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 2016, over 70,000 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 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 drug discovery efforts focus on the design, synthesis and optimization of low molecular weight compounds 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 CNS disorders and infectious diseases.
Our late-stage product candidates include cadazolid, a novel antibiotic under investigation for Clostridium difficile-associated diarrhea (CDAD), and ponesimod, an S1P1 receptor modulator investigated in multiple sclerosis.
In addition to advancing our own pipeline, Actelion is continuously looking externally for innovative compounds addressing diseases with unmet medical need which have the potential to change the standard of care. In November 2016, the company announced that it had obtained an option to in-license vamorolone from ReveraGen. Vamorolone, a novel compound for the treatment of Duchenne muscular dystrophy, holds potential to better preserve muscle function and prolong ambulation for the patient, without some of the side effects associated with glucocorticoid therapy.
Our commitment to patients is reflected by the patient access programs we have established in many countries. In Australia, only one PAH therapy per patient is currently reimbursed by the government and Actelion’s programs have allowed access to combination therapy for hundreds of patients who would not otherwise be able to receive optimum treatment. In the US, our programs have assisted more than 7,500 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, our financial assistance programs help to reduce out-of-pocket copayment costs. In addition, Actelion provides funding 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, global guidelines are 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.
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 information resources for patients living with a serious disease. Operating on a non-profit basis, they 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 such 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 to bring about positive change. Our activities include:
Our continued global advocacy work has strengthened Actelion’s position as a leader in our therapeutic areas and, importantly, as a trusted partner of the wider rare diseases community. In recognition of this commitment, the European Organisation for Rare Diseases honored Actelion with the EURORDIS Company Award for 2016. “This award is very special and important for Actelion as it acknowledges our long-term efforts and commitment to rare diseases. Managing a rare disease is much more than providing a drug, it includes an open and fruitful collaboration with all stakeholders. We are working together with over 120 patient organizations worldwide to support disease awareness, education and patient-centric initiatives.” Alessandro Maresta (Head of Global Medical Affairs)
In September, at the Evening of Hope Gala held by the California Chapter of the Pulmonary Hypertension Association, Jean-Paul and Martine Clozel were presented with the Chapter’s 2016 Legacy Award for their dedication as pioneers in the field of PAH.
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 into outcomes that are not amenable to clinical or laboratory evaluation, such as severity of pain or impact of disease on daily activities. In 2016, we introduced the Insomnia Daytime Symptoms and Impacts Questionnaire, thus bringing the total number of PRO projects to five:
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:
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.