Date: 2012-11-11
Type of information: Presentation of results at a congress
phase: 2
Announcement: presentation of results at the 2012 Annual Scientific Meeting of the American College of Rheumatology (ACR)
Company: Novartis (Switzerland)
Product: ACZ885 (canakinumab)
Action
mechanism: ACZ885 is a fully human monoclonal antibody that inhibits IL-1 beta, which is an important part of the body\'s immune system defenses. Excessive production of IL-1 beta plays a major role in certain inflammatory diseases, including SJIA and TRAPS. ACZ885 works by neutralizing IL-1 beta for a sustained period of time, thereby inhibiting inflammation.
Under the brand name Ilaris®, ACZ885 is approved in more than 60 countries, including the EU, US and Switzerland for the treatment of adults and children as young as four years with CAPS, a rare, lifelong, inflammatory disorder with debilitating symptoms. ACZ885 is also being studied in other diseases in which IL-1 beta plays a key role in causing inflammation, such as gouty arthritis and cardiovascular disease.
Disease: Familial Mediterranean Fever (FMF)
Therapeutic area: Rare diseases - Inflammatory diseases
Country:
Trial
details: The Phase II, open-label, single-centre study investigating the efficacy and safety of ACZ885 in patients with colchicine-resistant FMF involved nine patients with a median age of 22 years (range, 12-34 years). The patients, who had experienced >= 1 FMF attack per month in the three months before receiving ACZ885 (the run-in period), received ACZ885 150 mg every four weeks. Treatment began at the start of the first attack during the initial study month. The primary endpoint of the study was a >=50% reduction in attack frequency over three months.
Latest
news: Novartis has announced new data from two Phase II trials of ACZ885 (canakinumab), in patients with one of two rare syndromes characterized by periodic fevers. Key findings include reductions in disease attack frequency, maintenance of symptom relief, normalization of blood markers of inflammation, and quality of life improvements. Both rare orphan diseases, Familial Mediterranean Fever (FMF) and tumor necrosis factor (TNF) receptor-associated periodic syndrome (TRAPS), are serious, inherited autoinflammatory diseases characterized by recurrent (periodic) fever attacks, rash, arthritis, and severe symptoms that can lead to fatal complications.
In the Phase II FMF study, 100% of patients (nine out of nine) achieved at least a 50% reduction in the frequency of disease attacks during three months of ACZ885 treatment. All nine patients in the trial had previously experienced at least one attack per month over three months before receiving ACZ885, while using standard-of-care medication. During the three-month ACZ885 treatment period, eight of the nine patients were attack-free, while blood markers of inflammation (C-reactive protein, CRP, and serum amyloid A, SAA) normalized by Day 8 after ACZ885 dosing and remained low throughout the study. The overall response to treatment was reported to be \'very good\' by physicians in all cases, and by the patients in seven cases.
Adverse events (AEs) observed in the studies were similar to those already seen for ACZ885\'s approved indication in Cryopyrin-Associated Periodic Syndromes (CAPS). Infections, mostly upper respiratory tract infections (URIs) were the most commonly reported category of AE in both the FMF and TRAPS studies. No serious adverse events (SAEs) were reported in the FMF study; while in the TRAPS study, two SAEs were reported: a URI that lasted two days and a severe disease attack with chest pain.
