DIRA is an ultra-rare, autoinflammatory disease that typically presents in infancy and is characterized by life-threatening and painful skin and bone inflammation, intense, chronic pain and stunted growth^1,2

WALTHAM, Mass., Dec. 22, 2020 (GLOBE NEWSWIRE) -- Sobi™, an international biopharmaceutical company dedicated to rare diseases, announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for KINERET^® (anakinra) for the treatment of deficiency of IL-1 receptor antagonist (DIRA).

DIRA is an ultra-rare, autoinflammatory disease caused by a genetic mutation in the IL1RN gene, which encodes the interleukin-1 receptor antagonist (IL-1Ra) protein.² 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.²

“DIRA signs and symptoms, which can occur in the first weeks of life and may be fatal, require urgent diagnosis and management. The approval of KINERET for DIRA will allow Sobi to bring a new treatment option to this patient community,” said John Yee, MD, MPH, Chief Medical Officer, Sobi North America. “We are grateful to the patients, families, researchers and clinicians whose participation helped make this approval possible.”

The safety and efficacy of KINERET were evaluated in a long-term natural history study, including nine patients with DIRA (ages 1 month to 9 years at start of KINERET treatment) treated with KINERET for up to 10 years. All patients had genetically confirmed DIRA. The starting dose of KINERET was 1 to 2 mg/kg/day in the six patients for which the dose was reported (the remaining 3 patients’ starting dose was not reported). The dose was then individually adjusted to reach a stable efficacious dose to control active inflammation. The highest KINERET dose studied was 7.5 mg/kg/day. At the last visit during the first KINERET treatment period, the dose ranged from 2.2 and 6.1 mg/kg/day. Inflammatory remission was defined as achievement of all of the following criteria: CRP ≤ 5 mg/L, no pustulosis, no inflammatory bone disease and no concomitant glucocorticosteroids use. All nine patients achieved inflammatory remission while on KINERET treatment. This clinical research was conducted by investigators at the National Institutes of Health.

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 KINERET was consistent with the safety profile of KINERET use in patients with Neonatal-Onset Multisystem Inflammatory Disease (NOMID), a previously approved indication. There were no permanent discontinuations of KINERET due to adverse events. All nine patients achieved inflammatory remission, which was defined by normalization of C-reactive protein (CRP) levels to ≤ 5mg/L, no evidence of pustulosis (skin rash) or inflammatory bone disease and discontinuation of glucocorticosteroids on KINERET.

Karen Durrant, RN, BSN, President, the Autoinflammatory Alliance said, “We are excited about this approval and what it means for people living 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.”

KINERET was originally approved by FDA in November 2001 for moderate to severe active rheumatoid arthritis and in January 2013 for a severe form of Cryopyrin-Associated Periodic Syndromes (CAPS), known as NOMID.

For more information about KINERET, please visit KineretRX.com.

INDICATION

KINERET^® (anakinra) is a prescription medicine called an interleukin-1 receptor antagonist (IL-1Ra) used to:

• Reduce the signs and symptoms and slow the damage of moderate to severe active rheumatoid arthritis (RA) in people aged 18 years and older when 1 or more other drugs for RA have not worked
• Treat people with a form of Cryopyrin-Associated Periodic Syndromes (CAPS) called Neonatal-Onset Multisystem Inflammatory Disease (NOMID)
• Treat people with Deficiency of Interleukin-1 Receptor Antagonist (DIRA)
  

KINERET is not for children with Juvenile Rheumatoid Arthritis.

IMPORTANT SAFETY INFORMATION

KINERET should not be taken for those who are allergic to proteins made from bacteria called E. coli, or anakinra and any of its ingredients. Patients/caregivers should speak with their healthcare provider if they are not sure. A complete list of any of the ingredients in KINERET can be found at the end of the patient leaflet.

Before using KINERET, patients should talk with their healthcare provider if they have an infection, a history of infections that keep coming back, or other problems that can increase risk of infections, are scheduled to receive any vaccines as patients using KINERET should not receive live vaccines, or have kidney problems. Patients who are pregnant or plan to become pregnant should speak with their healthcare provider as it is not known if KINERET will harm the unborn baby, are breastfeeding or plan to breastfeed. It is not known if KINERET passes into breast milk. Patients and their healthcare provider should decide if they will use KINERET or breastfeed.

Patients should tell their healthcare provider about all medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. KINERET and other medicines may affect each other and cause serious side effects. Patients should especially tell their healthcare provider if they take certain other medicines that affect the immune system called Tumor Necrosis Factor (TNF) blockers. Patients should ask their healthcare provider for a list of these medicines if they are not sure.

ADVERSE EVENTS

KINERET may cause serious side effects, including serious infections, allergic reactions, decreased ability of the body to fight infections (immunosuppression) and low white blood cell count (neutropenia).

The most common side effects of KINERET include injection site skin reactions, including redness, swelling, bruising, itching, and stinging. Most injection site reactions are mild, happen early during treatment, and last about 14 to 28 days. Injection site reactions have been observed less frequently in people with NOMID.

Other common side effects of KINERET include RA gets worse with treatment, if patients already have RA, headache, nausea and vomiting, diarrhea, joint pain, fever, feeling flu-like symptoms, sore throat or runny nose, sinus infection or pain in your stomach area.

Patients should tell their healthcare provider if they have any side effect that bothers them or does not go away. These are not all of the possible side effects of KINERET. For more information patients should ask their healthcare provider or pharmacist.

Please click here for full Prescribing Information.

About Deficiency of IL-1 Receptor Antagonist (DIRA)

DIRA is an ultra-rare, autoinflammatory disease caused by a genetic mutation in the IL1RN gene.

The IL1RN gene produces the interleukin-1 receptor antagonist (IL-1Ra) protein which plays a critical role in helping regulate the interleukin-1 (IL-1) signaling pathway.² IL-1 is a powerful driver of inflammation and plays an important role in the body’s immune response. IL-1 proinflammatory signaling primarily occurs through IL-1α and IL-1β. IL-Ra competes with IL-1α and IL-1β, inhibiting proinflammatory signaling.³ In patients with DIRA, the deficiency of the IL-1Ra leads to unopposed action of IL-1α and IL-1β, resulting in life-threatening systemic inflammation with skin and bone involvement.²

About KINERET (anakinra)

KINERET is an interleukin-1 receptor antagonist (IL-1Ra) that is indicated for reduction in signs and symptoms and slowing the progression of structural damage in moderately to severely active rheumatoid arthritis, in patients 18 years of age or older who have failed 1 or more disease modifying antirheumatic drugs (DMARDs), for the treatment of Neonatal-Onset Multisystem Inflammatory Disease (NOMID), a form of Cryopyrin-Associated Periodic Syndromes (CAPS) and for the treatment of patients with Deficiency of Interleukin-1 Receptor Antagonist (DIRA).

About Sobi™ in North America

As the North American affiliate of international biopharmaceutical company Sobi™, our team is committed to Sobi’s vision of providing access to innovative treatments that make a significant difference in the lives of individuals with rare diseases. Our product portfolio includes multiple approved treatments, focused on inflammation/immunology and genetics/metabolism. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at www.sobi-northamerica.com or at www.sobi.com. 

For more information please contact

Investor Relations
Paula Treutiger, Head of Communication & Investor Relations
+ 46 733 666 599
paula.treutiger@sobi.com

Media
David Caruba, Director of Communications, Sobi North America
+ 1 908-347-0154
david.caruba@sobi.com

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1} Jesus AA, Goldbach-Mansky R. (2014). IL-1 Blockade in Autoinflammatory Syndromes. Annu Rev Med. Jan 14;65:223-44. PMID: 24422572
² Aksentijevich I, et al. N Engl J Med 2009 Jun 4;360(23):2426-37
³ Dinarello, C. A. (2019). The IL-1 family of cytokines and receptors in rheumatic diseases. Nature Reviews Rheumatology, 15(10), 612–632.