Johnson & Johnson Subsidiary Submits FDA Application For New Drug To Treat Castleman Disease, A Rare Blood Disorder
By Ed Miseta, Chief Editor, Clinical Leader
Clinical trials conducted by Janssen Research & Development, LLC points to evidence that its experimental drug, siltuximab improved symptoms and shrank growths in patients with Castleman disease, a rare blood disorder that causes enlarged lymph nodes. This condition can lead to a multitude of symptoms and weaken the immune system, making it hard to fight infection. Infections in people with Castleman can be very serious and even fatal. No other approved therapies currently exist for the disease.
The medicine, a monoclonal antibody, targets a molecule believed to fuel excess growth of lymphatic cells that is overproduced in patients. The study funded by the New Brunswick, New Jersey-based Johnson & Johnson subsidiary focused on people with numerous damaged lymph nodes. The disease is so rare that Helgi Van De Velde, senior director of oncology research at Janssen, couldn’t estimate how many people are actually affected by Castleman disease worldwide. It took two years to find 79 patients from nearly two dozen countries to enroll in the trial. One-third of patients on siltuximab responded to treatment, compared with none of those given a placebo, according to the results. Twenty-five percent of siltuximab-treated patients had all symptoms of the disease completely resolved for a median duration of 1.3 years. The most frequent side effects were itching, rash and swelling, with similar rates of other complications. One person developed an allergic reaction after taking the drug.
The company submitted its findings to regulators in the U.S. and Europe in September, looking for an early approval. The medicine, which is also being studied for smoldering multiple myeloma, was granted orphan drug status for Castleman disease. Medicines for rare diseases can qualify for orphan drug status in the U.S. and Europe, a designation that gives pharmaceutical companies exclusive marketing rights as an incentive to develop products that may benefit only a small patient population. The study is from the second of three phases needed to get U.S. regulatory approval.
Castleman disease is currently treated with chemotherapy to kill the dividing cells. Doctors also use RITUXAN®, from Roche Holding AG and Biogen Idec Inc., which destroys the immune system’s B cells. “Doctors have often done things that weren’t based entirely on science because it’s such a difficult disease to study,” said Stephen Ansell, a hematologist at the Mayo Clinic in Rochester, Minnesota, who wasn’t involved with the trial. “This looks like a pretty promising therapy. Hopefully it’s a stepping stone that we can use to build on the things we have.”
Janssen Research and Development, LLC is a division of Johnson and Johnson focuses on developing products and healthcare solutions for unmet medical needs in oncology, immunology, neuroscience, infectious diseases and vaccines, and cardiovascular and metabolic diseases.
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Source: Bloomberg Business Week