FDA Restricts Elevidys Use; Nuvalent and Zymeworks Report Positive Cancer Drug Data

In a series of significant developments across the pharmaceutical industry, regulatory actions and clinical trial results are reshaping the landscape for gene therapies and targeted cancer treatments.

FDA Tightens Restrictions on Sarepta's Duchenne Gene Therapy

The Food and Drug Administration has issued new restrictions on Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy, Elevidys. Following the deaths of two patients due to liver failure post-treatment, the agency has added a "boxed" warning—its most severe—to Elevidys' labeling. The FDA has also narrowed the therapy's use to ambulatory patients aged four and above, rescinding its previous clearance for non-ambulatory individuals.

Sarepta must now implement broader patient monitoring protocols and conduct a post-marketing study to assess the risk of liver damage. In response, the company plans to test a new regimen of immunosuppressive drugs to mitigate the likelihood of organ damage.

Nuvalent and Zymeworks Advance in Targeted Cancer Therapies

Nuvalent has reported positive results from a Phase 1/2 trial of its ALK-positive lung cancer drug, neladalkib. The study showed tumor responses in 31% of pre-treated patients, with 64% maintaining responses for a year and 53% for 18 months. Jefferies analyst Roger Song described these findings as demonstrating "unprecedented durability and broad activity." Nuvalent aims to provide an alternative for patients who have developed resistance to existing treatments like Roche's Alecensa or Pfizer's Lorbrena.

Meanwhile, Zymeworks saw its shares surge by nearly 40% following its partner Jazz Pharmaceuticals' announcement of positive Phase 3 results for Ziihera in HER2-positive advanced or metastatic gastroesophageal adenocarcinoma. The drug, combined with chemotherapy, showed a "clinically meaningful and statistically significant" improvement in disease progression compared to standard treatments. Jazz plans to seek broader clearance for Ziihera next year.

Industry Restructuring and Strategic Shifts

Nxera Pharma has announced a restructuring plan that includes a 15% workforce reduction in Japan and the UK, along with a streamlined executive team. The company aims to focus on "next-generation" medicines for obesity, metabolic, and endocrine disorders, while divesting non-core assets. Nxera targets an operating margin of at least 30% by 2030 through these initiatives.

In a strategic pivot, cell therapy developer Century Therapeutics has halted its early-stage trial of CNTY-101 for autoimmune diseases, its only clinical-stage program. The company will now concentrate on preclinical programs for Type 1 diabetes and B-cell mediated autoimmune conditions. This decision leaves Century without significant near-term catalysts, according to Leerink Partners analyst Daina Graybosch, who notes that the company's "platform-first approach" has left it in a challenging position.