elosulfase alfa

(redirected from Vimizim)

elosulfase alfa

(el-oh-sul-fase al-fa),

Vimizim

(trade name)

Classification

Therapeutic: orphan drugs
Pharmacologic: enzymes

Pregnancy Category: C

Indications

Treatment of patients with Mucopolysaccharidosis type IVA (MPS IVA, Morquio A Syndrome).

Action

Consists of an enzyme produced by recombinant DNA technology that hydrolyzes sulfate from galactose-6–sulfate or N-acetyl-galactosamine-6–sulfate. Lack of this enzyme in Mucoplysaccharidosis results in intracellular accumulation of glycosaminoglycans (GAG) producing widespread cellular, tissue and organ dysfunction.

Therapeutic effects

Decreased accumulation of GAG, decreased end organ dysfunction and improved musculoskeletal performance MPS IVA.

Pharmacokinetics

Absorption: IV administration results in complete bioavailability.

Distribution: Taken up by cells.

Metabolism and Excretion: Unknown.

Half-life: Initially—7 min ; after prolonged treatment—35.9 min (due to neutralizing antibodies).

Time/action profile (blood levels)

ROUTE	ONSET	PEAK	DURATION
IV	unknown†	end of infusion	unknown

†Improvement in musculoskeletal function may take wk-mos.

Contraindications/Precautions

Contraindicated in: None noted.

Use Cautiously in: Concurrent acute febrile/respiratory illness (↑ risk of life threatening complications of hypersensitivity reaction); Sleep apnea (supportive measures should be available during infusion); Obstetric: Use during pregnancy only if potential benefit justifies potential risk to fetus; Consider maternal need for elosulfase alfa and potential adverse effects to infant; Pediatric: Safe and effective use in children <5 yr has not been established.

Adverse Reactions/Side Effects

Central nervous system

fatigue (most frequent)
headache (most frequent)

Gastrointestinal

abdominal pain (most frequent)
nausea (most frequent)
vomiting (most frequent)

Miscellaneous

chills (most frequent)
fever (most frequent)
hypersensitivity reactions including anaphylaxis (life-threatening)

Interactions

Drug-Drug interaction

None noted.

Route/Dosage

Intravenous (Adults and Children ≥5 yr) 2 mg/kg once weekly; pretreatment with antihistamines with/without antipyretics recommended.

Availability

Solution for intravenous use (requires further dilution): 5 mg/5ml single-use vial

Nursing implications

Nursing assessment

Monitor for signs and symptoms of anaphylaxis (cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, nausea, abdominal pain, retching, vomiting). Discontinue infusion and manage symptomatically. May occur 30 min to 3 hr after start of infusion and as late as 47th infusion.
Evaluate airway patency and risk of sleep apnea. Provide supplemental oxygen or continuous positive airway pressure (CPAP) during sleep and infusion in case of acute reaction or extreme sleepiness induced by antihistamine.

Potential Nursing Diagnoses

Deficient knowledge, related to medication regimen (Indications)

Implementation

Pre-treat with antihistamines, with or without antipyretics, 30–60 min prior to start of infusion.
Intermittent Infusion: Determine number of vials required. Diluent: Dilute with 0.9% NaCl to a final volume of 100 mL for patients weighing less than 25 kg or 250 mL for patients weighing 25 kg or more. Solution is clear to slightly opalescent and colorless to pale yellow; do not administer solutions that are discolored or contain particulate matter. Gently rotate to mix; do not shake or agitate. Use diluted solution immediately or refrigerate up to 24 hr followed by up to 24 hr at room temperature during infusion.
Rate: Infuse over at least 3.5 (100 mL) or 4.5 hr (250 mL) through a low-protein binding infusion set with a low-protein binding 0.2 micron in-line filter.

Patient/Family Teaching

Explain the purpose of elosulfate alfa to patient.
Advise patient to notify health care professional immediately if signs and symptoms of anaphylaxis occur.
Advise female patients to notify health care professional if pregnancy is planned or suspected or if breastfeeding. Encourage patient to enroll in Morquio A Registry to collect data on pregnant women with MPS IVA treated with Vimizim by contacting MARS@bmrn.com or call 1-800-983-4587.

Evaluation/Desired Outcomes

Decreased end organ dysfunction and improved musculoskeletal performance.

References in periodicals archive ?

* Vimizim Net Product Revenues decreased by $4.9 million, or 4%, primarily due to decreased sales volume driven by government ordering patterns in certain Latin American, Middle Eastern and European countries.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN)

Clinical review report: elosulfase alfa (Vimizim) (2 mg/kg of body weight) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (Morquio A syndrome, or MPS IVA) [Internet].

Errata for trial publications are not uncommon, are frequently not trivial, and can be challenging to access: a retrospective review

In July 2014, BioMarin received USD67.5 m from Regeneron Ireland, an indirect, wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc., in exchange for a voucher awarded when it received approval of Vimizim for patients with the rare disease, Mucopolysaccharidosis type IVA, also known as Morquio A syndrome.

BioMarin Reaches Agreement to Sell Priority Review Voucher Received with Approval of Brineura for USD 125m

It costs [euro]400,000 a year for Vimizim which helps young Cezara Focsa cope with her rare genetic condition Morquio - but surely a price can't be put on a human life?

It's time to get priorities right; VOICE OF THE

She needs Vimizim which costs [euro]400,000 a year but the HSE is still deciding whether to make it available here.

Therapy 'best gift' for Grace

Previously, there was no cure for the condition, but now a drug called Vimizim as the potential to alter the course of the disease.

New rare disease drug on NHS

Previously, there was no cure for the condition, but now a drug called Vimizim has the potential to alter the course of the disease.

Drug to treat life-limiting disease available on NHS

The parents of Gracie Mellalieu have spoken of their relief after the Welsh Government announced it would fund Vimizim, which they say will extend their daughter's life.

Gracie's joy over family's wonder drug victory; 9-YR-OLD TO GET TREATMENT ON NHS AFTER 2-YEAR BATTLE

Acthar, for Multiple Sclerosis; Soliris, for PNH Disorder; and Vimizim for Progressive Metabolic Disorder.

Specialty drugs: A primer

M2 EQUITYBITES-November 24, 2015-BioMarin Pharmaceutical receives recommendation in England for Morquio A Syndrome patients' access to Vimizim treatment

BioMarin Pharmaceutical receives recommendation in England for Morquio A Syndrome patients' access to Vimizim treatment

There are six new drugs belonging to the endocrine/metabolic pharmacologic class: albiglutide (Tanzeum), dapagliflozin (Farxiga), dulaglutide (Trulicity), eliglustat (Cerdelga), elosulfase alfa (Vimizim), and empagliflozin (Jardiance).