Larimar’s Friedreich’s ataxia program free at last from FDA hold

Larimar Therapeutics’ Friedreich’s ataxia program is free at last, with the FDA fully lifting a clinical hold that had remained in part on the program since 2021.

The rare disease biotech can now proceed with dosing 50 mg in a phase 2 open-label extension dose exploration study for nomlabofusp, previously known as CTI-1601, according to an after-market release Monday.

The drug is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia. The autosomal recessive genetic disease causes difficulty walking, a loss of sensation in the arms and legs, and impaired speech that worsens over time and usually starts in childhood.

Trouble for Larimar began in May 2021, when the FDA placed a full clinical hold on the program after viewing data from a nonhuman primate toxicology study that showed multiple deaths occurred at the highest dose levels.

At that point,…
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