(RxWiki News) Patients with rare diseases often lack a variety of treatment options. Fortunately, children living with a rare form of arthritis appear to have a new option.
According to a press release from pharmaceutical company Genentech, the US Food and Drug Administration (FDA) has approved Actemra (tocilizumab) for the treatment of polyarticular juvenile idiopathic arthritis (PJIA) – a rare form of childhood arthritis.
The approval was based on results from a recent clinical trial showing that a majority of PJIA patients treated with Actemra had meaningful improvement in their symptoms.
Actemra is already approved to treat a similar form of childhood arthritis.
"Ask a pharmacist about arthritis treatment options."
PJIA is a type of juvenile idiopathic arthritis (JIA). Like rheumatoid arthritis in adults, JIA causes joint pain and inflammation. About 100 out of every 100,000 children are affected by JIA. Around 30 percent of these children have PJIA.
PJIA is defined as inflammation of five or more joints, and most commonly affects the knees, wrists and ankles. PJIA is often symmetrical, meaning it affects the same joints on both sides of the body.
Actemra, which is manufactured by Genentech, is the first interleukin-6 (IL-6) receptor inhibitor. It is most commonly used to treat adults rheumatoid arthritis who did not respond well to at least one disease modifying antirheumatic drug (DMARD).
"Actemra (tocilizumab, by Genentech) is an injectable medication previously only approved for adult rheumatoid arthritis (RA) and systemic juvenile idiopathic arthritis (SJIA). In both conditions it is generally utilized only after attempts to manage the conditions with other medications, such as NSAIDs or methotrexate, were ineffective," explained Jason Poquette, BPharm, RPh, a pharmacist with Decisions Resources Group in Boston.
In 2011, the FDA approved Actemra for the treatment of systemic juvenile idiopathic arthritis (SJIA) — a condition similar to PJIA that can also cause high fevers and rashes.
The recent approval of Actemra is the first FDA approval of a medication to treat PJIA in about five years.
"Polyarticular juvenile idiopathic arthritis is a rare, debilitating condition in children that worsens over time," said Hal Barron, MD, chief medical officer and head of Global Product Development at the Roche Group, of which Genentech is a member.
"We are pleased to offer Actemra to doctors and parents of children aged two or older as an important medicine to hep improve the signs and symptoms of this often painful disease."
According to Poquette, "This approval is good news for children with this rare condition. It offers hope to parents seeking effective options and gives doctors solid evidence that such an approach may work. Patients with questions about Actemra for PJIA should speak to their doctor or pharmacist."
The FDA based its approval of Actemra on data from the Phase III CHERISH study. This study had two parts: an open label phase, meaning both the researchers and participants knew which medication they were taking, and a randomized double-blind placebo-controlled withdrawal phase in which participants did not know whether they were taking Actemra or placebo.
Participants were assigned to take Actemra plus methotrexate (a commonly used arthritis medication), Actemra alone or placebo (fake medication).
According to the Genentech press statement, results from the CHERISH study showed that 91 percent of patients taking Actemra plus methotrexate and 83 percent of those taking Actemra alone had an ACR-30 response after 16 weeks of treatment.
ACR-30 is a measure of response to an arthritis treatment. A patient is said to have achieved ACR-30 if he or she has had 30 percent improvement or more in three out of six of the following measures:
- Physician Global Assessment of Disease Activity
- Parent/Patient Global Assessment of Overall Well-Being
- Number of joints with active arthritis
- Number of joints with limitation of movement
- Erythrocyte sedimentation rate (ESR), a laboratory measure of inflammation
- Physical function as measured by the Childhood Health Assessment Questionnaire disability index
The most common adverse events (negative side effects) observed in the CHERISH study were infections.
The researchers also found that Actemra was associated with decreases in white blood cell and platelet counts as well as increases in certain liver enzymes, which may be a sign of damage to cells in the liver.
Actemra comes with a Boxed Warning which reads, "Serious infections leading to hospitalization or death including tuberculosis (TB), bacterial, invasive fungal, viral, and other opportunistic infections have occurred in patients receiving Actemra."
