Drugs

Larimar Reports Positive Data for Nomlabofusp

Larimar Therapeutics has reported encouraging long-term data from its open-label study of nomlabofusp, an investigational therapy for Friedreich’s ataxia (FA), while confirming plans to seek accelerated FDA approval in the second quarter of 2026. The study, which is evaluating daily subcutaneous doses of nomlabofusp in adolescents and adults with FA, showed sustained increases in frataxin (FXN) protein levels, which the company describes as the root cause deficiency in the disease. After six months of treatment, all 10 participants with available data achieved FXN levels above 50% of those measured in healthy volunteers, a range comparable to asymptomatic carriers who do not develop FA, it says.

Safety data remains a key focus, according to the company. To date, out of 65 patients who have received nomlabofusp during clinical studies, seven cases of anaphylaxis were reported in the open-label trial, most within the first six weeks of dosing. All patients recovered after treatment. Larimar says it has since adjusted its dosing protocol, introducing a lower test dose and stepwise escalation to improve tolerability, a strategy reviewed and accepted by FDA.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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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