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

Sarepta's Duchenne Treatment Faces New Limitations

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

Sarepta must now conduct broader patient monitoring and commit to a post-marketing study assessing the risk of liver damage. The company plans to test a new regimen of immunosuppressive drugs to mitigate the likelihood of organ damage.

Promising Results for Targeted Cancer Therapies

Nuvalent has reported positive study results for its ALK-positive lung cancer drug, neladalkib. In a Phase 1/2 trial, the treatment led to tumor responses in 31% of pre-treated patients, with responses lasting for a year in 64% of those patients and 18 months in 53%. Jefferies analyst Roger Song described these findings as demonstrating "unprecedented durability and broad activity."

Nuvalent aims to provide an alternative for patients whose tumors have developed resistance to existing treatments like Roche's Alecensa or Pfizer's Lorbrena. The company has already filed for approval of another medicine targeting lung tumors with ROS1 alterations.

Meanwhile, Zymeworks' partner, Jazz Pharmaceuticals, announced 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 treatment. Jazz plans to seek broader clearance for Ziihera, which is already approved for a type of biliary tract tumor, next year.

Industry Restructuring and Strategic Shifts

Nxera Pharma has announced a restructuring plan, including layoffs of about 15% of its workforce in Japan and the UK. The company will reduce its executive team from ten to seven members as it refocuses on drugs with the "greatest value creation potential." Nxera will prioritize "next-generation" medicines for obesity, metabolic, and endocrine disorders while seeking partnerships or divestment for non-core therapies. The company aims to achieve an operating margin of at least 30% by 2030 through these initiatives.

In a strategic pivot, cell therapy developer Century Therapeutics has deprioritized its sole clinical-stage program, CNTY-101, in favor of advancing earlier, "innovative" therapies. The company has halted an early-stage trial testing a donor-derived therapy for autoimmune diseases, although an investigator-sponsored study will continue. Century will now focus on preclinical programs for Type 1 diabetes and B-cell mediated autoimmune conditions. This decision leaves the company without "value-inflecting catalysts" over the next 12 to 15 months, according to Leerink Partners analyst Daina Graybosch.