An Experimental Treatment for Pulmonary Arterial Hypertension Just Earned Orphan Drug Designation

According to a story from, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the company’s experimental product candidate sotatercept has earned Orphan Drug designation from the US Food and Drug Administration (FDA). This designation is for the treatment of pulmonary arterial hypertension, a rare lung condition. Acceleron focuses on the development of therapeutics based on the TGF-beta superfamily for the treatment of diseases considered rare or serious.

About Pulmonary Arterial Hypertension (PAH)

Pulmonary arterial hypertension is a condition in which the blood pressure in the arteries of the lungs is abnormally high. The cause of pulmonary arterial hypertension is often unknown in many cases. However, there are a variety of potential causes, such as certain heritable genetic mutations, exposure to certain toxins, and drug use (ex. methamphetamine). It can also appear as a symptom or complication in a number of other diseases, such as heart disease, connective tissue disease, and infection with HIV. The arteries in the lungs are often inflamed. Symptoms of this condition include rapid heartbeat, poor exercise tolerance, shortness of breath, fainting, leg swelling, fatigue, and chest pain. Treatment may include a number of medications and surgical operations, including lung transplant. A transplant can cure the condition, but it can cause many complications. Survival rate is often only about two or three years without treatment, but the latest drugs can prolong life by several years or more. Click here to learn more about pulmonary arterial hypertension.

About Orphan Drug Designation

Orphan Drug designation is reserved for treatments that are intended for diseases and conditions that are considered rare. This is defined as any disease that affects less than 200,000 people in the US. To earn this designation, an experimental drug must either fulfill a currently unmet medical need or display potential advantages in efficacy or safety in comparison to current therapies. This designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a period of market exclusivity lasting seven years if the FDA approves the drug.

Sotatercept is currently being tested as a treatment for the condition in a couple of phase 2 clinical trials. Acceleron hopes to have topline results from these studies by early next year.

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