Actelion’s mission is to treat more patients with groundbreaking therapies. With our growing portfolio of innovative medicines, we are serving increasing numbers of patients in countries throughout the world. At the end of 2015, over 65,000 patients were benefiting from Actelion’s drugs. We are leaders in the science and treatment of pulmonary arterial hypertension (PAH), with over 15 years’ experience in this area. Our understanding of the complex pathways and molecular mechanisms of this disease has enabled the development of tailored medicines that can make a real difference to patient outcomes.

 

 

Innovation is the cornerstone of our current and future success. We are set to continue our leadership in the field of PAH into the mid-2020s, thanks to our assets Veletri®, Opsumit® and Uptravi® (developed in partnership with Nippon Shinyaku).

Our expertise in human biology – especially our knowledge of specific families of molecular targets such as G protein-coupled receptors – led us to Opsumit and will facilitate advances into new areas.

Actelion’s efforts in drug discovery focus on the design, synthesis and optimization of low molecular weight molecules, which are active on molecular target families. This focus allows high productivity in the generation of innovative compounds potentially addressing a wide range of high unmet medical needs.

Initially, the company looked solely at G-protein coupled receptors (GPCRs) and a specific enzyme family known as aspartic proteinases. As the company’s capabilities have expanded, so have the target platforms, adding anti-infectives, ion channels and a broad range of soluble enzymes.

Actelion’s promising development pipeline comprises novel compounds addressing a broad range of diseases, including cardiovascular and immunological disorders as well as central nervous system disorders and infectious diseases.

Our late-stage product candidates include cadazolid, a novel antibiotic under investigation for Clostridium difficile-associated diarrhea (CDAD), and ponesimod, a S1P1 receptor modulator investigated in multiple sclerosis.

Our commitment to patients is reflected by the patient access programs we have established in many countries. For example, in the United States, our programs have assisted more than 6,000 patients since 2012. Such assistance includes the provision of treatment at no charge for eligible patients who do not have healthcare coverage and lack financial means. For eligible patients with commercial insurance, we have financial assistance programs to help reduce their out-of-pocket copayment costs. In addition, Actelion provides funding grants to third-party, non-profit organizations that may offer support to patients.

In other parts of the world, where Actelion’s drugs are not approved or reimbursed, we have global guidelines in place to facilitate access, while ensuring full compliance with local regulations. We also continue to work with governments and other stakeholders to widen accessibility in geographies around the world.

In Least Developed Countries, Actelion does not file or enforce existing patents on medicines, so as not to hinder access to Actelion’s medicines.

To gain deeper insights into patients’ day-to-day needs and the challenges they face, Actelion continues to strengthen its collaboration with patients and patient organizations around the globe.

Patient organizations can provide an important support network and source of information for patients living with a severe disease. They operate on non-profit basis and help to raise awareness of treatment options and advocate for better patient care.

Actelion believes that patient organizations should be independent, and we abide by the relevant codes of practice for interacting with patient organizations.

Through our collaborative work with patient groups and healthcare professionals and as part of our advocacy function, we lead and support a variety of far-reaching activities that raise awareness of the challenges facing those living with rare diseases and help bring about positive change. Our activities include:

  • the development of educational resources for patients such as our innovative PAHuman eBook to better explain the disease and its impact;
  • raising disease awareness through educational campaigns such as our international Think NP-C campaign to encourage earlier diagnosis of Niemann-Pick type C disease among health care professionals unfamiliar with this rare condition;
  • the support of parliamentary events to help shape health policy;
  • sharing expert and patient insights to inspire and empower others to live well with the disease;
  • stakeholder meetings and training;
  • surveys to better understand the patient and care-giver experience.

Currently, Actelion is conducting a clinical study with macitentan in Eisenmenger syndrome patients. Eisenmenger syndrome is the most advanced form of pulmonary arterial hypertension (PAH) in conjunction with congenital heart disease (CHD). Patients with Down syndrome represent an important subset of the Eisenmenger population (between 25% and 50%, depending on cohort studied). To address the high unmet medical need for effective, targeted PAH therapies in this vulnerable population, the study has been extended to the Down syndrome community. To ensure proper safeguards are established to protect the patients' rights and safety, we have been working with ethics committees, patient advocacy, support groups and patients’ families. Our outreach was incredibly well received by all parties involved, and their support has enabled us to enroll a number of patients with Down syndrome into the study. This is the first time that patients with Down syndrome afflicted with Eisenmenger syndrome are included in a randomized clinical trial.

Our continued global advocacy work has strengthened Actelion’s position as a leader in our respective therapy areas and, importantly, as a trusted partner of the wider rare diseases community. 

Improving health outcomes for patients is our main goal. We use patient-reported outcome (PRO) questionnaires to measure and provide information on the benefits of treatment from the patient’s perspective. PROs essentially reflect the voice of the patient, independent from the input and interpretation of physicians, other medical experts and carers. They provide insight on the outcomes that cannot easily be evaluated using clinical and laboratory means, such as severity of pain or the impact of the disease on daily activities.  We currently have 4 PRO projects: 

-          PAH-SYMPACT® (for pulmonary arterial hypertension)
-          HDISS-DU® (for digital ulcers)
-          FSIQ-RMS® (for relapsing forms of multiple sclerosis)
-          CDAD-DaySyms® (for Clostridium difficile-associated diarrhea)

Learn more about our interaction with patient associations on www.actelion.com

The safety of the patients we serve is of paramount importance. Our products are marketed and distributed with thorough labeling and product information. We work with regulators to develop prescribing information that provides the benefit and risk information healthcare professionals require. Comprehensive and rigorous systems for detecting and evaluating adverse effects ensure that any new safety data is immediately provided to regulators and healthcare professionals.

As the quality of our products is crucial to patient safety, we ensure that they are manufactured to the highest global standards. Our focus on quality assurance encompasses the complete product life cycle – from initial use in clinical trials to use in physicians’ practices.

Rather than manufacturing its own commercial drugs, Actelion outsources the process to contract manufacturers. The supply chain is subject to thorough risk assessment and regular audits. Depending on the product, security of product supply is assured through back-up suppliers and inventory management.

In view of the risks associated with counterfeit or adulterated drugs, we are working diligently to expand our serialization and track-and-trace systems, so as to ensure delivery of safe, secure and effective products to our patients.

Our product security and brand protection programs include the following measures:

  • Adoption of sophisticated product packaging technologies
  • Continuous auditing and monitoring of manufacturing and supply chain partners
  • Monitoring of drug sales and potential threats to the supply chain from counterfeit or diverted products

Patient organizations can be an important support network and source of information for patients who are living with illnesses. They are non-profit organizations that help to raise awareness of treatment options and advocate for better patient care.

Actelion believes that patient organizations should be independent, and we abide by the relevant codes of practice for interacting with patient organizations.

Medical Science Liaison, Greece 

 

"It's a tremendous feeling to have a positive impact on a person's life."

 

Grants & Donations

Actelion has either provided grants or given donations to the following patient organizations:

Actelion has either provided grants or given donations to the following patient organizations: