Novartis wins esteemed Prix Galien Foundation Best Biotechnology Product Award for CAR-T cell therapy, Kymriah®

EAST HANOVER, N.J., Oct. 29, 2018 /PRNewswire/ -- Novartis was awarded the 2018 Prix Galien USA Award for Best Biotechnology Product for Kymriah^® (tisagenlecleucel) following its first-in-class approval by the US Food and Drug Administration (FDA) for the treatment of children and young adults with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL). Kymriah is a ground-breaking one-time treatment that uses a patient's own T cells to fight cancer, and the only chimeric antigen receptor T cell (CAR-T) therapy approved for two distinct B-cell malignancies¹. The award, which recognizes excellence in scientific innovation that improves the state of human health, was presented at a ceremony in New York City.

"It is a great honor to receive this prestigious award for Kymriah," said Liz Barrett, CEO, Novartis Oncology. "The impact for patients with aggressive blood cancers who previously had limited treatment options is our greatest reward and drives us to continue pioneering efforts to reimagine the way cancer is treated."

The historic approval of Kymriah was a result of collaboration with researchers at University of Pennsylvania (Penn) who demonstrated the first successful use of gene transfer therapy to use the body's immune cells to fight cancer. Novartis further researched this emerging class of drug, initiating the first global CAR-T trials and in August 2017, became the first company to gain a regulatory approval of a CAR-T cell therapy. This year, Kymriah has also been approved in the United States for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL); and for the treatment of pediatric and young adult patients with r/r ALL and adults with r/r DLBCL in the European Union, Canada and Switzerland. Of note, Kymriah is not approved for the treatment of patients with primary central nervous system lymphoma¹.  

Both B-cell ALL and DLBCL are aggressive malignancies, and for patients who relapse or don't respond to therapy, there are limited treatment options^2,3.

Novartis Prix Galien Awards
Considered "the pharmaceutical industry's Nobel Prize," the Prix Galien recognizes excellence in scientific innovation that improves the state of human health, and acknowledges the technical, scientific and clinical research skills necessary to develop such innovative medicines. As a true testament to the company's commitment to reimagine medicine, since 1970, Novartis has received more than 40 national Prix Galien awards in 15 countries for innovative therapies including Gleevec^® (imatinib mesylate), Kymriah^® (tisagenlecleucel), Parlodel^® (bromocriptine mesylate), Rimactane^® (rifampin), Sandimmune^® (cyclosporine), Sandostatin^® (octreotide acetate), Simulect^® (basiliximab) and Visudyne^® (verteporfin)⁴.

About Kymriah^® (tisagenlecleucel)
Kymriah is an innovative immunocellular therapy, manufactured individually for each patient by reprogramming the patient's own immune system cells. Kymriah is the only approved CAR-T cell therapy manufactured using the 4-1BB costimulatory domain, which is critical for full activation of the therapy, enhancement of cellular expansion and durable persistence of the cancer-fighting cells. In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.

Kymriah^® (tisagenlecleucel) US Important Safety information 
Kymriah may cause side effects that are severe or life-threatening, such as Cytokine Release Syndrome (CRS) or Neurological Toxicities. Patients with CRS may experience symptoms including difficulty breathing, fever (100.4^◦F/38^◦C or higher), chills/shaking chills, severe nausea, vomiting and diarrhea, severe muscle or joint pain, very low blood pressure, or dizziness/lightheadedness. Patients may be admitted to the hospital for CRS and treated with other medications. 

Patients with neurological toxicities may experience symptoms such as altered or decreased consciousness, headaches, delirium, confusion, agitation, anxiety, seizures, difficulty speaking and understanding, or loss of balance. Patients should be advised to call their healthcare provider or get emergency help right away if they experience any of these signs and symptoms of CRS or neurological toxicities.

Because of the risk of CRS and neurological toxicities, Kymriah is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called Kymriah REMS.

Serious allergic reactions, including anaphylaxis, may occur after Kymriah infusion.

Kymriah can increase the risk of life-threatening infections that may lead to death. Patients should be advised to tell their healthcare provider right away if they develop fever, chills, or any signs or symptoms of an infection. 

Patients may experience prolonged low blood cell counts (cytopenia), where one or more types of blood cells (red blood cells, white blood cells, or platelets) are decreased. The patient's healthcare provider will do blood tests to check all of their blood cell counts after treatment with Kymriah. Patients should be advised to tell their healthcare provider right away if they get a fever, are feeling tired, or have bruising or bleeding.

Patients may experience hypogammaglobulinemia, a condition in which the level of immunoglobulins (antibodies) in the blood is low and the risk of infection is increased. It is expected that patients may develop hypogammaglobulinemia with Kymriah, and may need to receive immunoglobulin replacement for an indefinite amount of time following treatment with Kymriah. Patients should tell their healthcare provider about their treatment with Kymriah before receiving a live virus vaccine.

After treatment with Kymriah, patients will be monitored lifelong by their healthcare provider, as they may develop secondary cancers or recurrence of their cancer.

Patients should not drive, operate heavy machinery, or do other dangerous activities for eight weeks after receiving Kymriah because the treatment can cause temporary memory and coordination problems, including sleepiness, confusion, weakness, dizziness, and seizures.

Some of the most common side effects of Kymriah are difficulty breathing, fever (100.4°F/38°C or higher), chills/shaking chills, confusion, severe nausea, vomiting and diarrhea, severe muscle or joint pain, very low blood pressure, dizziness/lightheadedness, and headache. However, these are not all of the possible side effects of Kymriah. Patients should talk to their healthcare provider for medical advice about side effects.

Prior to a female patient starting treatment with Kymriah, their healthcare provider may do a pregnancy test. There is no information available for Kymriah use in pregnant or breast-feeding women. Therefore, Kymriah is not recommended for women who are pregnant or breast feeding. Patients should talk to their healthcare provider about birth control and pregnancy.

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

After receiving Kymriah, patients should be advised that some commercial HIV tests may cause a false-positive test result. Patients should also be advised not to donate blood, organs, or tissues and cells for transplantation after receiving Kymriah. 

Please see the full Prescribing Information for Kymriah, including Boxed WARNING, and Medication Guide at www.Kymriah.com  

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations 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 the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, 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 is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 1 billion people globally and we are finding innovative ways to expand access to our latest treatments. About 125 000 people of more than 140 nationalities work at Novartis around the world. Novartis Pharmaceuticals Corporation, a US affiliate of Novartis, is located in East Hanover, NJ. For more information, please visit http://www.novartis.com.

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References

1. Kymriah (tisagenlecleucel) Prescribing information. East Hanover, New Jersey, USA: Novartis Pharmaceuticals Corporation; May 2018.
2. Ronson, A., Tvito, A., Rowe, JM., "Treatment of Relapsed/Refractory Acute Lymphoblastic Leukemia in Adults." Current Oncology Reports, 2016 Jun;18(6):39. https://www.ncbi.nlm.nih.gov/pubmed/27207612. Accessed September 2018.
3. Crump M, Neelapu S, Farooq U, Van Den Neste E, Kuruvilla J et al. (2017) Outcomes in refractory diffuse large B-cell lymphoma: results from the international SCHOLAR-1 study. Blood. E-pub ahead of print August 03. doi: https://doi.org/10.1182/blood-2017-03-769620. Accessed September 2018.
4. "Prix Galien - Winners Sorted by Company." Prix Galien Global Website. http://www.galienfoundation.org/index.php/2017/01/21/winners/. Last accessed September 2018.

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