COMP Recommends CTI-1601 as Orphan Drug for Treating Friedreich’s Ataxia

COMP Recommends CTI-1601 as Orphan Drug for Treating Friedreich’s Ataxia
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The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), has issued a positive opinion about granting orphan drug status to CTI-1601, Larimar Therapeutics’ investigational treatment for Friedreich’s ataxia (FA).

The company now is expecting the European Commission, which makes the final decision based on the COMP’s recommendation, to formally grant CTI-1601 the designation of orphan drug later this year.

Orphan designation is given to medicines in Europe with the potential to be safe and effective treatments for rare, life-threatening or chronically debilitating conditions affecting no more than one in every in 2,000 people. If granted, it will provide Larimar with a range of incentives, including assistance with trial protocols and a 10-year period of market exclusivity if the treatment is approved.

“The positive opinion for orphan drug designation from the EMA COMP is an important milestone to bring a much-needed potential therapy to patients with FA, a devastating disease that currently has no approved medical treatments,” Carole Ben-Maimon, MD, president and CEO of Larimar Therapeutics, said in a press release.

CTI-1601 (formerly known as TAT Frataxin) is a lab-made, modified version of frataxin, which is the protein lacking in FA. The modified protein is designed to enter mitochondria (the cells’ powerhouses), where it is expected to be processed and integrated into mitochondrial metabolism.

By restoring frataxin levels where needed, CTI-1601 may improve mitochondrial function, ease symptoms, and halt disease progression. Preclinical data in a mouse model of FA showed that CTI-1601 extended mean lifespan by 53% and improved cardiac function.

A Phase 1 trial (NCT04176991) is currently investigating the safety and tolerability of increasing doses of CTI-1601 in adults with FA. A total of 32 participants will be recruited, and assigned randomly to either a single subcutaneous (under the skin) injection of CTI-1601 or a placebo.

Ongoing in New Jersey, the trial is recruiting patients for its third dosing group, after a temporary pause due to the COVID-19 pandemic. Treatment in the first two groups is completed and data is expected next year.

“We look forward to working closely with EMA and continuing our U.S. Phase 1 trial of CTI-1601, which has the potential to become the first frataxin replacement therapy for patients with FA. We remain on track to report topline data in the first half of 2021,” said Ben-Maimon.

The U.S. Food and Drug Administration has designated CTI-1601 an orphan drug and rare pediatric disease therapy to support its development, and placed it on fast track, which could allow for priority review should it come up for approval.

The potential frataxin replacement therapy was developed originally by Chondrial Therapeutics, which entered a merger agreement with Zafgen in late 2019, resulting in a new combined company, Larimar.

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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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência.
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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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