EU Panel Backs Bayer's Riociguat for Pulmonary Hypertension


Source: Drug Discovery and Development


The European Committee for Medicinal Products for Human Use (CHMP) recommended approval for riociguat tablets for use in two forms of pulmonary hypertension, a group of life-threatening and progressive diseases: The treatment of adult patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH) or persistent or recurrent CTEPH after surgical treatment to improve exercise capacity, and the treatment of adult patients with pulmonary arterial hypertension (PAH) to improve exercise capacity.
Riociguat is the first of a novel class of drugs, the soluble guanylate cyclase (sGC)-stimulators. Its new mode of action has been developed to target a key molecular mechanism underlying pulmonary hypertension. The final decision of the European Commission on the approval is expected in the first half of 2014.
"We appreciate today’s recommendation by the CHMP to approve riociguat, and we look forward to bringing riociguat to patients with these rare, serious and life-threatening diseases upon approval of the European Commission," said Kemal Malik, member of the Bayer HealthCare Executive Committee and head of Global Development. "Riociguat is the first drug to demonstrate efficacy in two forms of pulmonary hypertension, PAH and CTEPH. For CTEPH, pulmonary endarterectomy is the primary and potentially curative treatment option. There is no drug treatment approved to date in the European Union - however, a considerable number of CTEPH patients are not operable, and in up to 35% of operated patients the disease persists or reoccurs." 
"The consistency and robustness of the positive results from the clinical trial programs CHEST and PATENT are substantial: Riociguat has demonstrated significant and sustained clinical efficacy and safety in treatment-naïve PAH patients and patients pre-treated with endothelin-receptor antagonists (ERAs) or prostanoid monotherapy, as well as in patients with inoperable CTEPH or persistent or recurrent CTEPH after surgery. So far no other pharmacological treatment has shown significant effect in CTEPH," said Principal Investigator Professor Ardeschir Ghofrani, University Hospital Giessen and Marburg, Germany. "For both PAH and CTEPH patients and their treating physicians, the clinical improvements across multiple endpoints such as 6 Minute Walking Distance, cardiopulmonary hemodynamics, WHO Functional Class and a disease-related biomarker in the treatment with riociguat are of high relevance." 
The CHMP recommendation is based on results from the two randomized, double-blind, placebo-controlled, global Phase 3 studies CHEST-1 and PATENT-1 as well as on interim results of the long-term extension studies CHEST-2 and PATENT-2 available at the time. These assessed the efficacy and safety of oral riociguat in the treatment of CTEPH and PAH respectively. PAH and CTEPH are two rare and life-threatening forms of pulmonary hypertension characterized by significantly increased pressure in the pulmonary arteries.
Both Phase 3 studies with riociguat met their primary endpoint, a change in exercise capacity, after 16 and 12 weeks respectively. Riociguat also demonstrated consistent improvements across multiple, relevant secondary endpoints, and was generally well-tolerated, with a good safety profile. Results of both studies were published in the New England Journal of Medicine (NEJM) in July 2013. The Phase 3 trial programs CHEST and PATENT are ongoing with the long-term extension studies, CHEST-2 and PATENT-2, showing in first interim analyses a good safety profile and sustained efficacy over one year. The most commonly reported adverse reactions, occurring in greater than or equal to 10% of patients under riociguat treatment (up to 2.5 mg TID), were headache, dizziness, dyspepsia, peripheral edema, nausea, diarrhea, and vomiting. 
Riociguat was approved under the name Adempas in the United States for use in CTEPH and PAH in October 2013, and in Canada and Switzerland in the CTEPH indication in September and November 2013, as well as in Japan in CTEPH in January 2014. In February 2013, Bayer HealthCare submitted riociguat for regulatory approval in the European Union in both indications.


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