Sotatercept receives orphan drug designation in Korea

Treatment for pulmonary arterial hypertension

Demonstrated efficacy through Phase III STELLAR trial

US FDA will review whether to approve the drug by March

Sotatercept, a new drug candidate for pulmonary arterial hypertension (PAH), has been designated an orphan drug in Korea.

 

The Ministry of Food and Drug Safety recently announced the drug’s designation in the first orphan drug designation announcement it made in the new year.

 

Sotatercept, which is being developed by Merck, is being regarded to have changed the treatment paradigm for PAH.

 

The substance, which is a combination of a protein complex, activin, and the transforming growth factor-β (TGF-β), reverses disease progression by blocking abnormal signaling between cells in the pulmonary blood vessels.

 

Pulmonary arterial hypertension is a condition characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation.

 

which leads to heart failure.

 

In Korea, about half of the patients with PAH die within 5 years.

 

More than 10 drugs are available for the condition in Korea, including phosphodiesterase type 5 inhibitors and endothelin receptor antagonists, but many patients suffer from severe symptoms despite being on a combination of two or three drugs.

 

The efficacy of the drug has been confirmed in the pivotal Phase III STELLAR trial.

 

Patients were randomized 1:1 to sotatercept or placebo to assess the efficacy and safety of the drug.

 

Results showed that the sotatercept group improved in 6-minute walk distance (6MWD, primary endpoint) by 40.1 m, compared with a decline of –1.4 m in the placebo group Also, 38.9% of patients in the sotatercept group achieved all of the multi-component improvement endpoints, the secondary endpoint of the study, which included an improvement of 30m or more in the six-minute walk test.

 

This was 4 times longer than the 10.1% in the placebo group.

 

The US FDA will decide whether to approve sotatercept by March 26, the target action date it had set under the Prescription Drug User Fee Act (PDUFA).