The FDA recently granted Orphan Drug status to CS1, a drug therapy from Cereno Scientific, according to Pulmonary Hypertension News. CS1 is designed to provide safe and effective treatment for patients with pulmonary arterial hypertension. The drug was granted Orphan Drug designation following a 2019 application.
Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension is a chronic, progressive type of high blood pressure that causes pulmonary arteries (arteries in the lungs) to thicken. This normally occurs after the pulmonary arteries narrow or become blocked. The heart now experiences more pressure in circulating deoxygenated blood. As a result, the heart also becomes weakened and hurt.
PAH is usually caused by a mutated BMPR2 gene. However, additional causes include drug use, HIV, heart disease or failure, liver disease, and autoimmune disorders. Symptoms include chest pain, fatigue, dizziness, shortness of breath, and swelling in the legs and ankles. However, many people are asymptomatic for the disease onset. Learn more about pulmonary arterial hypertension.
CS1
Orphan Drug Designation
Orphan Drug designation is given to drug therapies that treat rare diseases, particularly those in which patient populations have unmet needs. To receive this designation, therapies must treat conditions with less than 200,000 patients. Investopedia describes the designation as a status in which:
companies researching cures for rare diseases [receive] a seven-year window of tax reductions and the exclusive right to develop a cure for a specific condition.
Treating Pulmonary Arterial Hypertension
CS1 is a specially formulated form of valproic acid. Previous uses of valproic acid include treatment for epilepsy and bipolar disorder.
In their studies, Cereno Scientific discovered that CS1 could inhibit histone deacetylase. These enzymes can change DNA by impacting histone tails, which help transcribe genes. So, histone deacetylase makes it more difficult to change or modulate genes. By blocking the enzyme, CS1 becomes gene-altering.
For patients with pulmonary arterial hypertension, CS1 works by reducing inflammation, preventing blood clots, reducing or eliminating tissue damage, and relieving pressure throughout the body.
The next step in the process is testing whether CS1 can also assist with preventing blood clots in surgical patients. However, because of the spread of COVID-19, the study may not launch until the end of 2020 or the start of 2021.
