This designation is given to medications with the potential to substantially improve care for rare diseases — defined as those that affect fewer than five individuals per 10,000 people in Europe. The designation comes with certain incentives, including scientific advice and assistance with clinical trial protocols, and the potential for a 10-year period of market exclusivity if the medication is eventually approved.
“We’re thrilled at the European Commission’s decision to grant orphan designation to sotatercept in PAH,” Habib Dable, president and CEO of Acceleron Pharma, the company developing sotatercept, said in a press release.
Bone morphogenic protein (BMP) signaling is a molecular pathway known to play a key role in the maintenance of healthy lung blood vessels. Disturbances in this pathway have been linked with the development of PAH, suggesting that targeting it may be an effective treatment strategy.
Sotatercept (ACE-011) is a lab-made protein designed to bind and trap certain types of proteins, including some involved in the BMP signaling pathway, thereby normalizing aberrant BMP signaling in PAH. It is administered via subcutaneous (under-the-skin) injection.
Sotatercept previously received priority medicines (PRIME) designation in the European Union for the treatment of PAH. It also has received orphan drug and breakthrough therapy designations in the U.S. for the same indication.
“We fully intend to take advantage of the benefits that this [orphan drug designation in Europe] and other special statuses — including Orphan Drug and Breakthrough Therapy designations in the United States and PRIME designation in Europe — provide to drug developers as we work with health authorities to deliver this potential new backbone therapy in PAH to patients in need as quickly as possible,” Dable said.
Early data from the ongoing Phase 2 clinical trial SPECTRA (NCT03738150) indicated that sotatercept improves heart health and exercise capacity in people with PAH. The open-label study, sponsored by Acceleron, is recruiting participants in Tucson, Arizona; Boston, Massachusetts; Rochester, Minnesota; and Pittsburgh, Pennsylvania. More information is available here.
Acceleron recently announced plans to start the first Phase 3 clinical trial of sotatercept in PAH, called STELLAR, before the end of this year. Two other Phase 3 trials — HYPERION and ZENITH, testing the investigational medication as an early- or late-stage treatment, respectively — are planned to start in 2021.
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