EMEA/CHMP/EWP/356954/2008: Guidelines on the Clinical Investigations of Medicinal Products for the Treatment of Pulmonary Arterial Hypertension; and CHMP/EWP/83561/2005: Guideline on Clinical Trials in Small Populations (effective December 2006). Regulatory status The international regulatory status at the time of this submission to TGA is listed in Table 1. Table 1: International regulatory status of Volibris at time of submission to TGA. Country Submission date Status Indications (approved or / region requested) EU 9 Dec 2014 CHMP: Volibris is indicated for positive treatment of pulmonary opinion arterial hypertension received on (PAH) in adult patients of 27 Oct 2015 WHO Functional Class (FC) EU: II to III, including use in Commission combination treatment Decision (see section 5.1). Efficacy estimated has been shown in by end of idiopathic PAH (IPAH) and Dec 2015 in PAH associated with connective tissue disease. US 5 Dec 2014 Approved: 2 Letairis is an endothelin Oct 2015 receptor antagonist indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO 1): - To improve exercise ability and delay clinical worsening. - In combination with tadalafil to reduce the risks of disease progression and hospitalisation for worsening PAH, and to improve exercise ability. Studies establishing effectiveness included trials predominantly in patients with WHO Functional Class II–III symptoms and etiologies of idiopathic or heritable PAH (60%) or PAH associated with connective tissue diseases (34%).
EMEA/CHMP/EWP/356954/2008: Guidelines on the Clinical Investigations of Medicinal Products for the Treatment of Pulmonary Arterial Hypertension; and CHMP/EWP/83561/2005: Guideline on Clinical Trials in Small Populations (effective December 2006). Regulatory status The international regulatory status at the time of this submission to TGA is listed in Table 1. Table 1: International regulatory status of Volibris at time of submission to TGA. Country / region Submission date Status Indications (approved or requested) EU 9 Dec 2014 CHMP: positive opinion received on 27 Oct 2015 EU: Commission Decision estimated by end of Dec 2015 Volibris is indicated for treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III, including use in combination treatment (see section 5.1). Efficacy has been shown in idiopathic PAH (IPAH) and in PAH associated with connective tissue disease. US 5 Dec 2014 Approved: 2 Oct 2015 Letairis is an endothelin receptor antagonist indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO 1): - To improve exercise ability and delay clinical worsening. - In combination with tadalafil to reduce the risks of disease progression and hospitalisation for worsening PAH, and to improve exercise ability. Studies establishing effectiveness included trials predominantly in patients with WHO Functional Class II–III symptoms and etiologies of idiopathic or heritable PAH (60%) or PAH associated with connective tissue diseases (34%).
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