Swiss drugmaker Roche has formed an alliance with the UK’s Department of Health to conduct a series of clinical trials aimed at determining whether the company’s arthritis biologic, tocilizumab (or Actemra), is effective at treating pulmonary arterial hypertension (PAH). The UK’s National Institute for Health Research (NIHR) will be funding the trials in an effort to identify a treatment for PAH which would slow progression of the disease.
PAH is a rare disease that is characterized by the narrowing of pulmonary arteries around the heart, restricting blood flow to the lungs. Symptoms of the disease include weakness, fatigue and angina, and patient have less than a 40 percent survival rate after five years post diagnosis.
Current treatments for PAH – including Actelion’s Uptravi – can help to lessen the symptoms of the disease and slow disease progression, however there is no cure for PAH. Papworth Hospital NHS Foundation Trust and Cambridge University Hospitals NHS Trust will be leading and hosting the clinical trials, respectively, which will require an enrollment of approximately 50 patients.
“Being able to look at the in-depth science of how one of our current treatments could be applied to a real unmet medical need is what attracted us to carry out this work in the UK,” said Madhi Farhan, Head of the Office of I2O Innovation at Roche. “We hope this research will soon lead to benefit for patients with this debilitating disease.”
Roche’s Actemra was first approved by the FDA in 2010, with an indication for treating rheumatoid arthritis. If the drug is effective in clinical trials at slowing disease progression of PAH, this could be Roche’s opportunity to break into the profitable orphan drug space.
“This innovative and exciting trial of an available biological drug is further evidence for the success of the NIHR in collaborating with industry on the development of groundbreaking therapies,” said Mark Samuels, managing director of the Office for Clinical Research Infrastructure (NOCRI). The NIHR’s Rare Disease Collaboration – supported by £20 million in investment capital – was established in 2013 with the aim of accelerating the rate of rare disease research.
Along with the newly FDA-approved Uptravi, Actelion also plays a role in marketing other PAH treatments including Veletri and Ventavis. Actelion has announced they plan to price Uptravi at $160,000 per patient, and will market the drug as a second-line treatment for patients with mid-stage PAH.
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