPS VI.

Naglazyme

a trademark for galsulfase.
References in periodicals archive ?
serves as an example of a manufacturer that sells some of the most expensive drugs for extremely rare diseases, including the $350,000-ayear enzyme replacement therapy Naglazyme.
Approved products include Naglazyme (galsulfase) for mucopolysaccharidosis VI, Kuvan (sapropterin dihydrochloride) for phenylketonuria, Aldurazyme (laronidase) for mucopolysaccharidosis I and Firdapse (amifampridine phosphate) for the treatment of Lambert Eaton Myasthenic Syndrome.
The portfolio in question includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of mucopolysaccharidosis VI (MPS VI), which expire between 2022 and 2023.
The company markets Kuvan (sapropterin dihydrochloride) for phenylketonuria, Naglazyme (galsulfase) for mucopolysaccharidosis VI and Aldurazyme (laronidase), with partner Genzyme Corp.
TSE: 4563) announced that on April 14 that the company launched Naglazyme (galsulfase) for patients with the genetic disease mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome).
BPPS currently provides support for MPS VI patients treated with Naglazyme, and a similar support program will be available for PKU patients.
lt;4563> announced August 13 that it submitted a Biologics License Application for Naglazyme (galsulfase) to the Ministry of Health, Labour and Welfare in Japan.
BioMarin modeled its GALNS program after Aldurazyme and Naglazyme, which BioMarin successfully developed in the last 10 years.
Analysts say that the firms other major drug, Naglazyme, would fetch $307.
This acquisition of the intellectual property includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of MPS VI, which expire between 2022 and 2023.
The multi-product manufacturing plant in Novato was first licensed for production of Aldurazyme for MPS I in 2003, then Naglazyme for MPS VI in 2005.
Naglazyme is a normal variant form of arylsulfatase B IUPAC name N-acetylgalactosamin 4-sulfatase) that is lacking in mucopolysaccharidosis VI patients.