FDA Grants Orphan Drug Status To Avaxia, Immunomedics, ChemoCentryx
By Cyndi Root
The Food and Drug Administration has moved decisively to find treatments for rare diseases, granting Orphan Drug status to three companies and their pharmaceutical agents. Avaxia, Immunomedics, and ChemoCentryx all received the designation this week, providing them with seven-year U.S. marketing exclusivity and other benefits upon approval. Avaxia’s AVX-470 is for the treatment of pediatric ulcerative colitis (UC). Immunomedics earned the designation for veltuzumab in the treatment of pemphigus. ChemoCentryx’ orphan drug designation is for CCX168 in the treatment of atypical Hemolytic Uremic Syndrome (aHUS).
Avaxia’s AVX-470
Avaxia’s AVX-470 treats pediatric ulcerative colitis (UC). The company announced the FDA’s action in a press release, stating that AVX-470 is a gut-targeted anti-TNF antibody. The agent was successful in a Phase 1b adult clinical trial. AVX-470 is orally administered, unlike marketed anti-TNF drugs that must be infused or injected. Avaxia says that patients prefer oral drugs and AVX-470 shows improvements in safety and efficacy compared to current drugs. The company believes the agent shows promise in inflammatory bowel disease (IBD).
Immunomedics’ Veltuzumab
Veltuzumab, a humanized anti-CD20 antibody, treats pemphigus, which usually presents as blisters in the mucous membranes in the mouth. The condition debilitates patients and is often fatal due to infections and immune system degradation from corticosteroid treatment. In Immunomedics’ announcement on the Orphan status of its agent, the company states that in a compassionate use study, a patient who did not respond to other treatments, did respond to veltuzumab, administered subcutaneously. The patient achieved complete remission and maintained it for 35 months with no serious adverse events. Cynthia L. Sullivan, President and CEO of Immunomedics, stated that her company intends to out-license the product.
ChemoCentryx’ CCX168
ChemoCentryx states that its agent is an inhibitor that targets the receptor for the complement protein C5a (C5aR). Preclinical data shows that C5a receptor inhibition reduces micro-vasculature thrombosis formation. CCX168 treats atypical Hemolytic Uremic Syndrome (aHUS), a progressive disease that primarily affects kidney function. Thomas J. Schall, PhD., President and CEO of ChemoCentryx, stated that his company intends to start a Phase 2 proof-of-concept study by the end of 2014.