According to the US Food and Drug Association, Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.
The PULSAR study achieved the objectives of demonstrating improvement in Pulmonary Vascular Resistance and in 6minute walking distance at week 24 vs placebo
Sotatercept was generally well tolerated in subjects with PAH, with a safety profile consistent with that observed in other patient populations
Proof of concept has been demonstrated and a phase 3 program is planned.
Image by felixioncool