Safety concerns have been raised after Sarepta Therapeutics Inc.'s (SRPT:NASDAQ) trial drug, Elevidys, has been facing "Sell" and "Neutral" ratings after several deaths in its 2025 clinical trials. The U.S. Food and Drug Administration (FDA) revoked Sarepta's platform technology designation when three deaths among its patient trial base. These deaths appear to be the results of acute liver failure treated with Elevidys. “Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury,” said Director of the FDA’s Center for Biologics Evaluation and Research Vinay Prasad, MD, M.P.H.
The FDA is continuing to investigate the risk of acute liver failure with serious outcomes, including those leading to hospitalization and death, by following gene therapies using Sarepta's AAVrh74 Platform Technology to assess the need for further regulatory actions.
In more positive news, on July 28, 2026, the company granted previously approved equity awards as part of its Employment Commencement Incentive Plan. The awards were a material inducement to the employment of Michael Severino, MD, as CEO of Sarepta.
According to the release: "Dr. Severino received options to purchase 2,224,342 shares of Sarepta's common stock and 756,104 restricted stock units. The options have an exercise price of US$17.74 per share, which is equal to a 15% premium to the closing price of Sarepta's common stock on July 28, 2026."
One quarter of the options will vest on the one-year anniversary of the grant date, and thereafter, 1/48th of Severino's options will vest monthly, making them fully vested on the fourth anniversary of the grant date — subject to Severino's continued employment. Likewise, one quarter of the RSUs will vest yearly on each anniversary of the grant date, making them fully vested by the fourth anniversary of the grant date, also subject to continued employment."
Sarepta Therapeutics Inc. is a worldwide biotech company that focuses on genetic medicine for rare diseases and is headquartered in Massachusetts, USA. The company currently has five FDA-approved drugs for treating Duchenne Muscular Dystrophy and has several other drugs in its pipeline.
DMD Market Set to Quadruple
The Duchenne Muscular Dystrophy (DMD) market was worth US$16.38 billion in 2025 and is expected to grow to US$62.13 billion by 2030, with a massive expected CAGR of 30.5%. North America is currently the largest market in need of muscular dystrophy treatments.
In February 2026, Iqvia discussed the global pharma market projection for 2026, noting that total drug usage is expected to surpass four trillion doses daily by 2030. They wrote, "The largest drivers of medicine spending growth through the next five years will continue to be the use in developed markets of innovative therapeutics, especially in oncology, immunology, diabetes, and obesity."
Pharma sector funding fell between 2024 and 2025, according to a March 26, 2026, article for Fierce Biotech by Nick Paul Taylor. He wrote that pharma funding had fallen from 2024 but noted that, "2025 was still the third-best year of the past decade. Similarly, overall funding was well above the pre-pandemic norm and only topped by 2020, 2021, and 2024."
BCG talked about trends biopharma companies need to be aware of in 2026 in order to stay competitive, saying, "Near term, companies need to continue to innovate to decrease the complexity and cost of these therapies, and governments can find ways to incentivize and pay for them. The longer-term challenge for companies is to factor operational and economic considerations into R&D decision making earlier, ensuring that trial designs match real-world usage, indication sequences match opportunity, and endpoints enable market access."
Wedbush, Wainwright Split on Rating
Andy Chen of Wolfe Research updated the company's rating from "Peer Outperform" to "Outperform", with a price target of US$27.00 on July 9, 2026.
Wedbush's Yun Zhong, Ph.D., rated Sarepta an "Outperform" on July 20, 2026, with a 12-month price target of US$35.00, after the company announced that the FDA had accepted for filing the sNDAs to convert Amondys 45 and Vyondys 53's accelerated approvals to traditional approvals, with an estimated PDUFA date of February 28, 2027. Zhong wrote: " We see the FDA's decision as an important step forward since it confirms that SRPT has submitted adequate data, including both results from the randomized, double-blind, placebo-controlled Ph3 ESSENCE confirmatory study and real-world evidence, to support a review. We expect the FDA to recognize Amondys 45 and Vyondys 53's favorable clinical profiles, and we remain optimistic in both therapies receiving traditional approvals by the PDUFA data to continue benefiting amenable DMD patients."
Also on July 20, 2026, Piper Sandler's Biren Amin lowered the price target for Sarepta from US$23.00 to US$20.00, maintaining a
Neutral" rating.
In contrast, Mitchell Kapoor of H.C. Wainwright & Co. reiterated a "Sell" rating for Sarepta on July 29, 2026, despite Severino's appointment as CEO, stating that "no shuffles in leadership can fix core issues" and that the new CEO's "impressive credentials" do not address "demand, safety, and data issues" within the company. "In all, this leadership change does not alter anything fundamental in our view: the stock still requires evidence that ELEVIDYS can recover from the safety-related disruption, PMOs can stabilize, and the late-2026 pipeline can generate credible value. Looking ahead, we see next week's 2Q26 update as a meaningful catalyst — we will be especially focused on whether ELEVIDYS declines again from the 1Q26 US$102M revenue print and if PMOs hold near the ~US$230M quarterly run-rate," wrote Kapoor. Along with reiterating the "Sell" rating, Kapoor reiterated a 12-month price target of US$5.00.
SRP-1005 Dosing Begins This Year
According to Sarepta's investor presentation, the next 18 months will hopefully show efficacy in MAD (Multiple Ascending Dose) studies, and results are expected in the second half of 2026 for both SRP-1003, while SRP-1003 should get Casi22 data at the same time. Another drug, SRP-1005, intended to treat Huntington's disease, commenced dosing in the first half of this year, with hopes of gathering proof-of-biology data in the first half of 2027.
Ownership & Share Information1
Sarepta Therapeutics Inc. has a market cap of US$1.57 billion, with 105.58 million shares outstanding. The company's 52-week range is US$14.68-US$25.32. Institutions own 54% of shares, while Management & Insiders own 5%. The remaining 41% of shares are Retail.
Streetwise Ownership Overview*
Sarepta Therapeutics Inc. (SRPT:NASDAQ)
Frequently Asked Questions
Q: What are stock options?
A: Stock options give an employee the right to buy company shares at a set price in the future. If the company's stock price rises above that price, the options may become more valuable, making them a common way to reward executives and align their interests with shareholders.
Q: What are restricted stock units (RSUs)?
A: Restricted stock units (RSUs) are a form of employee compensation that gives workers company shares after certain conditions are met, such as remaining with the company for a set period. Unlike stock options, employees do not have to purchase the shares once they vest.
Q: What does "vesting" mean?
A: Vesting is the process by which an employee earns ownership of stock-based compensation over time. Many companies use multi-year vesting schedules to encourage executives and employees to stay with the company for the long term.
Q: What is Duchenne muscular dystrophy (DMD)?
A: Duchenne muscular dystrophy (DMD) is a rare inherited disease that causes progressive muscle weakness and loss of muscle function. It primarily affects boys and is caused by mutations in the gene responsible for producing dystrophin, a protein that helps keep muscle cells healthy.
Q: What is accelerated FDA approval?
A: Accelerated approval is an FDA pathway that allows certain medicines for serious diseases to reach patients sooner based on early evidence that they are likely to provide a clinical benefit. Companies must still conduct additional studies to confirm the treatment's effectiveness before receiving traditional approval.
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- Cori Fisher wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an employee.
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1. Ownership and Share Structure Information
The information listed above was updated on the date this article was published and was compiled from information from the company and various other data providers.



















































