Risk Factors Update Summary
- ELEVIDYS has been approved for marketing only in the U.S., potentially limiting revenue growth.
- The commercial success of gene therapy products depends on public perception and regulatory approvals.
- Accelerated approval for ELEVIDYS was based on a surrogate endpoint, impacting long-term efficacy assessment.
- The Biden Administration may implement cost containment measures affecting drug pricing and reimbursement.
- Delays in obtaining FDA or EMA approvals could hinder commercialization efforts and revenue generation.
- The recently enacted FDORA expands FDA authority, potentially affecting marketing approval conditions.
- Failure to maintain revenues from eteplirsen, golodirsen, and casimersen could impact financial results.
Full Text Changes in Most Recent 10-K
Intended use: review the highlighted statements. These are additions to the risk factors disclosure in the most recent 10-K filing compared to the previous 10-K filing. Deleted and moved text is less important and is shown for context.
To view the full company filings, click on the following link to be taken to the SEC EDGAR database landing page for the company: https://www.sec.gov/edgar/browse/?CIK=873303&owner=exclude
This content requires a 'Free' membership to view. Please create one here.
This content requires a 'Free' membership to view. Please create one here.