Rethymic (allogeneic processed thymus tissue-agdc) for Pediatric Congenital Athymia

Rethymic (allogeneic processed thymus tissue-agdc) is a one-time regenerative tissue-based therapy for immune reconstitution in pediatric patients with congenital athymia. It is the first thymus tissue product approved in the US. The product is implanted in the quadriceps muscle through a surgical procedure; dosing is customized to the patient. The approval was based on data from 10 prospective, single-center, open-label clinical studies that included a total of 105 patients aged 1 month to 16 years with congenital athymia. Each patient received a single administration of Rethymic.

More information on Rethymic.

Scemblix (asciminib) for Ph+ Chronic Myeloid Leukemia in Chronic Phase

Scemblix (asciminib), an oral ABL/BCR-ABL1 tyrosine kinase inhibitor (STAMP inhibitor), received accelerated approved for the treatment of adults with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitors. The accelerated approval was based on data from the phase 3 ASCEMBL study (ClinicalTrials.gov Identifier: NCT03106779), which included 233 patients. The FDA also granted full approval to Scemblix for the treatment of adults with Ph+ CML in CP with T315I mutation based on data from a multicenter, open-label phase 1 study (N=45; ClinicalTrials.gov Identifier: NCT02081378).

Review the drug monograph here.


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Tavneos (avacopan) for ANCA-Associated Vasculitis

Tavneos (avacopan), a first-in-class oral complement inhibitor, was approved for use as an adjunctive treatment for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (granulomatosis with polyangiitis and microscopic polyangiitis) in combination with standard therapy including glucocorticoids.  The approval was based on data from the double-blind, active-controlled ADVOCATE trial (ClinicalTrials.gov Identifier: NCT02994927), which evaluated the efficacy and safety of avacopan in 330 adults with newly diagnosed or relapsed ANCA-associated vasculitis.

Review the drug monograph here.

Tecartus (brexucabtagene autoleucel) for Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia

Tecartus (brexucabtagene autoleucel), an autologous, anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, was approved for adult patients 18 years of age and older with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). It is the first CAR T-cell therapy approved for adults with ALL. The approval was based on data from the ZUMA-3 trial (ClinicalTrials.gov Identifier: NCT02614066), which evaluated the efficacy and safety of Tecartus in 71 adults with relapsed or refractory B-cell precursor ALL.

Review the drug monograph here.

Tyrvaya (varenicline solution) Nasal Spray for Dry Eye Disease

Tyrvaya (varenicline solution) is the first nasal spray approved for the treatment of signs and symptoms of dry eye disease. Tyrvaya contains varenicline, a highly selective nicotinic acetylcholine agonist, which stimulates natural tear film production by activating the trigeminal parasympathetic pathway in the nasal cavity. The approval was based on data from the phase 3 ONSET-2 (ClinicalTrials.gov: NCT04036292), phase 2b ONSET-1 (ClinicalTrials.gov: NCT03636061), and phase 2 MYSTIC trials that included over 1000 patients with mild, moderate or severe symptoms of dry eye disease.

Review the drug monograph here.

Reference

  1. Rethymic. Package Insert. Enzyvant Therapeutics, Inc.; 2021. https://enzyvant.com/wp-content/uploads/2021/10/prescribing-information.pdf.
  2. Scemblix. Package. Novartis Pharmaceuticals Corporation; 2021.  https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215358s000Orig1lbl.pdf.
  3. Tavneos. Package insert. ChemoCentryx, Inc; 2021. https://www.chemocentryx.com/wp-content/uploads/2021/10/FINAL-PI-and-Med-Guide-7-Oct-21.pdf.
  4. Tecartus. Package insert. Kite Pharma, Inc.; 2021. https://www.gilead.com/-/media/files/pdfs/medicines/oncology/tecartus/tecartus-pi.pdf.
  5. Tyrvaya. Package insert. Oyster Point Pharma; 2021. https://www.tyrvaya-pro.com/files/prescribing-information.pdf.