Rare Disease

A small, yet rapidly growing segment of commercially insured US patients have drug costs of more than $250,000 each year, and this group could account for 15% of all drug expenditures in the next 5 years.

This week, Russian drug maker Generium announced that it has received approval for—and has launched—its biosimilar dornase alfa in the Russian marketplace. Generium’s product is the first biosimilar dornase alfa biosimilar to be approved in any market worldwide.

During this week’s meeting of the European Committee for Treatment and Research in Multiple Sclerosis, held September 11-13 in Stockholm, Sweden, researchers are presenting new data for eculizumab in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disorder that typically affects the optic nerves and spinal cord and that can cause significant, irreversible disability.

Last week, Alexion disclosed in a filing to the US Securities and Exchange Commission that the European Patent Office did not grant Alexion its request for 2 patents on its brand-name eculizumab product, Soliris, a C5 complement inhibitor that treats rare and ultrarare diseases.

Samsung Bioepis has begun recruiting patients with paroxysmal nocturnal hemoglobinuria (PNH) in India for a phase 3 clinical study of its proposed eculizumab biosimilar, SB12, referencing Soliris.

Drug maker Alexion, developer of the rare disease drug eculizumab (Soliris), announced Friday that the FDA has accepted for priority review its long-acting C5 complement inhibitor, ravulizumab (Ultomiris), which offers less frequent administration than eculizumab.

Amgen is developing a biosimilar of the drug, ABP 959, and during last week’s 24th Congress of the European Hematology Association, held from June 13-16 in Amsterdam, the Netherlands, researchers reported on findings from a phase 1 trial of the proposed product.

The researchers wrote that that a numerically larger proportion of patients with myasthenia gravis (MG) stopped or reduced their doses of immunosuppressive therapies than started or increased, and that symptom improvement or worsening were the main reasons for changes to concomitant therapy.

Russian drug manufacturer Generium Pharmaceutical, which specializes in orphan drugs, announced yesterday that it has received Russian marketing approval for its biosimilar eculizumab, referencing Soliris, the most expensive orphan drug in Russia.

On this Rare Disease Day, learn more about how biosimilars are poised to help treat patients with rare diseases.

Advanced therapy medicinal products (ATMPs), including gene therapies, cell therapies, and tissue engineering products, are providing groundbreaking approaches to treating rare diseases. However, the costs associated with these drugs are expected to pose challenges for affordability and the sustainability. A newly published paper looked ahead to the potential for—and challenges related to—biosimilars of ATMPs.

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.

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).

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.

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.

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.

In a recently published editorial in The Official Journal of the World Federation of Hemophilia, author Angela Thomas, MB, PhD, discusses the advancements in hemophilia treatment as well as the place biosimilars may or may not have in reducing the cost of care.

A recent case report, published in the European Journal of Rheumatology, showed that switching to biosimilar infliximab resulted in a rapid loss of efficacy in 3 patients with Behçet’s disease (BD), a chronic, relapsing, systemic vasculitis.

While no one argues that orphan drugs that treat rare diseases can be expensive, the drugs’ reputation for being budget-busters is not borne out by a data presented in a recent report by Quintiles IMS Institute.

Drug manufacturer JCR announced last month that it has submitted a marketing approval in Japan for JR-051, a proposed agalsidase beta biosimilar referenced on Fabrazyme.