Ataluren

Pfizer's mini-dystrophin gene therapy fordadistrogene movaparvovec failure in the Duchenne muscular dystrophy treatment opens new opportunities for several pharmaceutical companies such as Sarepta Therapeutics (SRP-5051), Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), and others, to advance their therapies and potentially dominate the DMD market. LAS VEGAS, June 17, 2024 /PRNewswire/ -- Duchenne muscular dystrophy is the most prevalent type of muscular dystrophy found in children. This genetic condition leads to ongoing muscle weakening and degeneration. It is one among nine different forms of muscular dystrophy. According to DelveInsight's estimates, the US accounted for around ~17K total prevalent cases of Duchenne muscular dystrophy in 2023. The highest cases were found in the children of the 5-9 age group, these numbers are expected to grow by the year 2034. As per DelveInsight's analysis, the most commonly associated comorbidities accompanied with DMD included the highest cases of scoliosis accounting for around ~20% of cases followed by ADHD, cardiomyopathy, and obsessive-compulsive disorders accounting for around ~19%, ~18%, and ~15% cases respectively in the US in the year 2023. The standard of care for DMD typically involves a combination of corticosteroids and surgical interventions. Prednisone and deflazacort are commonly prescribed glucocorticoids that have been utilized for over two decades to increase muscular strength in DMD patients. Surgery may be necessary to address contractures, spinal curvature, or scoliosis, which can affect breathing and overall mobility. Additionally, interventions such as pacemakers or other cardiac devices may be utilized to improve heart function. Sarepta Therapeutics is a key player in the DMD market, boasting three approved assets and two in the current pipeline. Despite its dominance, emerging competition is evident. The historical success rate for market entry varies across the 7MM. Notably, the DMD market exhibits geographic disparities in regulatory approvals, with instances of products approved in one jurisdiction and rejected in another, despite identical clinical data, reflecting the distinct standards of regulatory bodies such as EMA and FDA. Learn more about the FDA-approved DMD drugs @ Drugs for Duchenne Muscular Dystrophy Treatment The current US DMD treatment market possesses approved products, EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), ELEVIDYS (delandistrogene moxeparvovec) in patients with DMD. In January 2024, Santhera Pharmaceuticals announced the introduction of its AGAMREE (vamorolone) to treat DMD patients aged four years and above in Germany. Santhera Pharmaceuticals has officially entered the commercial stage of its biopharma journey with the launch of this drug in Germany. ELEVIDYS, the first FDA-approved gene therapy for DMD, received accelerated approval in June 2023. However, ICER recently challenged the potential conversion of Sarepta's gene therapy to full approval, citing concerns about the surrogate endpoint used in the approval process. In the EU4 and the UK, the current market is dominated by steroid therapies along with an approved medication targeting DMD patients with the nonsense mutation, TRANSLARNA (ataluren). In Japan, the only approved treatment is VILTEPSO (viltolarsen). ELEVIDYS is a prescription gene therapy designed for ambulatory children aged 4 to 5 years with DMD and a confirmed dystrophin gene mutation. It received accelerated approval. Patients will take oral corticosteroids before and after receiving ELEVIDYS and will have weekly blood tests to monitor liver enzyme levels for three months post-treatment. In June 2023, the USFDA approved ELEVIDYS as the first gene therapy for treating pediatric patients aged 4-5 years with Duchenne Muscular Dystrophy and a confirmed DMD gene mutation. VYONDYS 53, developed by Sarepta Therapeutics, is prescribed for treating DMD in patients with a specific mutation of the DMD gene that can be addressed by exon 53 skipping. This approval is under the accelerated approval pathway, based on the observed increase in dystrophin production in the skeletal muscle of patients treated with VYONDYS 53. The FDA approved VYONDYS 53 (golodirsen) in December 2019. Additionally, the FDA granted VYONDYS 53 (golodirsen) New Chemical Entity (NCE) exclusivity until December 2024 and Orphan Drug Exclusivity until December 2026. To know more about DMD treatment options, visit @ New Treatment for Duchenne Muscular Dystrophy The DMD market is crowded with so many companies including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others are running clinical trials to improve the treatment space. The DMD pipeline possesses potential drugs in mid-stage developments to be launched shortly. Some of the promising drugs in the pipeline include Pizuglanstat (TAS-205), SRP-5051, Allogeneic Cardiosphere-derived Cells (CAP-1002), ITF2357 (Givinostat), and others that hold the potential to create a significant positive shift in the DMD market size. Discover which therapies are expected to grab major DMD market share @ Duchenne Muscular Dystrophy Market Report Italfarmaco is developing Givinostat (ITF2357), an oral small molecule that functions as a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic properties. ITF2357 increases the production of follistatin protein in muscle cells by inhibiting the HDAC enzyme, leading to increased muscle mass and prevention of muscle degeneration. This action counters the effects of myostatin, helping to alleviate the symptoms of DMD. Givinostat is currently in Phase III trials. The FDA has granted it Orphan Drug, Rare Pediatric Disease, and Fast Track designations for DMD treatment. Additionally, the European Commission has designated Givinostat as an Orphan Medicinal Product for DMD treatment. TAS-205 (pizuglanstat) is a selective inhibitor of hematopoietic prostaglandin D synthase (HPGDS), developed by Taiho Pharmaceutical. It is being advanced as a treatment for Duchenne muscular dystrophy (DMD), effective irrespective of the type of dystrophin gene mutation. By inhibiting HPGDS, which worsens the inflammatory response in the muscles of DMD patients, it helps control the decline in motor function. Currently, it is in Phase III of clinical trials. Discover more about drugs for DMD in development @ Duchenne Muscular Dystrophy Clinical Trials The anticipated launch of these emerging therapies for Duchenne muscular dystrophy are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Duchenne muscular dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for DMD is expected to grow from USD 2.1 billion in 2023 with a significant CAGR by 2034. The growth of the DMD market is expected to be mainly driven by increased diagnosed incidence, patient awareness, and a robust clinical pipeline during the forecast period (2024–2034). DelveInsight's latest published market report titled as Duchenne Muscular Dystrophy Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the DMD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The DMD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Duchenne Muscular Dystrophy Age-specific Cases of Duchenne Muscular Dystrophy Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy Mutation-specific Cases of Duchenne Muscular Dystrophy Associated Comorbidities in Duchenne Muscular Dystrophy The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM DMD market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this DMD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the DMD market. Also, stay abreast of the mitigating factors to improve your market position in the DMD therapeutic space. Related Reports Duchenne Muscular Dystrophy Epidemiology Forecast Duchenne Muscular Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Duchenne muscular dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonsense mutation Duchenne muscular dystrophy companies, including Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, among others. Becker Muscular Dystrophy Pipeline Becker Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key becker muscular dystrophy companies, including Epirium Bio, Ultragenyx, Strykagen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

- European Commission decides against adoption of negative opinion - - Translarna authorization remains active in Europe - - 2024 revenue guidance paused - WARREN, N.J., May 20, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has decided not to adopt the CHMP's negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of Translarna™ (ataluren) and has returned the opinion to the CHMP for re-evaluation. As a result, Translarna remains on the market and available for patients in Europe consistent with its current marketing authorization. The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion. "The maintenance of the current authorization of Translarna is a big win for boys and young men with nonsense mutation Duchenne muscular dystrophy in Europe," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "The efficacy and safety demonstrated in clinical trials and the long-term STRIDE registry support that Translarna fills an otherwise unmet need for nmDMD patients. We look forward to working collaboratively with CHMP on next steps once they are defined." In addition, the European Medicines Agency (EMA) has informed PTC that the decision has been taken to consider the Scientific Advisory Group (SAG) meeting for Translarna held on September 5, 2023, and all the procedural steps that followed, as invalid. Furthermore, the input from the SAG meeting held in September 2023 as well as the meeting held in January 2024 will not be considered by the CHMP in any future evaluation of Translarna. Given the inability to accurately forecast the upside impact of the continued authorization of Translarna in Europe on 2024 revenue, PTC will pause 2024 total revenue guidance at this time. Guidance will be updated at a future date. About Translarna™ (ataluren) Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States. About Duchenne Muscular Dystrophy (Duchenne) Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, Instagram, LinkedIn and Twitter at @PTCBio. For More Information: Investors: Kylie O'Keefe +1 (908) 300-0691 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statement This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this press release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the commercialization of its products and product candidates; PTC's future interactions with the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA); the outcome of any regulatory procedures; the clinical utility and potential advantages of Translarna; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from PTC's trials in Translarna; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. Acronyms: CHMP: Committee for Medicinal Products for Human Use nmDMD: nonsense mutation Duchenne muscular dystrophy SOURCE PTC Therapeutics, Inc.