Core Insights - The approval of Itvisma by the FDA marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), targeting patients aged two and older with specific genetic mutations [1][2] - Itvisma's wholesale price is set at $2.59 million, making it one of the most expensive drugs on the market, and it is the first gene therapy approved for a broader patient demographic [1][3] - The global gene therapy market is expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements and regulatory clarity [2][8] Drug Pricing and Market Strategy - Novartis has justified the high pricing of Zolgensma, its previous gene therapy, citing a total R&D investment of $9.4 billion, including an $8.7 billion acquisition of AveXis [3][4] - The pricing strategy for Zolgensma has been significantly higher than industry estimates, indicating a potential trend for future gene therapies [3][4] - Itvisma's pricing reflects a calculated commercial strategy, aiming to establish a precedent for high-cost life-saving treatments [3][4] Competitive Landscape - Itvisma competes directly with Spinraza, the first targeted SMA treatment, which has undergone significant price reductions in China after being included in the national insurance directory [6][7] - The gene therapy market is characterized by high R&D costs and significant barriers to entry, allowing early entrants to maintain pricing power and market exclusivity [2][7] - The market is expected to evolve towards platform-based and diversified pipelines as more companies enter the gene editing and delivery systems space [2][8] Market Growth and Investment - The global gene therapy market is projected to reach $9.03 billion in 2024 and grow to $64.64 billion by 2033, with a compound annual growth rate of 27.6% [8] - Major pharmaceutical companies are actively investing in gene therapy, with significant acquisitions and partnerships to enhance their portfolios [9][10] - The increasing number of approved gene therapy products and the rapid pace of regulatory approvals are expected to drive market expansion [8][10] Challenges and Future Directions - The development of gene therapies faces challenges such as high costs, short shelf life, and production capacity limitations [11][12] - The industry is exploring second and third-generation gene therapies to overcome the limitations of current technologies, such as AAV delivery systems [11][12] - Balancing innovation incentives with drug accessibility remains a long-term issue for the industry [11][12]

Core Viewpoint - The approval of Itvisma by Novartis marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), with a price tag of $2.59 million, indicating the market's tolerance for high-cost life-saving drugs [1][2]. Group 1: Gene Therapy Market Dynamics - The global gene therapy market is at a turning point, expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements, clearer regulatory pathways, and innovative payment models [2]. - The success of Zolgensma has validated the commercial viability of one-time curative treatments for single-gene hereditary diseases, setting a benchmark for future gene therapies in various fields [2]. - Currently, only three SMA drugs have been approved globally, with Itvisma being the only gene therapy product, highlighting its rarity and the high barriers to entry in the market [2]. Group 2: Pricing and Cost Justification - Novartis disclosed that the total R&D investment for Zolgensma reached $9.4 billion, including an $8.7 billion acquisition of AveXis [4]. - The pricing strategy for Zolgensma was significantly higher than industry estimates, with a suggested price range of $1.5 million to $5 million per patient, compared to UBS's estimate of $2 million and ICER's range of $310,000 to $1.5 million [4]. - The high price of Itvisma reflects a calculated commercial strategy, as the company aims to establish a strong market presence despite the high upfront costs associated with gene therapy development [2][4]. Group 3: Clinical Efficacy and Safety - Itvisma's approval was based on the results of the pivotal Phase 3 study STEER, which showed statistically significant improvements in motor function among treated patients, with effects sustained over a 52-week follow-up [5][6]. - The most common adverse events reported in the studies included upper respiratory infections and fever, indicating a consistent safety profile [5][6]. Group 4: Competitive Landscape - Itvisma competes directly with Spinraza, the first targeted SMA treatment, which has undergone significant price reductions in markets like China after being included in insurance coverage [7]. - The gene therapy market is expected to see explosive growth, with projections indicating a market size of $9.03 billion in 2024, increasing to $64.64 billion by 2033, driven by innovations in gene editing technologies and accelerated regulatory approvals [9]. Group 5: Industry Challenges and Future Outlook - The gene therapy sector faces challenges such as high production costs, short shelf life of viral vectors, and the need for stringent quality control, which can limit supply and increase prices [13]. - Balancing innovation incentives with drug accessibility remains a long-term issue for the industry, as the future of gene therapy will depend on its ability to reach a broader patient population beyond clinical settings [13].

Core Insights - The approval of Itvisma by the FDA marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), targeting patients aged two and older with specific genetic mutations [1][2] - Itvisma's wholesale price is set at $2.59 million, making it one of the most expensive drugs on the market, and it is the first gene therapy approved for a broader patient demographic [1][3] - The global gene therapy market is expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements and regulatory clarity [2][8] Pricing and Market Dynamics - The pricing strategy for Itvisma is influenced by the high R&D costs associated with gene therapies, with Zolgensma's development costing $9.4 billion [3][4] - The market for SMA treatments is currently limited, with only three approved drugs, highlighting Itvisma's unique position as a gene therapy product [2][4] - The competitive landscape is evolving, with companies focusing on gene editing and delivery systems, which may lead to increased competition and innovation [2][10] Clinical Efficacy and Safety - Itvisma's approval is based on significant improvements in motor function observed in clinical trials, with consistent safety profiles reported [4][5] - The treatment aims to provide a one-time solution by delivering a functional SMN1 gene, potentially reducing the need for ongoing therapies [5][6] Market Potential and Competition - The gene therapy market is projected to reach $90.3 billion by 2024, with a compound annual growth rate of 27.6% expected through 2033 [8] - Major pharmaceutical companies are actively investing in gene therapy, as evidenced by significant acquisitions and partnerships in the sector [9][10] - Itvisma will compete directly with Spinraza, the first SMA treatment, which has undergone significant price adjustments to improve accessibility [6][7] Future Outlook - The success of Itvisma could pave the way for further advancements in gene therapy, particularly for rare diseases, while also raising questions about drug pricing and accessibility [12][13] - Ongoing challenges include high production costs and the need for innovative delivery systems to enhance the efficacy and safety of gene therapies [11][12]

November 3, 2025 Q3 2025 Earnings Conference Call Patients can't wait for the next breakthrough in medical research. So neither will we. Doug Ingram CEO Louise Rodino-Klapac, PhD President, R&D and Technical Operations Patrick E. Moss, PharmD Executive Vice President, Chief Commercial Officer Ryan H. Wong Executive Vice President, Chief Financial Officer DILLON Living with Duchenne muscular dystrophy ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 1 Forward-looking statements In order to provide Sare ...

Core Viewpoint - Kuehn Law, PLLC is investigating potential breaches of fiduciary duties by certain officers and directors of Sarepta Therapeutics, Inc. related to the misrepresentation of safety risks associated with the ELEVIDYS treatment [1] Group 1: Allegations Against Sarepta Therapeutics - Insiders at Sarepta Therapeutics allegedly caused the company to misrepresent or fail to disclose significant safety risks posed by the ELEVIDYS treatment [1] - The trial regimes and protocols for ELEVIDYS reportedly failed to detect severe side effects, leading to concerns about patient safety [1] - The severity of adverse events from ELEVIDYS treatment may result in the company halting recruitment and dosing in trials, attracting regulatory scrutiny, and increasing risks around the therapy's approvals [1] - Insiders are accused of materially misleading stakeholders and lacking a reasonable basis for their positive statements regarding ELEVIDYS [1]

Core Viewpoint - A class action lawsuit has been filed against Sarepta Therapeutics, Inc. for allegedly misleading investors regarding the safety of its gene therapy drug, ELEVIDYS, during the period from June 22, 2023, to June 24, 2025 [1][2]. Allegations - The lawsuit claims that Sarepta failed to disclose significant safety risks associated with ELEVIDYS, including the inability of trial protocols to detect severe side effects and the potential for adverse events to halt recruitment and dosing in clinical trials [2]. - Specific allegations include that the severity of adverse events would attract regulatory scrutiny and increase risks around the therapy's approvals [2]. Stock Price Impact - Following a safety update on March 18, 2025, which reported a patient death after ELEVIDYS treatment, Sarepta's stock price dropped by $27.81, or 27.44%, closing at $73.54 [3]. - On June 15, 2025, after disclosing a second patient death due to acute liver failure and suspending shipments for non-ambulatory patients, the stock fell by $15.24, or 42.12%, closing at $20.91 [3]. - The FDA's investigation announcement on June 24, 2025, regarding acute liver failure risks led to an additional stock price decline of $1.52, or 8.01%, closing at $17.46 [4]. Class Action Participation - Shareholders may be eligible to participate in the class action against Sarepta and must file a motion for lead plaintiff by August 25, 2025, to represent other class members [5].

Core Viewpoint - A class action securities lawsuit has been filed against Sarepta Therapeutics, Inc. alleging securities fraud related to the company's gene therapy product, ELEVIDYS, which is intended for treating Duchenne muscular dystrophy [1][2]. Group 1: Lawsuit Details - The lawsuit seeks to recover losses for Sarepta investors affected by alleged securities fraud between June 22, 2023, and June 24, 2025 [1]. - The complaint claims that Sarepta made false statements regarding the safety of ELEVIDYS, including significant safety risks and failure to detect severe side effects during clinical trials [2]. - It is alleged that the adverse events from ELEVIDYS treatment could lead to a halt in recruitment and dosing in trials, increased regulatory scrutiny, and greater risks regarding the therapy's approvals [2]. Group 2: Next Steps for Investors - Investors who suffered losses during the specified timeframe have until August 25, 2025, to request to be appointed as lead plaintiff in the lawsuit [3]. - Participation in the lawsuit does not require investors to incur any out-of-pocket costs or fees [3]. Group 3: Firm Background - Levi & Korsinsky, LLP has a history of securing hundreds of millions of dollars for shareholders and has been recognized as one of the top securities litigation firms in the United States for seven consecutive years [4].

Core Viewpoint - A class action lawsuit has been filed against Sarepta Therapeutics, Inc. for allegedly misleading investors regarding the safety and revenue outlook of its gene therapy ELEVIDYS, which is intended to treat Duchenne muscular dystrophy [1][3]. Company Overview - Sarepta Therapeutics is a biopharmaceutical company focused on developing gene therapies, specifically ELEVIDYS, for treating Duchenne muscular dystrophy [3]. Allegations and Misleading Statements - The lawsuit claims that Sarepta made materially false and misleading statements about ELEVIDYS, leading investors to believe it was a safe therapy with no hindrances to broader application approval [3]. - Specific allegations include: - ELEVIDYS posed significant safety risks to patients [3]. - Trial protocols failed to detect severe side effects [3]. - Adverse events would lead to halted recruitment and dosing in trials, attracting regulatory scrutiny [3]. - Positive statements made by the company lacked a reasonable basis due to undisclosed material adverse facts [3]. Stock Price Impact - Following a safety update on March 18, 2025, where a patient died after treatment with ELEVIDYS, Sarepta's stock price fell by $27.81 per share, or 27.44%, closing at $73.54 [4]. - On April 4, 2025, after the company disclosed regulatory scrutiny and halted recruitment, the stock price fell by $4.18 per share, or 7.13%, closing at $54.43 [4]. - A second patient death was reported on June 15, 2025, leading to a significant drop of $15.24 per share, or 42.12%, closing at $20.91 [4]. - Following an FDA Safety Communication on June 24, 2025, the stock price fell by $1.52 per share, or 8.01%, closing at $17.46 [4].

Core Viewpoint - The Gross Law Firm has issued a notice to shareholders of Sarepta Therapeutics, Inc. regarding a class action lawsuit alleging that the company made materially false and misleading statements about its gene therapy product, ELEVIDYS, which poses significant safety risks to patients [1]. Group 1: Allegations - The complaint alleges that during the class period from June 22, 2023, to June 24, 2025, Sarepta Therapeutics failed to disclose significant safety risks associated with ELEVIDYS, a prescription gene therapy for Duchenne [1]. - It is claimed that the trial regimes and protocols for ELEVIDYS did not adequately detect severe side effects, leading to potential regulatory scrutiny and halting of recruitment and dosing in trials [1]. - The severity of adverse events from ELEVIDYS treatment is said to have misled investors regarding the therapy's safety and approval prospects [1]. Group 2: Class Action Details - Shareholders who purchased shares of SRPT during the specified class period are encouraged to register for the class action, with a deadline set for August 25, 2025 [2]. - Once registered, shareholders will receive updates through a portfolio monitoring software regarding the status of the case [2]. - There is no cost or obligation for shareholders to participate in this class action [2]. Group 3: Law Firm Background - The Gross Law Firm is a nationally recognized class action law firm dedicated to protecting the rights of investors affected by deceit and illegal business practices [3]. - The firm aims to ensure that companies adhere to responsible business practices and seeks recovery for investors who suffered losses due to misleading statements [3].

Core Viewpoint - A securities class-action lawsuit has been initiated against Sarepta Therapeutics due to safety concerns and regulatory setbacks related to its gene therapy, ELEVIDYS, alleging that the company misled investors about the treatment's safety and efficacy [1]. Company Developments - The lawsuit claims that Sarepta misrepresented ELEVIDYS as a safe and effective treatment with a clear path to regulatory approval, which has led to significant investor losses [1]. - The European Medicines Agency (EMA) rejected ELEVIDYS on July 24, 2025, after a trial involving 125 children showed no significant improvement in movement compared to a placebo, severely impacting Sarepta's European market expansion plans [3]. - The timeline of disclosures indicates that Sarepta's stock price has faced multiple declines due to safety and regulatory news, including a patient death in an ELEVIDYS trial reported on March 18, 2025, and a subsequent investigation by EU authorities [6]. Legal and Investigative Actions - Hagens Berman, a national shareholder rights firm, is investigating claims on behalf of investors who suffered substantial losses and is encouraging individuals with relevant information to come forward [4][5]. - The firm has set a deadline of August 25, 2025, for investors to be considered as Lead Plaintiff in the lawsuit, which covers the class period from June 22, 2023, to June 24, 2025 [2].