Bayer receives approval for Adempas as first drug to treat rare heart and lung disease
Bayer Inc. announced today the Health Canada approval ofAdempas® (riociguat). Adempas® is the first drug indicated to manage the treatment of inoperable, or persistent /recurrent chronic thromboembolic pulmonary hypertension (CTEPH) after surgery in adult patients with World Health Organization Functional Class II or III pulmonary hypertension.1,2
CTEPH is an uncommon form of pulmonary hypertension (PH), a severe, progressive and
«Before today, we had no proven drug treatments for patients with inoperable CTEPH or patients in whom surgery was not successful in curing their CTEPH. Adempas gives us an effective drug treatment with proven clinical efficacy and good tolerability," said Dr. John Granton, Head, Division of Respirology University Health Network, Mount Sinai Hospital, and Women’s College Hospital. He added that, because of the complex nature of PH, «Patients should be referred to an expert PH centre as soon as possible for a thorough assessment and timely treatment.»
Results from a major clinical trial showed that riociguat is the first ever drug to provide statistically significant clinical improvement in patients with inoperable CTEPH or persistent/recurrent PH disease at the end of 16 weeks of treatment. Improvements were seen in a range of
«Riociguat will be welcomed by patients with CTEPH who, up to now, have not had a proven drug treatment option available to them if their disease is inoperable or for those experiencing residual PH following surgery. CTEPH is a devastating diagnosis, and the symptoms of breathlessness, dizziness and fainting can be frightening and have a severe impact on daily activities. To have a treatment that achieves meaningful clinical improvements is a much needed step forward," said Frank Poon, President, Pulmonary Hypertension Association of Canada.
The standard and potentially curative treatment option for patients who have developed CTEPH is a surgical procedure called pulmonary endarterectomy that mechanically clears the blood vessels of the lungs of scar tissue caused by the disease.10 However, the disease persists or recurs after surgery in up to 35% of patients.11 Many patients (20%-50%)12,13 with CTEPH are not candidates for surgery and, like patients with residual PH, urgently need effective new treatments to manage their disease.14
About Pulmonary Hypertension
PH is a severe, progressive,
There are five different types of PH and each can affect the patient in a different way and every patient may have a different etiology and manifestation of PH.8,10,16 For the best chance of success patients need to be treated at an expert PH centre. 8,19
About Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
CTEPH is a progressive and
Riociguat is a soluble guanylate cyclase (sGC) stimulator, the first member of a novel class of compounds developed by Bayer to target a key molecular mechanism underlying PH. sGC is an enzyme found in the cardiopulmonary system and the receptor for nitric oxide (NO). When NO binds to sGC, the enzyme enhances synthesisof the signaling molecule cyclic guanosine monophosphate (cGMP). cGMP plays an important role in regulating vascular tone, proliferation, fibrosis, and inflammation.
PH is associated with endothelial dysfunction, impaired synthesis of NO and insufficient stimulation of sGC. Riociguathas a unique mode of action — it sensitizes sGC to endogenous NO by stabilizing the
With its novel mode of action, riociguat has the potentialto overcome a number of limitations of currently approved PAH therapies, including NO dependence. It is the first drug which has shown clinical benefits in CTEPH, where, until the approval of riociguat, no indicated pharmacological treatment was available.