RARE RECAP: Johnson and Johnson are now working on the first ever medicinal treatment for Multicentric Castleman Disease (MCD), previously the only other treatment was for a selected type of the disease and was surgical.

Johnson & Johnson (J&J) has filed for approval of its investigational drug siltuximab in both the EU and US as a treatment for a rare blood disorder.

The group’s pharma subsidiary, Janssen, has submitted market applications to both the Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the use of siltuximab in patients with multicentric Castleman disease (MCD) who do not have HIV or human herpes virus-8.

The condition is a rare disease that causes white blood cells to over-produce, leading to the enlargement of lymph nodes.

According to J&J, this can cause several symptoms, such as fever, nausea and nerve damage, and weaken a person’s immune system, making it hard to fight infection.

There is also a unicentric form of the condition, which affects only a single area or group of lymph node and can be treated with surgery.

However, the multicentric form of the condition affects lymphoid tissue around the body and is much harder to treat, with current therapies including corticosteroids, chemotherapy and immunotherapy.

If approved, siltuximab would be the first medicine approved in the US or Europe specifically for MCD and would mark a major step in treatment for the rare condition.

“As a rare disease with a small patient population, MCD is an area of significant unmet need,” said Dr Peter Lebowitz, global oncology therapeutic area head, Janssen.

He explained that siltuximab works by targeting interleukin-6, which is a cytokine, or cell signalling molecule, that is thought to be the critical driver of MCD.

“By focusing on core biologic mechanisms, we now have the potential of helping patients with a condition that is challenging to treat,” added Dr Lebowitz.

The submissions are based primarily on data from the randomised MCD2001 study, which compared siltuximab plus best supportive care with placebo plus best supportive care.

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