This is a pdf file of an unedited manuscript that has been accepted for publication. Nda 204819 adempas fda approved 12 jan 2018 full prescribing information warning. Riociguat had no effect on gas exchange or ventilationperfusion matching and was well tolerated. Interpretation riociguat may be used long term in patients with cteph. Riociguat adempas pulmonary hypertension association. Riociguat, a member of a new class of compounds soluble guanylate cyclase stimulators, has been shown in previous clinical studies to be beneficial in the treatment of chronic thromboembolic. In the treatment of inoperable cteph, riociguat is the only drug to consistently demonstrate clinical efficacy in a placebocontrolled study.
Riociguat constitutes the first drug of the class of sgc stimulators. Something new in pulmonary hypertension therapeutics. Riociguat is a firstinclass drug approved for the treatment of pah as a monotherapy or added to endothelinreceptor antagonists as a sequential combination therapy, and for the. Riociguat, a soluble uanylate cyclase sgc stimulator, is the first member of a new class of compounds. Predictors of longterm outcomes in patients treated with. Background the 12week, phase iii pulmonary arterial hypertension sgcstimulator trial patent1 study investigated riociguat in patients with pulmonary arterial hypertension pah. See full prescribing information for complete boxed warning do not administer adempas to a pregnant female because it may cause fetal harm. Serious adverse events were seen in 129 54% of 237 patients, and 14 6% discontinued riociguat therapy because of adverse events. Eligibility is often based on income, insurance or medicare status, and other factors. Pulmonary arterial hypertension is a rare but lifethreatening illness affecting around 10,000 people in the u. Mar 20, 2014 expert, exposure registry riociguat in patients with pulmonary hypertension expert the safety and scientific validity of this study is the responsibility of the study sponsor and investigators. The current study by rosenkranz et al is to be commended as it illustrates the potential of riociguat in selected patients with chdpah.
Riociguat trade name adempas is a drug by bayer that is a stimulator of soluble guanylate cyclase sgc. At week 16, the 6minute walk distance increased from baseline by a mean of 39m in the riociguat group, contrasted with a mean decrease of 6m in the placebo group difference of 46m, 95%ci 25 to 67, p riociguat versus an increase of 23 dynseccm5 in the. Jan 26, 2017 twoyear study of riociguat as cteph treatment identifies predictive disease markers. Twoyear study of riociguat as cteph treatment identifies predictive disease markers. Riociguat is also used to treat pulmonary arterial hypertension pah. Pulmonary hypertension riociguat clinical trial scleroderma. Riociguat could thus well represent an alternative to pde5 inhibitors in this setting in future.
In an early hemodynamic study evaluating doses of 1 mg or 2. Over the past two decades, we have witnessed significant improvements in the therapy of pulmonary arterial hypertension pah, in general, but also specifically in pah associated with congenital heart disease chdpah. Estimated survival and clinical worseningfree survival rates were 93% and 73%, respectively. Riociguat bay 632521 is a stimulator of sgc that works both in synergy with and independently of no to increase levels of cgmp. Riociguat for the treatment of pulmonary arterial hypertension. Riociguat was well tolerated by most patients and no new safety signals were identi. Practical management of riociguat in patients with pulmonary arterial.
Chris cannon discusses two studies, chest1 and patent1, of pulmonary hypertension patients and found that those who were taking riociguat showed significant improvement over those taking a placebo. Efficacy and safety of riociguat in patients with systemic sclerosis the safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Pregnancy tests will also be done every month while you take. With riociguat we have for the first time an effective drug treatment for two forms of ph. Riociguat for the treatment of chronic thromboembolic. Adempas bayer for oral treatment of pulmonary arterial hypertension pah and chronic thromboembolic pulmonary hypertension cteph following surgery or when surgery is not an option. Nov 25, 2014 in the study, the researchers will assess if treatment with riociguat for dcssc over 52 weeks performs better than a placebo. Background riociguat, a soluble guanylate cyclase stimulator, has been shown in a phase 2 trial to be beneficial in the treatment of pulmonary arterial hypertension.
Riociguat is used to treat chronic thromboembolic pulmonary hypertension cteph in people who cannot be treated with surgery, or in people who have undergone surgery but still have symptoms. Us fda committee recommends approval of bayers riociguat. Nov 05, 2014 efficacy and safety of riociguat in patients with systemic sclerosis the safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Riociguat is available to women only under a special program. Downloaded from at klinische bibliothek univ on august, 20. Here, we present a prospectively planned analysis of the safety and efficacy of riociguat in the subgroup of patients with pah associated with connective tissue disease pahctd. Riociguat can treat certain symptoms of cteph and can improve your ability to exercise. A pregnancy test will be done before starting riociguat to show that you are not pregnant.
Ghofrani ha1, galie n, grimminger f, grunig e, humbert m, jing zc, keogh. Pah is caused by remodeling of the small pulmonary arteries, whereas cteph is a result of obstructive thromboembolic. Individual subscribers are entitled to free 24hour access to 50 archive articles per year. Pdf riociguat for the treatment of chronic thromboembolic. Data sources include ibm watson micromedex updated 4 may 2020, cerner multum updated 4 may 2020. Hayeon na, cocopy editor contentfocused on october 8 th of 20, bayers new drug riociguat adempas was approved for the treatment of patients whose pulmonary hypertension ph belongs in who groups 1 and 4. Riociguat for the treatment of pulmonary arterial hypertension nejm. Riociguat is a white to yellowish, crystalline, nonhygroscopic substance with a molecular weight of 422. Background riociguat, a member of a new class of compounds soluble guanylate cyclase stimulators, has been shown in previous clinical. Adempas is a prescription medicine used to treat adults with. Pdf riociguat for interstitial lung disease and pulmonary. The goal of this therapy for pah is to improve exercise ability, who functional class and delay clinical worsening.
Riociguat adempas new drug for pulmonary hypertension. The soluble guanylate cyclase stimulator riociguat. It is administered orally and is currently being trialled at a dose of 1. Riociguat is intended as a substitute monotherapy or as an addon to current treatment options. Riociguat, a soluble guanylate cyclase stimulator, has been shown in a phase 2 trial to be beneficial in the treatment of pulmonary arterial hypertension. Riociguat clinical trials for pulmonary hypertension. Youll need to apply through each program, either online, over the phone, or with your doctors help. Importance pulmonary arterial hypertension pah is characterized by severe remodeling of the distal pulmonary arteries, increased pulmonary vascular resistance, and right ventricular dysfunction that promotes heart failure. Riociguat, a soluble guanylate cyclase sgc stimulator, is approved for the treatment of both pah and inoperable cteph or persistentrecurrent cteph after pea. A read is counted each time someone views a publication summary such as the title, abstract, and list of authors, clicks on a figure, or views or downloads the fulltext. Whilst this document may be printed, the electronic version posted on the intranet is the controlled copy.
Efficacy and safety of riociguat in patients with systemic. This progress has been largely driven by the availability of new therapeutic agents that have been gradually introduced into clinical practice and have been demonstrated to. Riociguat clinical effects studied in pulmonary arterial hypertension pah patients with insufficient treatment response to pde5 inhibitors pde5i. New pulmonary hypertension drug riociguat proves effective. It is the first drug to be approved for treatment of cteph. Riociguat may also be used for purposes not listed in this medication guide. Embryofetal toxicity do not administer adempas to a pregnant female because it may cause fetal harm see contraindications 4. Us fda committee recommends approval of bayers riociguat in. You must be registered in the program and understand the risks and benefits of riociguat. Riociguat for pulmonary arterial hypertension and chronic. In this phase 3, doubleblind study, we randomly assigned 443 patients with symptomatic pulmonary arterial hypertension to receive placebo, riociguat in individually. Riociguat for pulmonary arterial hypertension innovation.
To view other topics, please sign in or purchase a subscription. In the treatment of inoperable cteph, riociguat is the only drug to consistently demonstrate clinical efficacy in. There are no data on presence of riociguat in human milk, effects on breastfed infant, or on milk production. Expert, exposure registry riociguat in patients with pulmonary hypertension expert the safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Adempas riociguat dosing, indications, interactions. Background the nitric oxide nosoluble guanylate cyclase sgccyclic guanosine monophosphate cgmp signaltransduction pathway is impaired in many cardiovascular diseases, including pulmonary arterial hypertension pah. Only patients remaining on riociguat at the respective timepoints were included in these analyses. This medicine may cause harm to the unborn baby if you take it while you are pregnant.
The drug riociguat is a soluble guanylate cyclase sgc activator approved under the name adempas from bayer as an oral treatment to target a crucial molecular mechanism essential for the development of pulmonary. Riociguat adempas for pulmonary hypertension the medical. Nejm journal watch concise summaries and expert physician commentary that busy clinicians need to enhance patient care. At week 12, the 6minute walk distance had increased from baseline by a mean of 30 m in the 2. Riociguat is also used to treat chronic thromboembolic pulmonary hypertension cteph. Riociguat is highly bound to plasma proteins 95% and is metabolized by cyp1a1, cyp3a, cyp2c8 and cyp2j2.
Females must not be pregnant when starting riociguat or become pregnant during treatment. Feb 18, 2020 riociguat can improve your ability to exercise and help prevent pah from getting worse. The drug was discovered by bayer and being developed as an oral treatment to target a key molecular mechanism underlying pulmonary hypertension. Riociguat can cause serious birth defects if taken during pregnancy. Riociguat, a new pulmonary hypertension treatment pulmccm. Expert, exposure registry riociguat in patients with. Any printed copies of this document are not controlled. Riociguat is an oral medication called a soluble guanylate cyclase stimulator approved for the treatment of pulmonary arterial hypertension pah in world health organization who group 1 patients. In solid form it is stable to temperature, light, and humidity. Initial trials of riociguat, an oral medication that stimulates limited options are available for treating patients with pulmonary hypertension. Listing a study does not mean it has been evaluated by the u.
Pulmonary arterial hypertension in the contemporary era. This material is provided for educational purposes only and is not intended for medical advice, diagnosis or treatment. It is used to treat two forms of pulmonary hypertension ph. Drugs for pah current management of pah usually includes warfarin. Riociguat is the first drug approved by the us food and drug administration for the treatment of both pah and inoperable or persistentrecurrent cteph 17, 22, 23. The first study, patent1, covered 443 patients and showed. Riociguat for pulmonary hypertension article pdf available in new england journal of medicine 36923. Riociguat in combination with prostacyclin analogs. Pdf on jul 25, 20, christian m kahler and others published riociguat for the treatment of chronic thromboembolic pulmonary hypertension. European commission approves bayers adempas for hypertension. Background riociguat, a soluble guanylate cyclase stimulator, has been shown in a phase 2 trial to be beneficial in the treatment of pulmonary. Riociguat is a firstinclass drug of the soluble guanylate cyclase stimulator class of drugs, which works by producing. Riociguat also demonstrated consistent improvements across multiple, relevant secondary endpoints, and was generally welltolerated, with a good safety profile. However, as long as headtohead comparisons between riociguat and pde5 inhibitors are unavailable, this has to be based on individual patient evaluation.
The fda has approved the sgc stimulator riociguat rye oh sig ue at. Riociguat is used to treat pulmonary arterial hypertension pah. Many programs are available from federal and state governments, nonprofits, manufacturers, and other organizations to help you get the drugs you need at a reduced cost. At week 16, a total of 77% of patients who were still participating in the study were taking the maximal riociguat dose of 2. Pulmonary hypertension is a condition defined by a mean pulmonary artery pressure greater than or equal to 25 mm hg at rest measured by right heart catheterization. As a service to our customers we are providing this early version of the manuscript. Pulmonary arterial hypertension in the contemporary era jama. Pulmonary arterial hypertension pah and chronic thromboembolic pulmonary hypertension cteph are serious and progressive diseases, characterized by an increase in pulmonary vascular resistance, which can ultimately lead to death due to right heart failure. Riociguat for pulmonary hypertension in congenital heart. Adempas riociguat dose, indications, adverse effects. Survival, clinical worsening free survival, time to addition of concomitant ph treatment, and time to discontinuation were analyzed using kaplanmeier plots up to month 76. It is important to prevent pregnancy while taking this medication and for 1 month after treatment. Riociguat definition of riociguat by medical dictionary. Results of both studies were published in the new england journal of medicine nejm in july 20.
Based on data from animal reproduction studies, therapy may cause embryofetal toxicity and miscarriage when administered to a pregnant woman and is contraindicated during pregnancy. In this phase 3, doubleblind study, we randomly assigned 443 patients with symptomatic pulmonary arterial hypertension to receive placebo, riociguat in individually adjusted doses of up to 2. Once regarded as largely untreatable, evidencebased decision making now guides clinical management of pah and improves outcomes. Riociguat plasma concentrations directly correlate to hemodynamic parameters such as systemic vascular resistance, systolic blood pressure, pulmonary vascular resistance, and cardiac output. Riociguat is one of several approved therapies available for patients with pulmonary arterial hypertension pah. For this reason, females who can become pregnant must have regular testing to insure they are not pregnant, or become pregnant during therapy or within one month of stopping riociguat. Riociguat for the treatment of chronic thromboembolic pulmonary. Riociguat for pulmonary arterial hypertension superseded docs if applicable contact details for further information document status this is a controlled document.