Rare Disease

Corticosteroids are often given as therapy for neurosarcoidosis, but there remains a need for more effective treatment, and infliximab is increasingly used in this context.
Finnish-based biosimilar developer, Paras Biopharmaceuticals Finland Oy, said this week that it is ready to begin testing its potential romiplostim biosimilar in clinical trials.
JCR Pharmaceuticals announced yesterday that it has launched its biosimilar agalsidase beta product, referencing Fabrazyme, in Japan for the treatment of Fabry disease. JCR developed the drug in partnership with Amicus Therapeutics and GlaxoSmithKline.
In recent years, spending on specialty drugs, including spending on a proliferation of orphan drugs that treat rare diseases, has raised concerns about sustainability of the healthcare system. 
Pemphigus vulgaris (PV) is a rare immune-mediated skin disorder, typically occurring in middle-aged and older adults, that involves painful blistering of the skin and mucous membranes. While many patients are well controlled on systemic corticosteroid treatment, steroid-sparing therapy can be desirable for treating moderate to severe forms of the disease, and rituximab is one such agent to show promise in this indication.
Drug maker Alexion says that it is preparing for regulatory submissions in the United States, the European Union, and Japan for eculizumab (Soliris) for the treatment of anti-aquaporin-4 auto antibody-positive neuromyelitis optica spectrum disorder.
IgG4-related disease (IgG4-RD), a systemic disease that can affect any part of the body and can involve fibrosis, irreversible organ damage, and secondary amyloidosis, is a rare immune-mediated condition often treated with corticosteroids or immunosuppressive drugs. The pathogenesis of the disease is not clear, but treatment with B-cell depletion therapy, in the form of rituximab, has shown positive results in some patients.
Agalsidase beta (Fabrazyme), the first Fabry disease-specific therapy to be approved by the FDA, effectively reduces globotriaosylceramide deposits. However, the drug comes with a high price tag of approximately $300,000 per patient per year, and has been subject to shortages due to manufacturing issues.
Alexion’s existing C5 complement inhibitor, targeted by biosimilar developers, is used to treat patients with paroxysmal nocturnal hemoglobinuria, but the biologic must be administered every 2 weeks versus ALXN1210’s proposed 8-week intravenous dosing schedule.
Antibodies that deplete B cells, including rituximab, have demonstrated efficacy in the treatment of neurological conditions such as progressive multiple sclerosis and neuromyelitis optica spectrum disorder. The efficacy of these treatments hinges on adequate B-cell depletion, but there is a lack of standardization in treatment and monitoring protocols to guide clinical practice.

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