• Live Feeds
    • Press Releases
    • Insider Trading
    • FDA Approvals
    • Analyst Ratings
    • Insider Trading
    • SEC filings
    • Market insights
  • Analyst Ratings
  • Alerts
  • Subscriptions
  • Settings
  • RSS Feeds
Quantisnow Logo
  • Live Feeds
    • Press Releases
    • Insider Trading
    • FDA Approvals
    • Analyst Ratings
    • Insider Trading
    • SEC filings
    • Market insights
  • Analyst Ratings
  • Alerts
  • Subscriptions
  • Settings
  • RSS Feeds
Dashboard
    Quantisnow Logo

    © 2025 quantisnow.com
    Democratizing insights since 2022

    Services
    Live news feedsRSS FeedsAlerts
    Company
    AboutQuantisnow PlusContactJobs
    Legal
    Terms of usePrivacy policyCookie policy

    June 20, 2024 - FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy

    6/20/24 5:24:42 PM ET
    $SRPT
    Biotechnology: Pharmaceutical Preparations
    Health Care
    Get the next $SRPT alert in real time by email
    For Immediate Release:
    June 20, 2024

    Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene. 

    Elevidys was previously approved under accelerated approval for ambulatory individuals 4 through 5 years of age with DMD with a confirmed mutation in the DMD gene. With today’s action, Elevidys received traditional approval in ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene, and accelerated approval in non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene. In making this decision, the FDA considered the totality of the evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent unmet medical need.

    “Today’s approval broadens the spectrum of patients with Duchenne muscular dystrophy eligible for this therapy, helping to address the ongoing, urgent treatment need for patients with this devastating and life-threatening disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “We remain steadfast in our commitment to help advance safe and effective treatments for patients who desperately need them.” 

    Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body’s muscle cells intact. 

    As a result of this genetic defect, individuals with DMD may have symptoms such as trouble walking and running, falling frequently, fatigue and learning disabilities/difficulties. They may also experience heart issues as a result of the impact on heart muscle function and breathing problems due to weakening of respiratory muscles involved in lung function. Symptoms of muscle weakness associated with DMD typically begin in childhood, often between 3 to 6 years of age. 

    DMD mainly affects males and in rare cases may affect females. About one in every 3,300 boys are affected by this disorder. As the disease progresses, life-threatening heart and respiratory problems can occur. Although disease severity and life expectancy vary, patients often succumb to the disease in their 20s or 30s because of heart and/or respiratory failure. 

    Elevidys is a recombinant gene therapy designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin, a shortened protein (138 kDa, compared to the 427 kDa dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. The product is administered as a single intravenous dose. 

    Elevidys was initially approved in June 2023 through the Accelerated Approval pathway, under which the FDA may approve drugs for serious or life-threatening diseases where there is an unmet medical need and the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit to patients (improving how patients feel or function, or whether they survive longer), or an effect on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit. This pathway can allow earlier approval, while the company conducts clinical trials to verify the predicted clinical benefit.  

    Data Supporting Traditional Approval

    The FDA granted today’s approval based on an evaluation of data submitted by the sponsor. The efficacy of Elevidys was evaluated in two double-blind, placebo-controlled studies and two open-label studies, which enrolled a total of 218 male patients (including those who received placebo) with a confirmed disease-causing mutation in the DMD gene. 

    In one of the studies, the efficacy outcome measures were to evaluate expression of micro-dystrophin in skeletal muscle, and to evaluate the effect of Elevidys on the total score of patients on the North Star Ambulatory Assessment (NSAA) — a scale commonly used to rate the motor function in males with DMD who are capable of walking. In another study, the primary efficacy outcome measure was to evaluate the effect of Elevidys on physical function as assessed by the NSAA total score. Key secondary outcome measures were to evaluate expression of micro-dystrophin in skeletal muscle, time to rise from floor and time of 10-meter walk/run. Additional efficacy outcome measures included time of 100-meter walk/run and time to ascend four steps.

    While the large, randomized study of Elevidys failed to meet its statistical primary endpoint of improvement versus placebo in the NSAA, the FDA found the observations regarding the secondary endpoints and exploratory endpoints to be compelling and to indicate clinical benefit compared to placebo. These endpoints include improvements in time to rise from the floor, 10-meter walk/run, time to ascend four steps and creatine kinase levels. 

    Based on the totality of the evidence, the FDA determined the available evidence verifies the product’s clinical benefit for its original indication, which was initially approved in June 2023 under accelerated approval, and provides substantial evidence of effectiveness to support traditional approval of Elevidys in ambulatory individuals 4 years of age and older with a confirmed mutation in the DMD gene except in those with any deletion in exon 8 and/or exon 9 in the DMD gene, in whom its use is contraindicated. An inadequate amount of safety data is available currently to support the use of Elevidys in individuals under 4 years of age.

    Data Supporting Accelerated Approval

    In granting accelerated approval for non-ambulatory individuals aged 4 and older, the FDA considered the totality of the evidence, including clinical data in ambulatory individuals from a study in 4- to 7-year-olds, as well as from a study in 4- to 5-year-olds indicating a correlation of Elevidys micro-dystrophin levels with clinical outcome measures. Based on the evidence and given that the mechanism of action of Elevidys is similar for ambulatory and non-ambulatory populations, the FDA determined that increased levels in micro-dystrophin is reasonably likely to predict clinical benefit in the non-ambulatory population. This conclusion, along with the evidence that Elevidys elevates micro-dystrophin levels, provides substantial evidence of effectiveness to support accelerated approval in non-ambulatory individuals at least 4 years of age with DMD considering the serious nature of the disease and the extent of unmet medical need in this group of individuals. A confirmatory randomized, controlled clinical trial in the non-ambulatory population is currently underway. 

    The safety of Elevidys was established based on evaluation of 156 male patients with a confirmed mutation of the DMD gene who received the product in four clinical studies, including one completed open-label study, one ongoing open-label study, and two studies that included a double-blind, placebo-controlled period. No new safety concerns appear to have been identified in the population of ambulatory individuals treated with the marketed product. A modest amount of safety data on non-ambulatory individuals was submitted in the context of an ongoing randomized clinical trial; safety data in non-ambulatory individuals is limited, given the number of non-ambulatory individuals included in the trial and treated with the marketed product to date. 

    The most commonly reported side effects by individuals who received Elevidys were vomiting, nausea, acute liver injury, fever and thrombocytopenia (abnormally low platelet count in the blood). Patients’ liver function should be monitored before treatment with Elevidys, and weekly for the first three months after treatment. Patients given Elevidys may also be at risk for severe immune-mediated myositis (muscle inflammation). Additionally, myocarditis (inflammation of heart muscle) and elevations of troponin-I (a heart protein found in the blood after heart muscle injury) have been observed following use of Elevidys in clinical trials. Troponin-I levels should be monitored before administration of Elevidys and weekly for the first month after treatment. 

    The FDA granted the approval and accelerated approval for Elevidys to Sarepta Therapeutics Inc.

    Related Information

    Related Information
    • ELEVIDYS | FDA
    • Package Insert
    • FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy

    ###

    Boilerplate

    The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.


    Inquiries

    Media:
    Carly Pflaum
    240-672-8872
    Consumer:
    888-INFO-FDA

     
    Get the next $SRPT alert in real time by email

    Chat with this insight

    Save time and jump to the most important pieces.

    Recent Analyst Ratings for
    $SRPT

    DatePrice TargetRatingAnalyst
    5/8/2025$50.00Outperform → In-line
    Evercore ISI
    4/11/2025$115.00Overweight
    Wells Fargo
    4/2/2025$75.00Sell → Neutral
    H.C. Wainwright
    3/31/2025$161.00 → $87.00Outperform → Sector Perform
    RBC Capital Mkts
    2/11/2025$136.00Hold
    Deutsche Bank
    11/27/2024$205.00 → $202.00Buy
    Needham
    11/25/2024$80.00Sell
    H.C. Wainwright
    11/7/2024$152.00 → $167.00Neutral → Overweight
    Cantor Fitzgerald
    More analyst ratings

    $SRPT
    Analyst Ratings

    Analyst ratings in real time. Analyst ratings have a very high impact on the underlying stock. See them live in this feed.

    See more
    • Sarepta Therapeutics downgraded by Evercore ISI with a new price target

      Evercore ISI downgraded Sarepta Therapeutics from Outperform to In-line and set a new price target of $50.00

      5/8/25 8:27:43 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Wells Fargo initiated coverage on Sarepta Therapeutics with a new price target

      Wells Fargo initiated coverage of Sarepta Therapeutics with a rating of Overweight and set a new price target of $115.00

      4/11/25 9:10:00 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Sarepta Therapeutics upgraded by H.C. Wainwright with a new price target

      H.C. Wainwright upgraded Sarepta Therapeutics from Sell to Neutral and set a new price target of $75.00

      4/2/25 8:22:16 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $SRPT
    FDA approvals

    Live FDA approvals issued by the Food and Drug Administration and FDA breaking news

    See more
    • FDA Approval for AMONDYS 45 issued to SAREPTA THERAPS INC

      Submission status for SAREPTA THERAPS INC's drug AMONDYS 45 (SUPPL-8) with active ingredient CASIMERSEN has changed to 'Approval' on 07/11/2024. Application Category: NDA, Application Number: 213026, Application Classification: Labeling

      7/12/24 4:40:36 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • June 20, 2024 - FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy

      For Immediate Release: June 20, 2024 Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene.  Elevidys was previously approved under accelerated approval for amb

      6/20/24 5:24:42 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • FDA Approval for VYONDYS 53 issued to SAREPTA THERAPS INC

      Submission status for SAREPTA THERAPS INC's drug VYONDYS 53 (SUPPL-11) with active ingredient GOLODIRSEN has changed to 'Approval' on 06/04/2024. Application Category: NDA, Application Number: 211970, Application Classification: Labeling

      6/5/24 4:41:31 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $SRPT
    Insider Trading

    Insider transactions reveal critical sentiment about the company from key stakeholders. See them live in this feed.

    See more
    • Director Nicaise Claude exercised 9,746 shares at a strike of $25.18 and sold $248,203 worth of shares (2,491 units at $99.64), increasing direct ownership by 35% to 27,812 units (SEC Form 4)

      4 - Sarepta Therapeutics, Inc. (0000873303) (Issuer)

      3/13/25 8:00:04 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Director Boor Kathryn Jean was granted 2,383 shares, increasing direct ownership by 41% to 8,263 units (SEC Form 4)

      4 - Sarepta Therapeutics, Inc. (0000873303) (Issuer)

      3/11/25 8:00:18 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Director Connelly Deirdre P was granted 2,383 shares, increasing direct ownership by 84% to 5,225 units (SEC Form 4)

      4 - Sarepta Therapeutics, Inc. (0000873303) (Issuer)

      3/11/25 8:00:20 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $SRPT
    Large Ownership Changes

    This live feed shows all institutional transactions in real time.

    See more
    • Amendment: SEC Form SC 13G/A filed by Sarepta Therapeutics Inc.

      SC 13G/A - Sarepta Therapeutics, Inc. (0000873303) (Subject)

      11/13/24 10:27:59 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • SEC Form SC 13G filed by Sarepta Therapeutics Inc.

      SC 13G - Sarepta Therapeutics, Inc. (0000873303) (Subject)

      2/9/24 6:05:54 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • SEC Form SC 13G/A filed by Sarepta Therapeutics Inc. (Amendment)

      SC 13G/A - Sarepta Therapeutics, Inc. (0000873303) (Subject)

      2/14/23 3:23:55 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $SRPT
    Press Releases

    Fastest customizable press release news feed in the world

    See more

    $SRPT
    Financials

    Live finance-specific insights

    See more
    • Sarepta Therapeutics to Present at the BofA Securities Health Care Conference

      Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will participate in a fireside chat at the BofA Securities Health Care Conference at the Encore Hotel in Las Vegas, Nev. on Wednesday, May 14, 2025 at 8:40 a.m. P.T./11:40 a.m. E.T. The presentations will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and will be archived there following the presentation for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be

      5/8/25 4:30:00 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Sarepta Therapeutics Announces First Quarter 2025 Financial Results and Recent Corporate Developments

      Net product revenues for the first quarter 2025 totaled $611.5 million, a 70% increase over the same quarter of the prior year ELEVIDYS net product revenue for the first quarter totaled $375.0 million; Royalty revenue from the sales of ELEVIDYS by Roche for the quarter totaled $4.0 million Revised 2025 total net product revenues guidance to $2.3 to $2.6 billion Meaningfully advanced multiple clinical candidates in limb-girdle muscular dystrophy portfolio Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2025. "In the first quarter, we achieved net product revenue of $611.5 mi

      5/6/25 4:05:00 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Sarepta Therapeutics to Announce First Quarter 2025 Financial Results

      Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report first quarter 2025 financial results after the Nasdaq Global Market closes on Tuesday, May 6, 2025. Subsequently, at 4:30 p.m. E.T., the Company will host a conference call to discuss its first quarter 2025 financial results. The event will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone

      4/22/25 8:30:00 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Sarepta Therapeutics Announces First Quarter 2025 Financial Results and Recent Corporate Developments

      Net product revenues for the first quarter 2025 totaled $611.5 million, a 70% increase over the same quarter of the prior year ELEVIDYS net product revenue for the first quarter totaled $375.0 million; Royalty revenue from the sales of ELEVIDYS by Roche for the quarter totaled $4.0 million Revised 2025 total net product revenues guidance to $2.3 to $2.6 billion Meaningfully advanced multiple clinical candidates in limb-girdle muscular dystrophy portfolio Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2025. "In the first quarter, we achieved net product revenue of $611.5 mi

      5/6/25 4:05:00 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Sarepta Therapeutics to Announce First Quarter 2025 Financial Results

      Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report first quarter 2025 financial results after the Nasdaq Global Market closes on Tuesday, May 6, 2025. Subsequently, at 4:30 p.m. E.T., the Company will host a conference call to discuss its first quarter 2025 financial results. The event will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone

      4/22/25 8:30:00 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2024 Financial Results and Recent Corporate Developments

      – Net product revenues for the fourth quarter 2024 totaled $638.2 million, a 75% increase over the same quarter of the prior year – ELEVIDYS net product revenue for the quarter totaled $384.2 million; Royalty revenue from the sales of ELEVIDYS by Roche for the quarter totaled $4.9 million – Achieved GAAP and non-GAAP net income of $159.0 million and $206.0 million for the fourth quarter of 2024, respectively Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2024. "2024 performance represented the fruition of our multi-year strategy to become a self-sustaining pro

      2/26/25 4:05:00 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $SRPT
    Leadership Updates

    Live Leadership Updates

    See more
    • Sarepta Therapeutics Appoints Deirdre Connelly to its Board of Directors

      - Veteran biopharmaceutical executive from GSK and Eli Lilly brings more than 30 years of experience to Sarepta's Board Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the appointment of Deirdre P. Connelly to its Board of Directors. Ms. Connelly is a well-regarded executive with more than 30 years of experience in the pharmaceutical industry. Following Connelly's appointment, Sarepta's Board will comprise nine directors, eight of whom are independent. "Ms. Connelly is a seasoned board leader with extensive operating experience in biopharma and we're pleased to welcome her to the Sarepta Board of Directors as we work to

      9/16/24 8:30:00 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Cassava Sciences Announces Changes in Executive Leadership, Enhanced Corporate Governance and Other Initiatives

      Rick Barry appointed Executive Chairman of the BoardRemi Barbier resigns as President and CEO and from the Board of DirectorsCassava initiates search for a new CEO AUSTIN, Texas, July 17, 2024 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (NASDAQ:SAVA) today announced that the Board of Directors has appointed Richard (Rick) Barry as Executive Chairman of the Board and as the Company's principal executive officer, effective immediately. The Company is undertaking a search for a new permanent CEO. Mr. Barry succeeds Remi Barbier, the Company's Chairman, President and CEO, who resigned from the Company and the Board. Mr. Barbier will remain employed by the Company until September 13, 2024 in a

      7/17/24 8:00:00 AM ET
      $MDXG
      $SAVA
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Sarepta Therapeutics Set to Join S&P MidCap 400

      NEW YORK, May 29, 2024 /PRNewswire/ -- Sarepta Therapeutics Inc. (NASD: SRPT) will replace Shockwave Medical Inc. (NASD: SWAV) in the S&P MidCap 400 effective prior to the opening of trading on Monday, June 3. S&P 500 constituent Johnson & Johnson (NYSE:JNJ) is acquiring Shockwave Medical in a deal expected to be completed soon pending final closing conditions. Following is a summary of the changes that will take place prior to the open of trading on the effective date: Effective Date Index Name       Action Company Name Ticker GICS Sector June 3, 2024 S&P MidCap 400 Addition Sarepta Therapeutics SRPT Health Care June 3, 2024 S&P MidCap 400 Deletion Shockwave Medical SWAV Health Care For

      5/29/24 5:48:00 PM ET
      $JNJ
      $SPGI
      $SRPT
      $SWAV
      Biotechnology: Pharmaceutical Preparations
      Health Care
      Finance: Consumer Services
      Finance

    $SRPT
    Insider Purchases

    Insider purchases reveal critical bullish sentiment about the company from key stakeholders. See them live in this feed.

    See more
    • Director Chambers Michael Andrew bought $4,955,728 worth of shares (37,038 units at $133.80) (SEC Form 4)

      4 - Sarepta Therapeutics, Inc. (0000873303) (Issuer)

      8/20/24 8:00:04 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Barry Richard bought $3,940,500 worth of shares (50,000 units at $78.81) (SEC Form 4)

      4 - Sarepta Therapeutics, Inc. (0000873303) (Issuer)

      11/6/23 8:00:11 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • Ingram Douglas S bought $2,001,800 worth of shares (25,225 units at $79.36), increasing direct ownership by 7% to 390,307 units (SEC Form 4)

      4 - Sarepta Therapeutics, Inc. (0000873303) (Issuer)

      11/6/23 8:00:10 AM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care

    $SRPT
    SEC Filings

    See more
    • SEC Form 10-Q filed by Sarepta Therapeutics Inc.

      10-Q - Sarepta Therapeutics, Inc. (0000873303) (Filer)

      5/6/25 4:01:26 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • SEC Form DEFA14A filed by Sarepta Therapeutics Inc.

      DEFA14A - Sarepta Therapeutics, Inc. (0000873303) (Filer)

      4/24/25 4:10:03 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care
    • SEC Form DEF 14A filed by Sarepta Therapeutics Inc.

      DEF 14A - Sarepta Therapeutics, Inc. (0000873303) (Filer)

      4/24/25 4:05:17 PM ET
      $SRPT
      Biotechnology: Pharmaceutical Preparations
      Health Care