Investment Rating - The investment rating for Sarepta Therapeutics Inc. (SRPT) is "Buy" with a 12-month price target of $100, indicating a potential upside of 160.8% from the current price of $38.35 [8][9]. Core Insights - The commentary from the newly-appointed FDA CBER Director, Dr. Vinay Prasad, suggests that the full approval of Elevidys for ambulatory patients is likely to remain intact, barring any serious safety events [1][6]. - There is a risk to Elevidys' accelerated approval in non-ambulatory patients, which constitutes approximately half of the market, depending on confirmatory data expected in 2027 [1][6]. - The company has projected $13 billion in free cash flow by the end of 2030 and is focusing on pipeline diversification, including early Phase 1 data in other muscular dystrophies [1]. Financial Projections - Revenue projections for SRPT are estimated to grow from $1.9 billion in 2024 to $4.3 billion by 2027 [9]. - The EBITDA is expected to improve significantly, moving from a loss of $102.6 million in 2025 to a profit of $2.1 billion by 2027 [9]. - The forecasted Free Cash Flow yield is projected to increase from -2.8% in 2024 to 64.0% by 2027 [9]. Regulatory Environment - The new CBER Director emphasized the importance of regulatory flexibility in rare diseases and gene therapy, indicating a supportive stance towards maintaining previous approvals unless new safety concerns arise [2][6]. - The Director's approach suggests that accelerated approval pathways will continue to be utilized, particularly in the context of rare diseases and oncology [6]. Market Context - The market capitalization of SRPT is currently $3.7 billion, with an enterprise value of $3.3 billion [9]. - The company is facing competitive risks as other firms are also developing treatments for Duchenne muscular dystrophy (DMD), including RNA-based therapies and gene therapies [8].