Novartis receives positive CHMP opinion for Kymriah® for treating two aggressive blood cancers, marking important medical advance for patients in Europe
Posted on July 2, 2018
• First CHMP opinion for a CAR-T cell therapy in two distinct indications – DLBCL in adults and B-cell ALL in children
• Marketing Authorization Application (MAA) based on landmark global CAR-T clinical trials ELIANA and JULIET, the only CAR-T registrational trials to include European patients
• Novartis has demonstrated strong and durable response rates, and a consistent safety profile for Kymriah
• Recommendation brings Novartis closer to making Kymriah available in the EU to patients who are in critical need of new treatment options
Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of Kymriah® (tisagenlecleucel, formerly CTL019) – a novel one-time treatment that uses a patient’s own T cells to fight cancer. The positive opinion includes two B-cell malignancies: B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse in patients up to 25 years of age; and diffuse large B-cell lymphoma (DLBCL) that is relapsed or refractory (r/r) after two or more lines of systemic therapy in adults.
“The positive CHMP opinion for Kymriah is a watershed moment for pediatric and adult patients in Europe with aggressive blood cancers,” said Liz Barrett, CEO, Novartis Oncology. “This truly transformative therapy helps address a profound unmet need, and Novartis is proud that our leadership in CAR-T innovation will make a meaningful difference to patients in the EU.”
If approved by the European Commission, Kymriah will be the first CAR-T cell therapy available in the European Union (EU) for both DLBCL and B-cell ALL.
Both B-cell ALL and DLBCL are aggressive malignancies with significant treatment gaps for patients. In Europe, ALL accounts for approximately 80% of leukemia cases among children, and for those patients who relapse, the outlook is poor. This low survival rate is in spite of patients having to undergo multiple treatments, including chemotherapy, radiation, targeted therapy or stem cell transplant, and further highlights the need for new treatment options. DLBCL is the most common form of non-Hodgkin lymphoma, accounting for up to 40% of all cases globally. For patients who relapse or don’t respond to initial therapy, there are limited treatment options that provide durable responses, and survival rates are low for the majority of patients due to ineligibility for autologous stem cell transplant (ASCT) or because salvage chemotherapy or ASCT have failed.
The positive CHMP opinion is based on two pivotal Novartis-sponsored global, multi-center, Phase II trials, ELIANA and JULIET, which included patients from Europe, the US, Australia, Canada and Japan.
The collaboration of Novartis and the University of Pennsylvania has led to historic milestones in CAR-T cell therapy since 2012, including the initiation of the first global CAR-T trials, the PRIME designation granted by the EMA for Kymriah in pediatric patients with r/r B-cell ALL, and the approval of Kymriah in two distinct indications by the US Food and Drug Administration (FDA).
“Kymriah is already transforming the way we treat certain types of leukemia and lymphoma in the United States, and showing us that personalized cell therapies are an incredibly powerful tool in the fight against cancer,” said Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine and director of the Center for Cellular Immunotherapies in the Abramson Cancer Center. “We are excited to see that through our collaboration with Novartis, physicians in more countries around the world may be able to use this novel and innovative CAR-T cell therapy to improve the treatment outcomes for their patients.”
ELIANA is the first pediatric global CAR-T cell therapy registration trial, treating patients in 25 centers in the US, Canada, Australia, Japan and the EU, including: Austria, Belgium, France, Germany, Italy, Norway and Spain. JULIET is the first multi-center global registration study for Kymriah in adult patients with r/r DLBCL.
JULIET is also the largest global study evaluating a CAR-T cell therapy in patients with DLBCL, enrolling patients from 27 sites in 10 countries across the US, Canada, Australia, Japan and the EU, including: Austria, France, Germany, Italy, Norway and the Netherlands.
The Novartis CAR-T cell manufacturing platform includes cryopreservation, the process of freezing patients’ harvested cells in order to preserve them, which provides physicians with the flexibility to decide when to initiate both the harvesting of patients’ cells and the infusion of Kymriah, based on each patient’s condition, and allows for this individualized treatment approach on a global scale.
“Today’s positive opinion from the CHMP marks a truly extraordinary moment for those who have been impacted by these types of advanced and aggressive B-cell malignancies,” said Dr. Ulrich Jäger, Professor of Hematology at the Medical University of Vienna and Head of Hematology at the General Hospital of the City of Vienna. “European patients and doctors have been anxiously awaiting the introduction of Kymriah, which will bring a significant advancement in the treatment landscape for these patients who face a poor prognosis.”
The European Commission will now review the CHMP recommendation to deliver its final decision, applicable to all 28 EU member states, plus Iceland, Liechtenstein and Norway.
“This CHMP decision brings an innovative option closer to European patients who have exhausted most alternative treatments,” said Zack Pemberton-Whiteley, Chair of the Global Acute Leukemia Advocates Network and Campaigns and Advocacy Director at Leukaemia Care. “It’s important to note that CAR-T therapy may be suitable for a limited group of patients. Working with healthcare professionals to ensure safe and timely access for those who need it will be of paramount importance.”
Additional regulatory filings are under review for Kymriah in Canada, Switzerland, Australia and Japan.
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