Pharmaceutical Executive Daily: FDA Approves Subcutaneous Sarclisa Escena

Welcome to Pharmaceutical Executive Daily, your quick briefing on the top news shaping the pharmaceutical and life sciences industry.

In today's Pharmaceutical Executive Daily, FDA quietly pauses its real-time public release of complete response letters, FDA approves Sanofi's Sarclisa Escena as the first anticancer treatment in the U.S. to be administered via an on-body injector, and AstraZeneca shares fall nearly 10 percent after its Phase III Cardio-TTRansform trial of Wainua in transthyretin-mediated amyloid cardiomyopathy fails to meet its primary endpoint.

FDA has temporarily paused its practice of publishing complete response letters in real time following an April citizen petition filed by law firm Covington and Burling on behalf of an unnamed pharmaceutical company challenging the legal basis of the practice. The petition argues that FDA's current approach violates federal statutes protecting confidential commercial information, contravenes the agency's own longstanding regulations, and fails to give drug sponsors the opportunity to review and propose redactions before their rejection letters are made public. The most recent CRL in FDA's database is the April 22 rejection of AbbVie's investigational botulinum toxin candidate, with more recent rejections including a late June denial for a Sobi gout prospect going unpublished, while FDA simultaneously moves to codify a formal rule that would expand the commissioner's authority to release CRLs, with a proposed rulemaking expected in October 2026.

FDA has approved subcutaneous Sarclisa Escena for the treatment of multiple myeloma patients across all existing indications for which the intravenous Sarclisa formulation is approved, making it the first anticancer treatment in the United States cleared for administration via an on-body injector and the first multiple myeloma treatment available in both subcutaneous on-body injector and manual subcutaneous administration formats. The approval is supported by Phase III Iraklia trial data demonstrating that Sarclisa Escena administered via the CirCLIQ on-body injector achieved non-inferior efficacy and comparable pharmacokinetics and safety to the intravenous formulation, while delivering meaningfully shorter administration times and fewer administration-related reactions.

Finally, AstraZeneca shares fell nearly 10 percent Thursday, their worst single-day decline in nine years, erasing more than $30 billion in market value, after the Phase III Cardio-TTRansform trial of Wainua, its eplontersen gene-silencing therapy co-developed with Ionis Pharmaceuticals, failed to meet its primary endpoint of reducing cardiovascular deaths and recurrent cardiovascular events over 140 weeks in patients with transthyretin-mediated amyloid cardiomyopathy. Analysts rapidly identified trial design as the central issue: 57 percent of the 1,432 enrolled patients were already receiving a stabilizer drug at baseline, and an additional 24 percent initiated stabilizer therapy during the trial, making it effectively impossible to isolate the incremental benefit of Wainua layered on top of an active comparator class it was never designed to complement, and leaving analysts at Jefferies to warn that the failure would cost management a degree of credibility given the confidence they had expressed ahead of the readout.

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