FDA OKs Novartis AG Drug for a Form of Childhood Arthritis

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Novartis Drug Ilaris® Approved by FDA to Treat Active Systemic Juvenile Idiopathic Arthritis, a Serious Form of Childhood Arthritis

EAST HANOVER, N.J., May 10, 2013 /PRNewswire/ -- Novartis announced today that the US Food and Drug Administration (FDA) has approved Ilaris^® (canakinumab) for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients aged 2 years and older. Ilaris is the first interleukin-1 beta (IL-1 beta) inhibitor approved for SJIA and the only treatment approved specifically for SJIA that is given as a once-monthly subcutaneous injection⁽¹⁾. SJIA is a rare and disabling form of childhood arthritis characterized by spiking fever, rash and arthritis that can affect children as young as 2 years old and can continue into adulthood^(2,3).

This approval was based on two Phase III trials in SJIA patients, aged 219, showing significant improvement in the majority of Ilaris-treated patients⁽¹⁾. Study 1 showed that 84% of patients treated with one subcutaneous dose of Ilaris achieved the primary endpoint of the adapted pediatric American College of Rheumatology 30 (ACR30), compared to 10% achievement of ACR30 for placebo at Day 15⁽¹⁾. In the open-label part of Study 2, 92 of 128 patients attempted corticosteroid tapering. Of those 92 patients, 62% were able to substantially reduce their use of corticosteroids, and 46% completely discontinued corticosteroids⁽¹⁾. In the controlled portion of Study 2, there was a 64% relative reduction in the risk of flare for patients in the Ilaris group as compared to those in the placebo group (hazard ratio of 0.36; 95% CI: 0.17 to 0.75).

“The efficacy of Ilaris, along with its monthly subcutaneous dosing, make it an exciting new option for children who are living with this debilitating disease,” said Daniel Lovell, MD, MPH, study investigator and Joseph E. Levinson Professor of Pediatrics at the Cincinnati Children’s Hospital Medical Center. “Additionally, the potential to reduce corticosteroid use is particularly beneficial in this patient population given the side effects associated with long-term use of corticosteroids in children.”

SJIA affects 5-15 children per 100,000 in the United States,and is the most severe subtype of juvenile idiopathic arthritis^(3-5). Although the disease can be life-threatening, treatment options are limited. Corticosteroids are often used to treat symptoms and pain despite their association with potentially serious adverse effects, including Cushing syndrome, growth suppression and osteoporosis^(1,6,7).

“In the US, this approval marks the second Ilaris indication for patients living with rare, autoinflammatory conditions,” said Timothy Wright, MD, Global Head of Development, Novartis Pharmaceuticals. “We are committed to studying Ilaris in other IL-1 beta mediated inflammatory diseases, including several rare diseases for which treatment options do not currently exist.”

About the Pivotal Phase III Studies
Study 1, a 4-week, randomized, double-blind, placebo-controlled study, involved 84 patients between the ages of 2 and 19 years with active SJIA^(1,2). Patients were treated with either a single subcutaneous dose of Ilaris (4 mg/kg, up to 300 mg) (n=43) or placebo (n=41)⁽¹⁾. The primary endpoint was the proportion of patients achieving the adapted pediatric American College of Rheumatology (ACR) 30 response criteria and resolution of fever from baseline at Day 15⁽¹⁾. This means that patients had at least a 30% improvement in systemic and arthritic symptoms versus baseline. The study met its primary endpoint.

Study 2, a two-part study, had an open-label, single-arm active treatment in Part I followed by a randomized, double-blind, placebo-controlled, event-driven withdrawal design in Part II⁽¹⁾. A total of 177 patients between the ages of 2 and 19 years with active SJIA were enrolled in the study⁽¹⁾. Some of these patients had previously participated in Study 1. In Part I, patients received a subcutaneous dose of Ilaris (4 mg/kg, up to 300 mg) every 4 weeks⁽¹⁾. The primary endpoint of Part I was to assess whether treatment with Ilaris allowed successful tapering of corticosteroids in at least 25% of SJIA patients who entered the study using a corticosteroid.

In Part II of the study, patients were randomized to either continue receiving Ilaris, or to receive placebo every 4 weeks (“placebo-after-Ilaris group”), until a pre-specified number (37) of flare-events (“flares”) had occurred⁽¹⁾. The primary endpoint of Part II was to demonstrate that the time to flare was longer with Ilaris than with placebo.

The primary endpoints for Study 1 and Study 2 were all met.

About Ilaris
Ilaris is a selective, fully human, monoclonal antibody that inhibits IL-1 beta, which is an important part of the body’s immune system defenses⁽¹⁾. Excessive production of IL-1 beta plays a prominent role in certain inflammatory diseases⁽⁸⁾. Ilaris works by neutralizing IL-1 beta for a sustained period of time, therefore inhibiting inflammation⁽¹⁾.

In addition to its approval for SJIA in the US, Ilaris is approved in more than 60 countries, including in the EU, US, Switzerland and Japan for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), a rare, lifelong, genetic disorder with debilitating symptoms⁽¹⁾. The approved indication may vary depending upon the individual country.

Important Safety Information
Ilaris can cause serious side effects, including increased risk of serious infections. Ilaris can lower the ability of your immune system to fight infections. Your healthcare provider should:

• test you for tuberculosis (TB) before you receive Ilaris
• monitor you closely for symptoms of TB during treatment with Ilaris
• check you for symptoms of any type of infection before, during, and after treatment with Ilaris.

Tell your healthcare provider right away if you have any symptoms of an infection such as fever, sweats or chills, cough, flu-like symptoms, weight loss, shortness of breath, blood in your phlegm, sores on your body, warm or painful areas on your body, diarrhea or stomach pain, or feeling very tired.

You should not receive Ilaris if you are allergic to canakinumab or any of the ingredients in Ilaris.

Before receiving Ilaris, tell your healthcare provider about all your medical conditions, including if you:

• have or are being treated for an active infection
• have symptoms of infection
• have a history of infections that keep coming back
• have a history of low white blood cells
• have or have had HIV, Hepatitis B, or Hepatitis C
• are scheduled to receive any immunizations (vaccines). You should not get ‘live vaccines’ if you are receiving Ilaris.

Patients should tell their healthcare provider if they are pregnant or planning to become pregnant. It is not known if Ilaris will harm an unborn baby. Patients who become pregnant while receiving Ilaris should tell their healthcare provider right away. Breast-feeding is not recommended during Ilaris therapy.

Tell your healthcare provider about all the medicines you take, including prescription and nonprescription medicines, vitamins and herbal supplements. Especially tell your healthcare provider if you take:

• medicines that affect the immune system
• medicines called interleukin-1 (IL-1) blocking agents such as Kineret® (anakinra) or Arcalyst® (rilonacept)
• medicines called Tumor Necrosis Factor (TNF) inhibitors such as Enbrel® (etanercept), Humira® (adalimumab), Remicade® (infliximab,) Simponi® (golimumab), or Cimzia® (certolizumab pegol)
• medicines that affect enzyme metabolism.

Ask your healthcare provider if you are not sure.

Ilaris can cause serious side effects including:

• serious infections
• decreased ability of the body to fight infections (immunosuppression). For people treated with medicines that cause immunosuppression like Ilaris, the chances of getting cancer may increase.
• allergic reactions. Allergic reactions can happen while receiving Ilaris. Call your healthcare provider right away if you have any of these symptoms of an allergic reaction: rash, itching and hives, difficulty breathing or swallowing, dizziness or feeling faint.
• risk of infection with live vaccines. You should not get live vaccines if you are receiving Ilaris. Tell your healthcare provider if you are scheduled to receive any vaccines.

The most common side effects of Ilaris when used for the treatment of CAPS include: cold symptoms, diarrhea, flu (influenza), runny nose, nausea, headache, cough, weight gain, feeling like you are spinning (vertigo) and injection site reactions (such as redness, swelling, warmth, or itching)

The most common side effects of Ilaris when used for the treatment of SJIA include: cold symptoms, upper respiratory tract infection, pneumonia, runny nose, sore throat, urinary tract infection, nausea, vomiting and diarrhea (gastroenteritis), stomach pain and injection site reactions

Tell your healthcare provider about any side effect that bothers you or does not go away.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see full US prescribing information, including Medication Guide at:
http://www.pharma.us.novartis.com/products/ilaris.shtml.

Disclaimer
The foregoing release contains forward-looking statements that can be identified by terminology such as “is being studied,” “committed,” “is being investigated,” “investigational,” “potential,” “could,” or similar expressions, 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 reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results with Ilaris to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that Ilaris will be submitted or approved for any additional indications or labeling in any market. Nor can there be any guarantee that Ilaris will achieve any particular levels of revenue in the future. In particular, management’s expectations regarding Ilaris could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; government, industry and general public pricing pressures; competition in general; the company’s ability to obtain or maintain patent or other proprietary intellectual property protection; unexpected manufacturing issues; the impact that the foregoing factors could have on the values attributed to the Novartis Group’s assets and liabilities as recorded in the Group’s consolidated balance sheet, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. 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 Pharmaceuticals Corporation researches, develops, manufactures and markets innovative prescription drugs used to treat a number of diseases and conditions, including cardiovascular, dermatological, central nervous system, bone disease, cancer, organ transplantation, psychiatry, infectious disease and respiratory. The company’s mission is to improve people’s lives by pioneering novel healthcare solutions.

Located in East Hanover, New Jersey, Novartis Pharmaceuticals Corporation is an affiliate of Novartis AG, which 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, cost-saving generic pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2012, the Group achieved net sales of USD 56.7 billion, while R&D throughout the Group amounted to approximately USD 9.3 billion (USD 9.1 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 129,000 full-time-equivalent associates and operate in more than 140 countries around the world. For more information, please visit http://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at http://twitter.com/novartis.

References:

1. Ilaris [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals Corp; 2013.
2. Woo P. Systemic juvenile idiopathic arthritis: diagnosis, management, and outcome. Nat Clin Pract Rheumatol 2006; 2(1):28-34.
3. Ramanan AV, Grom AA. Does systemic-onset juvenile idiopathic arthritis belong under juvenile idiopathic arthritis? Rheumatology (Oxford) 2005; 44(11):1350-3.
4. Beukelman T, Patkar NM, Saag KG, et al. 2011 American College of Rheumatology Recommendations for the Treatment of Juvenile Idiopathic Arthritis: Initiation and Safety Monitoring of Therapeutic Agents for the Treatment of Arthritis and Systemic Features. Arthritis Care & Research 2011; 63(4):465-482.
5. Dewitt EM, Kimura Y, Beukelman T, et al. Consensus Treatment Plans for New-Onset Systemic Juvenile Idiopathic Arthritis. Arthritis Care & Research 2012; 64(7):1001-1010.
6. U.S. National Library of Medicine (NLM), National Institutes of Health (NIH). Cushing Syndrome. Available at: http://www.nlm.nih.gov/medlineplus/ency/article/000389.htm. Last accessed: 12/12/12.
7. Teitelbaum SL, Seton MP, Saag KG. Should Bisphosphonates be Used for Long-Term Treatment of Glucocorticoid-Induced Osteoporosis? Arthritis Rheum. 2011 February 63(2): 325328. doi:10.1002/art.30135.
8. Martinon F, Petrilli V. Gout-associated uric acid crystals activate the NALP3 inflammasome. Nature 2006; 440(9): 237-241.

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SOURCE Novartis Pharmaceuticals Corporation

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