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TGA Approved Zolgensma | TGA Drug approvals

Updated: Jun 27, 2023

03 May 2019

TGA approved Yondelis for liposarcoma

TGA approved the new chemical entity, Yondelis (trabectedin), for treating patients with unresectable or metastatic liposarcoma or leiomyosarcoma. The drug is indicated in patients with prior anthracycline-containing treatment.

The drug acts by binding to the minor groove of DNA, thereby affecting a cascade of events, including DNA binding proteins, and DNA repair pathways, resulting in perturbation of the cell cycle.

The drug is marketed by Specialised Therapeutics in Australia.

16th April 2021

TGA approved Adakveo for sickle cell disease

Australian Therapeutic Goods Administration (TGA) announced that it had approved a new biological entity, Adakveo (crizanlizumab), to prevent recurrent vaso-occlusive crises in patients with sickle cell disease. The drug is indicated for patients aged 16 years and older.

Adakveo, a selective immunoglobulin G2 (IgG2) kappa humanized monoclonal antibody (mAb), acts by blocking the interaction of P-selectin with its ligands, including P-selectin glycoprotein ligand 1 (PSGL-1). P-selectin-mediated multi-cellular adhesion is a critical factor in the pathogenesis of vaso-occlusion and vasoocclusive crises. Blocking the interaction prevents the interaction between endothelial cells, platelets, red blood cells, and leukocytes, thereby preventing vaso-occlusion.

The Swiss giant, Novartis, developed Adakveo.

March 24, 2021

TGA approved Vertex Pharma’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for cystic fibrosis patients of 12 years and older

Vertex Pharmaceuticals has announced the Australian Therapeutic Goods Administration (TGA) has announced the approval of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for cystic fibrosis patients of 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

As per the company report, about 90% of patients with cystic fibrosis have at least one F508del mutation. Further, the company has estimated that around 750 patients with cystic fibrosis might benefit from the treatment.

The approval is based on the global Phase 3 trials which included the centers of Australia as well.

10th March 2021

Zolgensma is approved for SMA patients in Australia

Therapeutic Goods Administration announced that it approved Zolgensma (onasemnogene abeparvovec) for treating pediatric patients with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 1 to 3 copies of the SMN2 gene.

The approval is based on the AVXS-101-CL-303 trial, a Phase-3, open-label, single-arm study. Pediatric patients of 0.5 months to 5.9 months and weight of 3.9 to 7.5 kg were included in the trial. The number of patients sitting straight for 30 seconds without support and event-free survival (patients without ventilation) was considered primary endpoints.

The product comes up with a black triangle scheme; the product received orphan designation on 13 November 2019.

A mutation in the survival motor neuron 1 (SMN1) gene results in inadequate expression of survival motor neuron (SMN) protein. Zolgensma delivers the transgene that codes for the human survival motor gene protein.


TGA approved Novartis Luxturna in Australia

Australian Therapeutic Goods Administration has approved Luxturna (voretigene neparvovec) for treating patients with inherited retinal dystrophy because of biallelic RPE65 mutations.

Luxturna is gene therapy; the drug acts by delivering a copy of the gene that codes the retinal pigment epithelial 65 kDa protein in patients who lacks biologically active RPE65. Patients with inherited retinal dystrophy have visual impairment because of biallelic RPE65 mutation, which leads to reduced or absence of RPE65 isomerohydrolase activity. This impacts the visual cycle.

The efficacy of Luxturna was evaluated in 31 patients. Of the 31 patients, Luxturna was administered in 21 patients. Ten subjects were included as a control group, among whom nine patients were randomized and treated with Luxturna after one year of observation. The efficacy was measured using the multi-luminance mobility testing (MLMT) score. Eleven of twenty-one patients who were on Luxturna had an MLMT score change of two or more, whereas only one patient in the control group had a difference in the MLMT score.

The drug is approved in the black triangle scheme. Luxturna received orphan designation in 2019.

The product has already approved in other major markets, including the United States, Europe.


TGA approved Tukysa for breast cancer in Australia

The Australian Therapeutic Good Administration has given a positive recommendation for Tukysa (tucatinib) for treating breast cancer. The drug is administered in combination with trastuzumab and capecitabine for patients suffering from advanced unresectable or metastatic HER2-positive breast cancer. The drug is indicated after treating with one or more anti-HER2 based regiments.

Tucatinib is a tyrosine kinase inhibitor, acts by inhibiting the phosphorylation of HER2 and human epidermal growth factor receptor (HER3), which eventually leads to mitogen-activated protein kinase (MAPK) inhibition and SKT signaling and cell proliferation.

AA-Med Pty Ltd is the sponsor of Tukysa in Australia.

The product has been approved in other countries, including the United States and Switzerland.

Seattle Genetics has market authorization for the product in the United States. The approval in the US is based on a randomized, double-blind, placebo-controlled clinical trial that enrolled 612 patients. Progression-free survival was considered as the primary outcome. 7.8 was the median months in Tukysa + trastuzumab + capecitabine compared to 5.6 months in placebo + trastuzumab + capecitabine.


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