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Monthly News Roundup - August 2017

Kymriah from Novartis Approved as First U.S. CAR-T Cell Therapy

History was made this month as the U.S. Food and Drug Administration (FDA) approved the first U.S. cell-based gene therapy known collectively as chimeric antigen receptor (CAR) T-cell therapy. Kymriah (tisagenlecleucel or CTL019) from Novartis was approved for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Kymriah is a genetically-modified T-cell immunotherapy customized for each patient using their own T-cells, a type of white blood. In one study of 63 patients with relapsed or refractory B-cell precursor ALL, the overall remission rate within three months of treatment was 83 percent. Kymriah contains a boxed warning for cytokine release syndrome (CRS) and neurotoxicity. To learn more, visit the slideshow: CAR T-Cell Therapy: A Healthcare Professional's Guide - The Top 10 Questions Answered.

Cyltezo: Boehringer Ingelheim Wins Approval on Their First Biosimilar

Cyltezo (adalimumab-adbm) is an anti-TNF-α monoclonal antibody biosimilar to Humira, now approved in a pre-filled syringe for the treatment of various inflammatory diseases including rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, and plaque psoriasis. It can be used alone or in combination with methotrexate or other non-biologic disease-modifying anti-rheumatic drugs. (DMARDs). No FDA Advisory committee convened for discussion of Cyltezo; it was approved based on a comprehensive data package comprised of analytical, pharmacological, non-clinical and clinical development studies. The other biosimilar for adalimumab is Amgen’s Amjevita (adalimumab-atto).

Vabomere Clears FDA for Complicated Urinary Tract Infections

The FDA has approved intravenous Vabomere (meropenem and vaborbactam) for adults with complicated urinary tract infections (cUTI), including pyelonephritis caused by specific bacteria. Vabomere contains meropenem, an antibacterial, and vaborbactam, a β-lactamase inhibitor which inhibits certain types of resistance mechanisms used by bacteria. In studies with 545 adults with cUTI, including those with pyelonephritis, roughly 98% of patients treated with Vabomere had a cure or improvement in symptoms and a negative urine culture test. The most common adverse reactions in patients taking Vabomere were headache, infusion site reactions and diarrhea, among others.

Pfizer's Besponsa FDA-Approved for Advanced Forms of ALL

Life expectancy after a return of acute lymphoblastic leukemia is low and treatment options are few. In response, the FDA has approved Pfizer's Besponsa (inotuzumab ozogamicin), a CD22 monoclonal antibody and calicheamicin cytotoxic agent conjugate for relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in adults. Besponsa binds to B-cell ALL cancer cells that express the CD22 antigen blocking the growth of cancerous cells. In studies, 35.8% of those who received Besponsa experienced complete response (CR) for a median 8 months; of the patients who received alternative chemotherapy, 17.4 percent experienced CR for a median 4.9 months.

Mavyret Cleared as First 8-Week Hepatitis C Treatment for All Genotypes

The FDA has approved AbbVie's oral Mavyret (glecaprevir and pibrentasvir), the first 8-week treatment for all chronic hepatitis C virus (HCV) genotypes (1-6) in adult patients without cirrhosis (liver disease) and without previous treatments. Standard treatment length for HCV was previously 12 weeks or more. Glecaprevir inhibits NS3/4A protease and pibrentasvir inhibits NS5A. Mavyret is approved for two patient populations: either chronic HCV genotype 1, 2, 3, 4, 5 or 6 infection without cirrhosis or with compensated cirrhosis (Child-Pugh A), or adult patients with HCV genotype 1 infection, previously treated with an HCV NS5A inhibitor or an NS3/4A protease inhibitor, but not both. Studies demonstrated that 92 to 100 percent of patients had no virus detected in the blood 12 weeks after finishing treatment, suggesting a cure. The most commonly reported adverse reactions (> 10%) are headache and fatigue.

Posted: August 2017