FDA Approves Drug to Treat Rare Autoimmune Disease

The U.S. Federal Drug Administration (FDA) has approved a drug manufactured by ChemoCentryx to treat a rare group of autoimmune diseases, called anti-neutrophil cytoplasmic autoantibody-associated vasculitis, or ANCA-AAV for short. According to ANCA Vasculitis News, ANCA-AAV causes inflammation and damage to the body’s small blood vessels. This inflammation is the result of antibodies that bind to certain cells of the immune system, called neutrophils, and overly activate them.

Since small blood vessels are found throughout the body, ANCA-AAV causes a variety of symptoms, including:

  • Poor kidney function, leading to kidney failure
  • Severe respiratory problems, including shortness of breath, hoarse voice, cough with blood or mucus, and chest pain
  • Neurological symptoms, including tingling, burning, numbness, and weakness
  • Central nervous system (CNS) involvement, such as headaches, cognitive impairment, and memory deficits. In severe cases, seizures, paralysis or loss of consciousness may also result.
  • Ear, nose, and throat (ENT) symptoms, like sinusitis, nasal discharge, rhinitis, and tinnitus (ringing in the ears)
  • Eye symptoms, including eye pain, vision impairment, and vision loss
  • Joint pain, muscle pain, and muscle loss
  • Skin lesions, including rashes, sores, ulcers, bumps, and bleeding underneath the skin
  • Digestive problems, like vomiting, diarrhea, nausea, abdominal pain, and blood in one’s stool
  • And more…

Due to the numerous symptoms ANCA-AAV causes, it can often be fatal for patients; in fact, ChemoCentryx states that the first year mortality rate for patients living with the disease is between 11 and 18 percent. Current ANCA-AAV treatments on the market include steroids and immunosuppressant medications which compromise the body’s ability to fight off infections. That’s why having a new treatment on the market that works via a different mechanism may be a saving grace for many ANCA-AAV patients.

The new drug, called avacopan, will be sold by ChemoCentryx under the brand name Tavneos. It works by blocking a protein called C5a receptor that is responsible for causing numerous inflammatory diseases. The drug’s wholesale price will be an astronomical $150,000-$200,000 per patient per year. However, it could be the life-saving treatment that the 40,000 ANCA-AAV patients in the US need.

The drug is currently being tested for use on other conditions as well, including the autoimmune skin disease Hidradenitis Suppurativa (HS). The drug has already received approval for commercialization in Japan, and is expected to be approved in Europe by the end of 2021.

Martina, a 29-year-old mother-of-two from Stuttgart, Germany, describes what it was like being diagnosed with this challenging autoimmune condition:

“It began rather unspectacularly, with flu-like symptoms, fever, headaches, and joint pain. So anyone would think, ‘It’s just a normal flu.’ Then it got worse over the course of a week and I went to the hospital.”

She was eventually diagnosed with ANCA-AAV after undergoing a series of blood tests. Unfortunately, she had to give up her career as an educator in the process, since she didn’t have a strong enough immune system to be interacting with children while taking steroids and immunosuppressants to control the disease. Patients like Martina are the ones that could potentially stand to benefit from new drug therapies like avacopan.

Martina, 29, appears in an ad to raise awareness for vasculitis conditions.

To find resources for those living with ANCA-AAV and their caretakers, visit: https://www.myancavasculitis.com/living-with-aav/

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