Novartis receives positive CHMP recommendation for Ilaris®

• Periodic Fever Syndromes are rare diseases mostly affecting children[1] and cause recurrent and disabling fevers with potentially life-threatening complications[2]  
• Ilaris® (canakinumab) is recommended for approval in Europe for the treatment of three Periodic Fever Syndromes: TRAPS, HIDS/MKD and FMF disease  
• If approved, Ilaris will be the first and only biologic treatment in Europe for these rare diseases, providing rapid and sustained disease control for patients[3]

Basel, December 16, 2016 - Novartis announced today that the Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Ilaris ^® (canakinumab) in Europe to treat three rare and distinct Periodic Fever Syndromes.

If approved, Ilaris will be the first and only approved biologic treatment in Europe for Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF).

"Few treatments exist for people with these potentially life-threatening conditions. Today's positive recommendation is a great stride forward for patients who urgently need new treatment options," said Paul Hudson, CEO, Novartis Pharmaceuticals. "Novartis is committed to paving the way forward for rare diseases especially through treatments like Ilaris which has the potential to raise the standard of care."

The CHMP positive opinion, which also recommends the use of a new formulation which is a 150 mg/ml solution for injection, is based on results from the pivotal Phase III CLUSTER study. Results showed rapid (at Day 15) and sustained disease control with Ilaris compared to placebo through 16 weeks, in patients with either TRAPS, HIDS/MKD or FMF[3]. Data show Ilaris to be a well-tolerated and effective treatment for these three rare conditions.

All three conditions are part of a group of rare autoinflammatory diseases called Periodic Fever Syndromes, which are also referred to as Hereditary Periodic Fevers (HPF). These can cause disabling and persistent fevers which may be accompanied by joint pain, swelling, muscle pain and skin rashes with complications that can be life-threatening[2]. The most common syndrome is FMF, which mainly affects people of Eastern Mediterranean ancestry. It affects 1 in 250 to 1 in 1,000 individuals in these populations, many of whom are children[1].

In August 2016, the European Commission approved Ilaris for a license extension to treat patients with Adult-Onset Still's Disease (AOSD), a rare type of inflammatory arthritis. The US Food and Drug Administration (FDA) previously granted three simultaneous approvals of Ilaris for the treatment of TRAPS, HIDS/MKD and FMF in September 2016.

About Periodic Fever Syndromes Periodic Fever Syndromes are a group of diseases that cause serious recurrent fever and pathogenic inflammation through non-infectious activation of the immune system. Most patients present with symptoms in infancy or childhood, but in some patients the condition only becomes apparent or diagnosed in adulthood[1].

Previous treatments for these rare conditions consisted of oral anti-inflammatory drugs, such as corticosteroids, which were used only to help manage the symptoms. While other medicines, such as non-steroidal anti-inflammatory drugs, have also been used to help reduce symptoms, they do not prevent or change the overall course of a flare[2].

About Ilaris Ilaris is a selective, high-affinity, human monoclonal antibody that inhibits Interleukin-1 (IL-1) beta, which is an important part of the body's immune system defences[4]. Excessive production of IL-1 beta plays a prominent role in certain inflammatory diseases[5],[6]. Ilaris works by blocking the action of IL-1 beta for a sustained period of time, therefore inhibiting inflammation that is caused by its over-production[4]. Novartis has reformulated Ilaris from a powder that needed to be reconstituted into a solution prior to use to a solution for injection (ready to use) which will ease administration of the product.

Ilaris is currently approved and marketed for the treatment of Systemic Juvenile Idiopathic Arthritis (SJIA) in the US and EU and for the treatment of AOSD and the symptomatic treatment of refractory acute gouty arthritis in the EU. Ilaris is also approved in more than 70 countries, including in the EU, Switzerland, US, Canada, and Japan for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS): rare, lifelong, genetic disorders with debilitating symptoms. In the EU, Ilaris is approved for following subtypes of CAPS: Muckle-Wells syndrome (MWS), neonatal-onset multisystem inflammatory disease (NOMID)/chronic infantile neurological, cutaneous, articular syndrome (CINCA), severe forms of familial cold autoinflammatory syndrome (FCAS)/familial cold urticaria (FCU) presenting with signs and symptoms beyond cold-induced urticarial skin rash. The approved indications may vary depending upon the individual country.

Disclaimer The foregoing release contains forward-looking statements that can be identified by words such as "positive CHMP recommendation," "recommended for approval," "will," "recommended approval," "positive recommendation," "stride forward," "committed," "potential," "positive opinion," "recommends," or similar terms, or by express or implied discussions regarding potential new indications or labeling for Ilaris, or regarding potential future revenues from Ilaris. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Ilaris will be submitted or approved for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that Ilaris will be commercially successful in the future. In particular, management's expectations regarding Ilaris could be affected by, among other things, the uncertainties inherent in research and development, including unexpected clinical trial results and additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; unexpected safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care and cost-saving generic pharmaceuticals. Novartis is the only global company with leading positions in these areas. In 2015, the Group achieved net sales of USD 49.4 billion, while R&D throughout the Group amounted to approximately USD 8.9 billion (USD 8.7 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 118,000 full-time-equivalent associates. Novartis products are available in more than 180 countries around the world. For more information, please visit http://www.novartis.com .

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References [1] National Amyloidosis Centre. Amyloidosis Patient Information Site: The inherited periodic fever syndromes - general information. Available at: www.amyloidosis.org.uk/fever-syndromes/inherited-fever-syndromes/ . Accessed December 2016. [2] Cleveland Clinic. Periodic Fever Syndrome. Available at: https://my.clevelandclinic.org/services/ortho...r-syndrome . Accessed December 2016. [3] Novartis Data on File.  [4] Dhimolea E. Canakinumab, MAbs.  2010 Jan-Feb;2(1):3-13. [5] Jesus AA, Goldbach-Mansky R. IL-1 blockade in autoinflammatory syndromes. Annu Rev Med. 2014;65:223-244.            [6] Toker O, Hashkes PJ. Critical appraisal of canakinumab in the treatment of adults and children with cryopyrin-associated periodic syndrome (CAPS). Biologics. 2010;4:131-138.

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