FDA grants orphan drug status to calcium channel blocker for systemic sclerosis

Key findings:

• An ongoing study showed improvements in SSc severity, disease-related pain, Raynaud's symptoms, and gastrointestinal dysfunction.
• The manufacturer states that the drug is a cost-effective treatment that can be taken once daily.

According to a press release, the FDA has granted orphan drug designation to AISA-021, a fourth-generation calcium channel blocker, for the treatment of systemic sclerosis. from Aisa Pharma.

The drug is a form of cilnidipine, a dihydropyridine calcium channel blocker that is approved and used in some Asian countries to treat high blood pressure, the press release said. It is characterized by increased selectivity for the N-type calcium channel.

“To our knowledge, this is the first time the FDA has granted orphan drug status to a calcium channel antagonist for an autoimmune disease,” Andrew Sternlicht, MD, CEO and founder of Aisa Pharma, said in the press release: “We hope this approval will accelerate our development program for AISA-021, which is designed to provide a once-daily, well-tolerated and cost-effective treatment that we hope can improve the lives of patients with SSc. We are actively seeking a development partner and investors to help us bring this much-needed treatment to patients.”

The FDA's orphan drug designation provides incentives for the development of drugs for rare diseases or conditions, including seven years of market exclusivity.

Aisa's application for orphan drug designation includes data from an ongoing Phase 2 study that demonstrated improvements in overall SSc severity, disease-related pain, Raynaud's syndrome symptoms, gastrointestinal dysfunction and other endpoints, the press release said.

A meeting with the FDA is scheduled for later this month to pre-screen a new drug involving AISA-021 for SSc and secondary Raynaud's syndrome, the company added.

At this year's ACR Convergence Scientific Meeting in Washington, DC, researchers will present two abstracts on the efficacy of AISA-021 in SSc and secondary Raynaud's syndrome.

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