Cereno Scientific’s preclinical candidate CS-1 (valproic acid) was recently granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA) for the US market for pulmonary arterial hypertension (PAH) —a rare, fatal cardiopulmonary disease with no curative treatment. Provided its successful clinical development, CS-1 has the potential to carve out a space on the market, due to its novel mechanism of action, says GlobalData, a leading data and analytics company.

Kajal Jaddoo, MSc, Associate Healthcare Analyst at GlobalData, comments: “This ODD will help Cereno Scientific transform its investments into a development program for CS-1 in PAH. The ODD could result in CS-1 achieving a more simplified, fast-tracked path to regulatory approval and market launch.

“If approved, CS-1 will likely experience competition from other brands already established on the market. However, key opinion leaders interviewed by GlobalData have emphasized a longstanding unmet need in the PAH market related to finding new targets and developing more efficient therapies.”

CS-1 has the potential to be commercially successful as its mechanism of action is novel. CS-1, an antihypertensive, acts by inhibiting histone deacetylase, thereby helping prevent the disease condition. The successful clinical development of CS-1 is significant because it is a new class of therapy to be developed, despite the ongoing high need for PAH therapies. The current PAH drug treatment space consists of five therapeutic classes: endothelin receptor antagonists (ERAs), phosphodiesterase type 5 (PDE5) inhibitors, soluble guanylate cyclase (sGC) stimulators, prostacyclin derivatives, and prostacyclin (IP) receptor agonists.

Jaddoo concludes: “Cereno Scientific continues to grow its drug pipeline, mainly focusing on epigenetic modulation of cardiovascular and rare diseases. The PAH market is known for its high level of clinical need, granting opportunity for CS-1, which offers an additional treatment for the disease, if approved.”