FDA Expands Indication for Anakinra to Include Rare Autoinflammatory Disease


Anakinra was initially approved by FDA in November 2001 for treatment of moderate to severe active rheumatoid arthritis.

Officials with the FDA have expanded the indication for anakinra (Kineret; Sobi) to include treatment of deficiency of IL-1 receptor antagonist (DIRA), an ultra-rare, autoinflammatory disease.

Anakinra was initially approved by FDA in November 2001 for treatment of moderate to severe active rheumatoid arthritis. Additionally, anakinra was approved in January 2013 for treatment of a severe form of Cryopyrin-Associated Periodic Syndromes (CAPS), known as Neonatal-Onset Multisystem Inflammatory Disease (NOMID).

DIRA is caused by a genetic mutation in the IL1RN gene, which encodes the interleukin-1 receptor antagonist (IL-1Ra) protein. In patients with DIRA, the deficiency of IL-1Ra leads to unopposed action of IL-1 signaling, resulting in life-threatening systemic inflammation with skin and bone involvement.

“DIRA signs and symptoms, which can occur in the first weeks of life and may be fatal, require urgent diagnosis and management,” said John Yee, MD, MPH, Chief Medical Officer, Sobi North America, in a prepared statement. “We are grateful to the patients, families, researchers and clinicians whose participation helped make this approval possible.”

The safety and efficacy of anakinra for DIRA were evaluated in a long-term natural history study conducted by investigators at the National Institutes of Health. The study included 9 patients with genetically confirmed DIRA—ages 1 month to 9 years at the start of the study—who were treated with anakinra for up to 10 years.

The starting dose of anakinra used in the study was 1 to 2 mg/kg/day in the 6 patients for which the dose was. The dose was then individually adjusted to reach a stable efficacious dose to control active inflammation. The highest anakinra dose studied was 7.5 mg/kg/day. At the last visit during the first anakinra treatment period, the dose ranged from 2.2 and 6.1 mg/kg/day.

Inflammatory remission was defined in the study as achievement of all of the following criteria: CRP ≤ 5 mg/L, no pustulosis, no inflammatory bone disease and no concomitant glucocorticosteroids use. All 9 patients achieved inflammatory remission while on anakinra treatment.

According to Sobi, the most common adverse events in patients with DIRA were upper respiratory tract infections, rash, pyrexia, influenza-like illness and gastroenteritis. The safety profile observed in patients with DIRA treated with anakinra was consistent with the safety profile of this drug used in patients with NOMID. There were no permanent discontinuations of anakinra due to adverse events.

In a prepared statement, Karen Durant, RN, BSN, president, the Autoinflammatory Alliance, said the organization was excited about the FDA’s approval of anakinra for patients with DIRA.

“We hope that the approval will spark greater awareness about this ultra-rare disease and, in turn, bring patients one step closer to finding the treatment they need,” added Durrant.


FDA Approves KINERET® (anakinra) for the Treatment of Deficiency of IL-1 Receptor Antagonist (DIRA) [news release]. Waltham, MA; December 22, 2020: Sobi North America. Accessed December 22, 2020. [email]

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