Rare Disease

Patients With Pemphigus Vulgaris May Benefit From Early Rituximab

October 14th 2018

By The Center for Biosimilars Staff

Article

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.

Alexion to Seek Fourth Indication for Eculizumab Ahead of Biosimilar Competition

October 9th 2018

By Kelly Davio

Article

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.

Rituximab May Hold Promise in Treating IgG4-RD

September 3rd 2018

By Kelly Davio

Article

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.

JR-051 Shows High Degree of Similarity to Rare Disease Drug Fabrazyme

August 23rd 2018

By Kelly Davio

Article

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.

With Eculizumab Biosimilars Closing in, Alexion Granted Priority Review for ALXN1210

August 22nd 2018

By Kelly Davio

Article

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.

Monitoring B-Cell Recovery Is Key in Treating Neurological Diseases With Rituximab

May 9th 2018

By Kelly Davio

Article

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.

Alexion Announces Positive Phase 3 Data for Long-Acting C5 Complement Inhibitor

May 1st 2018

By Samantha DiGrande

Article

Drug manufacturer Alexion has announced positive topline results of a phase 3 study that determined that ALXN1210, a long-acting C5 complement inhibitor, can be safely and effectively switched among patients with paroxysmal nocturnal hemoglobinuria currently being treated with the shorter-acting eculizumab (Soliris).

Bevacizumab May Have Antitumor Activity in Adult Pilocytic Astrocytoma

April 24th 2018

By Samantha DiGrande

Article

A case series was recently published in CNS Oncology that investigated whether adult pilocytic astrocytoma—a rare and highly vascular tumor—is responsive to bevacizumab treatment.

New Anti-C5 Agents on the Way as Biosimilar Competition for Eculizumab Nears

March 16th 2018

By Kelly Davio

Article

Biosimilars of eculizumab, which have the potential to reduce costs for both patients and the healthcare system, have been eagerly awaited, yet many drug makers are developing novel anti-C5 agents.

Phase 1 Clinical Trial of Dornase Alfa Biosimilar to Begin March 2018

March 5th 2018

By The Center for Biosimilars Staff

Article

The proposed biosimilar references Genentech’s Pulmozyme, a biologic first approved in the early 1990s in both the European Union and the United States. Pulmozyme is used in conjunction with other therapies to improve pulmonary function in patients with cystic fibrosis.