EMA Grants Priority Medicines Designation to Sotatercept for PAH

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by David Melamed, PhD |

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The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to investigational therapy sotatercept (ACE-011) for the treatment of patients with pulmonary arterial hypertension (PAH).

This designation is given to medications in development that have the potential to treat conditions with an unmet medical need and that may offer an advantage over existing options.

For Acceleron Pharma, sotatercept’s developer, the new designation offers access to EMA resources to expedite the development process, with the overarching goal of getting sotatercept to patients as quickly as possible.

Sotatercept was also recently granted breakthrough therapy status by the U.S. Food and Drug Administration (FDA), providing similar benefits to Acceleron for the development of sotatercept in the U.S.

“Receiving PRIME designation for sotatercept from the EMA mere weeks after the FDA granted it Breakthrough Therapy designation further strengthens our belief that sotatercept could eventually alter the treatment landscape in PAH dramatically,” Habib Dable, president and CEO of Acceleron, said in a press release.

Sotatercept is designed to trap proteins of the transforming growth factor (TGF)-beta family. By blocking the TGF-beta signaling pathway, it promotes a rebalancing of the bone morphogenic protein pathway, which is thought to be a primary cause of PAH.

While existing treatments are designed to alleviate the symptoms of PAH, sotatercept is being developed to target a key underlying molecular cause of the disease.

Initial results from the randomized Phase 2 PULSAR clinical trial (NCT03496207), which is comparing the safety and efficacy of sotatercept to a placebo in people with PAH, showed that sotatercept was well tolerated, and eased heart strain and improved exercise capacity in individuals with PAH.

Another Phase 2 trial, called SPECTRA (NCT03738150), has been launched to further investigate the safety and efficacy of sotatercept in PAH. The trial is still recruiting people with PAH in the U.S. For more information on contacts and locations, go here.

“We’re delighted that in their initial assessments of our clinical trial data, regulatory authorities in the US and Europe have put forth a path that could potentially help us expedite delivery of sotatercept to patients in need of new therapeutic options,” Dable said.

A Conversation With Rare Disease Advocates