FDA Approves Kite’s CAR T-Cell Therapy For Leukemia

October 07, 2021 - FDA recently approved Kite’s chimeric antigen receptor (CAR) T-cell therapy, Tecartus, to treat adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). 

Median overall survival for patients living with ALL is nearly eight months with current standard-of-care treatments. And about half of patients with ALL will relapse Tecartus is the first and only CAR T-cell therapy approved for adults 18 years of age and older living with this disease.

“Today marks Kite’s fourth FDA-approved indication in cell therapy in under four years, demonstrating our commitment to advancing CAR T for patients across many different hematologic malignancies,” Christi Shaw, chief executive officer of Kite, said in the announcement. 

“Tecartus has already transformed outcomes for adults living with mantle cell lymphoma, and we look forward to offering the hope for a cure to patients with ALL,” Shaw continued. 

FDA based its approval of Tecartus on results from a global study, ZUMA-3, in which 65 percent of patients achieved complete remission or complete remission with incomplete hematological recovery at a median follow-up of 12.3 months. 

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The complete remission exceeded 12 months for over half of the patients in the trial. 

Tecartus is also under review in the EU and UK to treat adult patients with relapsed or refractory B-cell precursor ALL.

“Developing new therapies that would be life-changing for people with cancer has been a dream of LLS. We are proud to see the potential of CAR T realized for even more people with this approval for brexucabtagene autoleucel,” said Lee Greenberger, PhD, chief scientific officer of The Leukemia & Lymphoma Society (LLS).

FDA Approves First Medication for Cholestatic Pruritus 

FDA recently approved Mirum Pharmaceuticals’ rare liver disease drug, Livmarli, to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older.

Livmarli is an oral ileal bile acid transporter (IBAT) inhibitor and is the first and only FDA-approved medication for cholestatic pruritus, which affects about 2,000 to 2,500 children in the US. 

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FDA based its approval of Livmarli on the ICONIC study and five years of data from supportive studies resulting in a robust body of evidence in 86 patients with ALGS. 

In the study, Livmarli elicited statistically significant reductions in pruritus. 

To date, no approved treatments are available for cholestatic pruritus in ALGS, and many children require major surgical interventions such as liver transplantation for refractory pruritus. 

“Today is a great day for the Alagille syndrome community with the approval of a much-needed new treatment option to address one of the most debilitating effects of this disease,” Chris Peetz, president and chief executive officer of Mirum, said in the announcement. 

“We are grateful to the patients, families, and healthcare professionals who advanced the research and participated in the LIVMARLI clinical studies. Today is also a landmark day for Mirum as we take steps forward in developing potentially life-changing medicines for rare liver disease,” Peetz continued. 

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Along with the approval, Mirum Pharmaceuticals also received a rare pediatric disease priority review voucher. 

FDA Approves AbbVie’s Preventative Episodic Migraine Drug 

FDA recently approved AbbVie’s Qulipta, the first and oral calcitonin gene-related peptide (CGRP) receptor antagonist for the preventative treatment of migraines. 

Migraines affect about 1 billion people worldwide, including 39 million people in the US alone. Migraines are also the highest cause of disability worldwide for people under 50 years of age.

Qulipta can help reduce monthly migraine days with a once-daily, oral dose that works quickly and continuously.

“This approval reflects a broader shift in the treatment and management paradigm for the migraine community. Qulipta provides a simple oral treatment option specifically developed to prevent migraine attacks and target CGRP, which is believed to be crucially involved in migraine in many patients,” Peter J. Goadsby, MD, PhD, DSc, neurologist and professor at The University of California, and King’s College, London, said in the announcement. 

FDA approved Qulipta based on results from a clinical program that evaluated the drug’s efficacy, safety, and tolerability in nearly 2,000 patients who experienced four to 14 migraine days per month.

In the Phase 3 ADVANCE study, patients treated with 60 milligrams of Qulipta across 12 weeks experienced a 4.2-day reduction from a baseline of 7.8. 

Additionally, 56 percent, 59 percent, and 61 percent of patients in the 10-milligram, 30-milligram, and 60-milligram groups, respectively, achieved a 50- to 100-percent reduction, compared to 29 percent of patients in the placebo group.