FDA grants orphan drug designation to calcium channel blocker for systemic sclerosis
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Key takeaways:
- An ongoing study showed improvements in SSc severity, disease-related pain, Raynaud’s symptoms and gastrointestinal dysfunction.
- The manufacturer says the drug would be a once-daily, cost-efficient treatment.
The FDA has granted orphan drug designation to AISA-021, a fourth-generation calcium channel blocker, for the treatment of systemic sclerosis, according to a press release from Aisa Pharma.
The drug is a form of cilnidipine, a dihydropyridine calcium channel blocker approved and used for the treatment of hypertension in some Asian countries, read the release. It is marked by heightened selectivity for the N-type calcium channel.
“To our knowledge, this is the first time the FDA has granted orphan drug designation to a calcium channel antagonist for an autoimmune illness,” Andrew Sternlicht, MD, CEO and founder of Aisa Pharma, said in the release. “We hope this designation will accelerate our development program for AISA-021, which is designed to provide a once-daily, well-tolerated and economical treatment that we hope can improve the lives of patients with SSc. We are actively seeking a development partner and investors to support bringing this much-needed treatment to patients.”
The FDA’s orphan drug designation provides incentives, including 7 years of market exclusivity, to develop drugs for rare diseases or conditions.
Aisa’s application for orphan drug status included data from an ongoing phase 2 study, which demonstrated improvements in overall SSc severity, disease-related pain, Raynaud’s symptoms, gastrointestinal dysfunction and other endpoints, according to the release.
A pre-investigational new drug meeting with the FDA is scheduled for later this month concerning AISA-021 for SSc and secondary Raynaud’s phenomenon, the company added.
At this year’s ACR Convergence Scientific Meeting in Washington, D.C., researchers will present two abstracts on AISA-021’s efficacy in SSc and secondary Raynaud’s phenomenon.
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