Rare Daily Staff

The European Medicines Agency granted Priority Medicines (PRIME) designation to Larimar Therapeutics’ CTI-1601 for the treatment of Friedreich’s ataxia. 

The PRIME program is designed to enhance support for the development of medicines that target an unmet medical need. Through PRIME, the EMA offers early and proactive support to medicine developers to optimize the generation of robust data on a medicine’s benefits and risks and enable accelerated assessment of medicines applications so that these medicines can reach patients earlier. To receive a PRIME designation, a product candidate must show its potential to benefit patients with unmet medical needs based on early clinical data or on compelling nonclinical data and tolerability data from initial clinical trials.

Friedreich’s ataxia (FA) a is progressive, neurodegenerative condition caused by mutations in the FXN gene. Signs and symptoms usually begin in puberty and lead to progressive impaired muscle coordination, gradual loss of muscle strength and sensation in the arms and legs, muscle stiffness, and impaired speech. Larimar’s CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with FA who are unable to produce enough of this essential protein.

The PRIME designation was based on positive data from Larimar’s nonclinical studies and phase 1 clinical program in patients with FA. Pharmacodynamic data from this program showed that daily subcutaneous injections of CTI-1601 at doses of 50 mg or 100 mg resulted in frataxin levels in peripheral tissues (buccal cells) that were at or in excess of those that would be expected in phenotypically normal heterozygous carriers. Safety data from the program indicated that repeated subcutaneous injections of CTI-1601 were generally well tolerated at doses up to 100 mg administered daily for 13 days.

“Notably, we believe CTI-1601 is the only drug candidate to receive such a designation for the treatment of FA as well as the only clinical-stage candidate that we are aware of that is designed to address the root cause of the disease,” said Carole Ben-Maimon, president and CEO of Larimar. She said the company plans to initiate an open label extension and pediatric multiple-ascending dose trials in the second half of the year.”   

In addition to PRIME designation, CTI-1601 has also been granted Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the U.S. Food and Drug Administration and Orphan Drug designation by the European Commission.

Photo: Carole Ben-Maimon, president and CEO of Larimar

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