Larimar Therapeutics Completes Rolling BLA Submission Seeking Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia
Key Highlights
- ➤Larimar (LRMR) completed rolling BLA submission seeking accelerated approval for nomlabofusp.
- ➤FDA indicated existing data could support submission and review; approval remains subject to review.
- ➤FDA is willing to consider skin FXN as a potential novel surrogate endpoint.
- ➤FORWARD-FA Phase 3 study is enrolling ambulatory participants ages 2 to 40.
- ➤BLA acceptance is expected early 2027; U.S. approval targeted mid-2027, if approved.
Expert Statements
Carole Ben-Maimon, Chief Executive Officer of Larimar Therapeutics
“Completing our BLA submission is a major milestone for Larimar and the FA community, bringing years of development work together in an application seeking accelerated approval of nomlabofusp.”
Carole Ben-Maimon, Chief Executive Officer of Larimar Therapeutics
“Our confidence in the program is grounded in the sustained increases in skin frataxin and consistent directional improvements observed in multiple clinical outcomes in patients.”
Carole Ben-Maimon, Chief Executive Officer of Larimar Therapeutics
“It is important to note that patients with FA are expected to experience continued neurologic decline, not improvement.”
Carole Ben-Maimon, Chief Executive Officer of Larimar Therapeutics
“After treatment with nomlabofusp, we have not observed worsening in most participants but rather signs of improvement.”
Carole Ben-Maimon, Chief Executive Officer of Larimar Therapeutics
“Our previously disclosed discussions with FDA have helped inform the content of the submission.”
Carole Ben-Maimon, Chief Executive Officer of Larimar Therapeutics
“As we support the potential FDA review and advance FORWARD-FA, our confirmatory Phase 3 study, our team is preparing for a potential U.S. launch with the goal of making nomlabofusp available to patients as quickly as possible following approval, if granted.”
Carole Ben-Maimon, Chief Executive Officer of Larimar Therapeutics
“In parallel, we continue to evaluate options to optimize our global commercialization strategy.”
Jen Farmer, M.S., Chief Executive Officer of Friedreich’s Ataxia Research Alliance (FARA)
“Larimar’s completion of its BLA submission for nomlabofusp marks an important and exciting milestone for the Friedreich’s ataxia community.”
Jen Farmer, M.S., Chief Executive Officer of Friedreich’s Ataxia Research Alliance (FARA)
“As the first potential frataxin replacement therapy, nomlabofusp is designed to address the underlying cause of FA.”
Jen Farmer, M.S., Chief Executive Officer of Friedreich’s Ataxia Research Alliance (FARA)
“The fact that the submission includes data from adolescents ages 12 to 17 is especially meaningful, as there are currently no approved therapies for children under 16 years of age.”
Jen Farmer, M.S., Chief Executive Officer of Friedreich’s Ataxia Research Alliance (FARA)
“FA is a complex and variable disease, and patients and families need multiple treatment options that can address their individual needs across different ages and stages of disease.”
* Completed BLA submission marks a major milestone in advancing nomlabofusp as the first potential disease-modifying therapy to address the underlying cause of FA
* Submission follows FDA alignment on key elements of the BLA package for submission, including willingness to consider skin frataxin as a potential novel surrogate endpoint
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