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Novartis sBLA for CAR-T Therapy Kymriah Accepted by FDA

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Novartis AG‘s (NVS - Free Report) supplemental Biologics License Application (sBLA) for Kymriah suspension for intravenous infusion, for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are ineligible for or relapse after autologous stem cell transplant (ASCT) has been accepted by the FDA for Priority Review.

Moreover, the European Medicines Agency (EMA) also granted accelerated assessment to the Marketing Authorization Application (MAA) for Kymriah for the treatment of children and young adults with r/r B-cell acute lymphoblastic leukemia (ALL) and for adult patients with r/r DLBCL who are ineligible for ASCT.

We remind investors that Kymriah was the first CAR-T therapy to obtain FDA approval in August 2017 for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

A novel immunocellular therapy and one-time treatment, Kymriah uses patient's T cells to fight cancer. CAR-T is manufactured for individual patients using their own cells thereby making it different from typical small molecule or biologic therapies. During the treatment, T cells are drawn from a patient's blood. These cells are then reprogrammed in the manufacturing facility to create genetically coded cells to express a chimeric antigen receptor to recognize and fight cancer cells and other B cells expressing a specific antigen. The drug was developed in collaboration with the University of Pennsylvania. The approval is a major boost for Novartis given the potential in the CAR-T therapy space.  The approval opens up new frontiers in the treatment of cancer by advancing immunocellular therapy for children and young adults with r/r B-cell ALL which comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the United States. Kymriah has been launched in the United States.

The respective designations from the FDA and EMA will expedite the standard review time. Assuming approval from the FDA and EMA, Kymriah will be the first chimeric antigen receptor T cell (CAR-T) therapy available for two distinct indications in non-Hodgkin lymphoma and B-cell ALL. The applications were based on data from the sponsored global clinical trial program of Kymriah in children and young adults with r/r B-cell ALL and adult patients with r/r DLBCL demonstrating the efficacy and safety of Kymriah across studies. The results from the pivotal phase II trial, JULIET, served as the basis of the sBLA and MAA for Kymriah in adult patients with r/r DLCBL. Results from the pivotal phase II ELIANA study were submitted as part of the MAA for Kymriah in children and young adults with r/r B-cell ALL.

Novartis plans additional regulatory submissions for Kymriah in pediatric and young adult patients with r/r B-cell ALL and adult patients with r/r DLBCL beyond the United States and EU in 2018.

 

 

Novartis’ stock has rallied 27.2% in the past one year compared with the industry’s 25.7% gain.

The CAR-T therapy space was in the spotlight in 2017 with the approval of Kymriah. Subsequently, Gilead Sciences, Inc. (GILD - Free Report) acquired Kite Pharma and obtained FDA approval of Yescarta (axicabtagene ciloleucel), the latter’s chimeric CAR-T therapy candidate.

Meanwhile, rumours surfaced that Celgene Corporation is in advanced talks to acquire Juno Therapeutics .  Juno’s pipeline includes CD19 and CD22 directed CAR T-cell product candidates.

Given the immense potential of the therapy, we expect the spotlight in this space in 2018.

Zacks Rank

Novartis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here..

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