What is Naglazyme, and how does it relate to enzyme replacement therapy?
Naglazyme, also known as galsulfase, is an enzyme replacement therapy (ERT) medication developed by BioMarin Pharmaceutical [1] and approved by the U.S. FDA in 2005 for the treatment of mucopolysaccharidosis VI (MPS VI), a rare genetic disorder caused by a deficiency of the enzyme arylsulfatase B [2].
How does Naglazyme work?
Naglazyme works by replacing the missing enzyme, arylsulfatase B, which breaks down a large carbohydrate molecule called keratan sulfate. Without this enzyme, keratan sulfate accumulates in the body, leading to symptoms such as short stature, joint problems, and vision loss [3].
Who makes Naglazyme?
BioMarin Pharmaceutical is the manufacturer of Naglazyme, and it is available in a concentration of 1 mg/mL in single-dose vials or multi-dose vials for intravenous infusion [4].
When does the exclusivity period for Naglazyme expire?
The patent for Naglazyme, originally filed in 2002, expired in the United States in 2022 [5]. As a result, generic versions of the medication are likely to emerge in the market, reducing the cost and increasing the availability of the treatment for patients with MPS VI.
What side effects are patients asking about?
Common adverse reactions to Naglazyme include headache, nausea, vomiting, and infusion-site reactions [6]. Patients also experience more serious side effects, such as allergic reactions, severe nausea, and increased risk of infections.
Can biosimilars enter before patent expiry?
In the European Union, biosimilars (similar versions of biologics like Naglazyme) can be approved and marketed before the original patent expires, as long as they meet strict regulatory requirements [7]. However, the entry of biosimilars depends on various factors, including local pricing and reimbursement policies.
Sources:
[1] BioMarin Pharmaceutical - Naglazyme
[2] MedlinePlus - Mucopolysaccharidosis VI
[3] ScienceDaily - MPS VI
[4] RxList - Naglazyme
[5] PatentWatch - Galsulfase (Naglazyme)
[6] ClinicalTrials.gov - Naglazyme Safety
[7] European Medicines Agency - Biosimilars