Acceleron gets EC orphan designation for sotatercept to treat PAH

Acceleron Pharma has secured orphan designation from the European Commission (EC) for its sotatercept to treat patients with pulmonary arterial hypertension (PAH).

PAH is a rare and chronic progressing disorder characterised by the narrowing of small pulmonary arteries and inflated blood pressure in the pulmonary circulation.

Sotatercept, an investigational reverse-remodelling agent, has been designed as a selective ligand trap for members of the TGF-beta superfamily to rebalance BMPR-II signalling, a crucial molecular driver of PAH.

At present, Acceleron is accelerating a phase 3 development plan for sotatercept, starting with the registrational trial called STELLAR. It is expected to be commenced by the end of this year.

The PULSAR phase 2 study evaluates sotatercep plus approved PAH-specific medicines in patients with PAH reached its primary endpoint of improvement in pulmonary vascular resistance and its key secondary endpoint of improvement in six-minute walk distance.

Based on PULSAR results, sotatercept secured breakthrough therapy designation from the US Food and Drug Administration (FDA) and priority medicines designation from the EMA in PAH. The company is also evaluating sotatercept in the SPECTRA phase 2 exploratory trial.

In addition, the firm is planning two additional phase 3 studies in patients with PAH, including HYPERION trial to explore early intervention with sotatercept and ZENITH trial to assess later-stage intervention.

Acceleron president and CEO Habib Dable said: “We’re thrilled at the European Commission’s decision to grant orphan designation to sotatercept in PAH.

“We fully intend to take advantage of the benefits that this 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.”

In April this year, Bristol Myers Squibb (BMS) and Acceleron Pharma secured FDA approval for Reblozyl (luspatercept-aamt) to treat anaemia in adults with lower-risk myelodysplastic syndromes (MDS).