FDA Decisions Loom for Innovative Therapies in Rare Diseases and Cancer

The pharmaceutical industry is poised for a series of significant regulatory decisions this month, with the U.S. Food and Drug Administration (FDA) set to rule on several groundbreaking therapies. These potential approvals span rare genetic disorders, various cancers, and could reshape treatment landscapes in their respective fields.

Arrowhead's RNAi Therapy Targets Rare Lipid Disorder

Arrowhead Pharmaceuticals is awaiting an FDA decision by November 18 for plozasiran, an RNA interference (RNAi) therapy designed to treat familial chylomicronemia syndrome (FCS). This rare genetic disorder, affecting only 1 to 2 patients per million, is characterized by the body's inability to break down fats, leading to dangerously high triglyceride levels and potentially fatal acute pancreatitis.

Plozasiran works by suppressing the production of apolipoprotein C-III (APOC3), a key molecule in triglyceride metabolism. In the Phase III PALISADE study, the therapy demonstrated impressive efficacy, with an 80% median reduction in triglyceride levels and an 83% lower risk of acute pancreatitis compared to placebo.

Potential Market Disruptions in Cancer and Growth Disorders

The FDA's docket this month includes decisions that could significantly impact existing treatment paradigms:

1. Bayer's sevabertinib for HER2-mutant non-small cell lung cancer (NSCLC) is under priority review, with a decision expected by November 28. The oral tyrosine kinase inhibitor showed promising results in the Phase I/II SOHO-01 study, with overall response rates ranging from 38% to 71% depending on prior treatment history.

2. Ascendis Pharma's TransCon CNP for achondroplasia, the most common cause of dwarfism, could challenge BioMarin's Voxzogo monopoly if approved by November 30. The therapy improved annual growth velocity by 5.42 cm per year in the Phase II ACcomplisH trial, potentially positioning it as a "major threat" to the existing treatment option.

3. AbbVie and Genmab are seeking to expand the indication of their bispecific antibody Epkinly to include relapsed or refractory follicular lymphoma, with a target action date of November 30. The Phase III EPCORE study showed a 95.7% objective response rate and a 79% reduction in the risk of disease progression or death when Epkinly was combined with rituximab and lenalidomide.

4. Kura Oncology and Kyowa Kirin await a verdict on ziftomenib, a menin blocker for relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutations, by November 30. The Phase II KOMET-001 study reported that 23% of patients achieved complete remission or complete remission with partial hematological recovery, with a median duration of 3.7 months.

These impending decisions underscore the industry's continued focus on developing targeted therapies for both rare diseases and more common cancers, with the potential to offer new hope to patients with limited treatment options.