FDA Grants Orphan Drug Designation to ACE-1334 for Scleroderma

FDA Grants Orphan Drug Designation to ACE-1334 for Scleroderma
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The U. S. Food and Drug Administration (FDA) has granted orphan drug designation to ACE-1334, an investigational therapy that aims to ease scarring in the lungs of people with scleroderma.

Acceleron Pharma, the company developing ACE-1334, plans to launch a Phase 1b/2 clinical trial next year, testing the treatment candidate in patients with scleroderma-associated interstitial lung disease (SSc-ILD).

“We’re excited that ACE-1334 has received orphan drug designation, as it aligns with our vision to develop novel therapies for pulmonary diseases of high unmet medical need,” Habib Dable, president and CEO of Acceleron, said in a press release.

“We’re pleased to be planning for a Phase 1b/Phase 2 study to determine whether the anti-fibrotic activity ACE-1334 has demonstrated in preclinical models of fibrosis can be replicated in patients with SSc-ILD,” he said.

Medicines meant to treat disorders affecting fewer than 200,000 people in the U.S. are eligible for orphan drug status. This designation provides certain financial incentives, including seven years of market exclusivity if the treatment is approved, and waives FDA fees.

Although skin thickening caused by a buildup of collagen is scleroderma’s hallmark trait, the systemic form of the disorder affects multiple organs, including the lungs, and is characterized by the accumulation of scar tissue. Among those who develop ILD, most start experiencing symptoms within three years of diagnosis, limiting their respiratory function.

Current treatments used in people with SSc-ILD include immunosuppressants, stem cell transplants, anti-inflammatory medications, and pain relief therapies. Yet, none of these approaches directly targets the progressive scarring of scleroderma.

ACE-1334 specifically targets and blocks transforming growth factor (TGF)-beta 1 and 3 molecules, which scientists believe to be key in the lung scarring, or fibrosis, seen in ILD. The potential therapy has shown anti-fibrotic activity in preclinical models, according to Acceleron, and in a recently completed Phase 1 clinical trial in healthy volunteers.

The FDA has previously granted fast track designation to ACE-1334 for the treatment of SSc-ILD, which is intended to speed the development and review of new therapies that fill an unmet medical need in serious disorders. That status includes more frequent communication with the FDA, as well as eligibility for accelerated approval and priority review.

Other treatments for ILD include Ofev (nintedanib), which is indicated for slowing the decline of lung function. Esbriet (pirfenidone), an anti-fibrotic therapy approved for idiopathic pulmonary fibrosis, is currently being tested in scleroderma patients.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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José holds a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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  • ACE-1334, orphan drug designation

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