Drugs

Hold Removed on Friedreich’s Ataxia Therapy

FDA has removed a partial clinical hold on Larimar Therapeutics’ nomlabofusp (CTI-1601), which is being evaluated for treating patients with Friedreich’s Ataxia (FA). Nomlabofusp is described as a novel protein replacement therapy designed to address the root cause of FA by delivering frataxin to mitochondria. The company says the agency removed the partial hold after reviewing data from its recently completed four-week, placebo-controlled Phase 2 dose exploration study. The review included data from both the 25 mg and 50 mg dosage cohorts in patients who received nomlabofusp daily for 14 days followed by every other day dosing until day 28.

“In the Phase 2 dose exploration study, nomlabofusp was generally well-tolerated throughout the four-week treatment period," Larimar says. “Nomlabofusp had a predictable pharmacokinetic profile and demonstrated dose-dependent increases in frataxin levels in skin and buccal cells."

The long-term safety and tolerability, pharmacokinetics, and frataxin levels in peripheral tissues following nomlabofusp are currently being evaluated in an ongoing open label extension (OLE) study in patients with FA. “The OLE study will initially evaluate daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver,” it says. “Larimar plans to dose escalate to 50 mg in the OLE study following additional characterization of frataxin PD at the 25 mg dose. Further dose escalation above 50 mg, if necessary, would require submission of additional data for FDA review to support the increased dose.”

The company previously said the 2021 hold followed its notifying FDA of mortalities occurring at the highest dose levels in an 180-day non-human primate toxicology study intended to support extended dosing of patients with CTI-1601 (see earlier story).

The company now says it is on track to submit a BLA on the therapy in the second half of 2025.

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FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

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FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome