Latest news with #Cardiomyopathy
Yahoo
6 days ago
- Business
- Yahoo
FDA Rejects CAPR's Cell Therapy BLA for Genetic Disorder, Stock Tanks
Shares of Capricor Therapeutics CAPR tanked 33% on Friday after it announced that the FDA issued a complete response letter ('CRL') to the biologics license application ('BLA') seeking approval for cell therapy deramiocel, to treat cardiomyopathy associated with Duchenne muscular dystrophy ('DMD'). DMD is a serious genetic disorder showing progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. Per the CRL, the FDA informed the company that it had concluded review of the BLA but could not approve the application in the current form. The regulatory body specifically noted that the application failed to meet the statutory requirement for substantial evidence of effectiveness and highlighted the need for additional clinical data. The CRL also highlighted certain unresolved issues within the Chemistry, Manufacturing and Controls ('CMC') section of the application. Management believes that most of these concerns have already been addressed in its prior communications with the FDA. However, the FDA did not review these materials, as they were submitted too close to the issuance of the CRL. Year to date, shares of Capricor have plunged 44.7% compared with the industry's decline of 0.9%. Image Source: Zacks Investment Research CRL to CAPR's BLA Comes as a Major Surprise The investor community seemed surprised by the CRL, especially after the FDA accepted and granted a priority review to the BLA for deramiocel in March. A decision was due on Aug. 31, 2025. More so, the FDA also did not identify any review issues with the BLA. In a regulatory update provided last month, the company also confirmed that the FDA indicated an Advisory Committee meeting would not be required for the application. The BLA was based on data from its phase II HOPE-2 and an open-label extension study compared with natural history data. A potential approval could have made deramiocel the first therapy to treat DMD cardiomyopathy. However, the latest CRL is likely to delay the approval for deramiocel, which could have given Capricor its first approved product. The company plans to hold further discussions with the FDA to determine the next steps of development for deramiocel. Capricor Therapeutics, Inc. Price Capricor Therapeutics, Inc. price | Capricor Therapeutics, Inc. Quote CAPR's Zacks Rank & Stocks to Consider Capricor currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks from the biotech sector are Arvinas ARVN, Alkermes ALKS and BioXcel Therapeutics BTAI, each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today's Zacks #1 Rank stocks here. In the past 60 days, Arvinas' 2025 loss per share estimates have improved from $1.60 to $1.51. Loss per share estimates for 2026 have narrowed from $3.28 to $2.98 during the same period. ARVN stock has plunged 59.3% year to date. Arvinas' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 82.09%. In the past 60 days, estimates for Alkermes' earnings per share have increased from $1.74 to $1.79 for 2025. During the same time, earnings per share estimates for 2026 have risen from $1.79 to $1.91. Year to date, shares of ALKS have gained 5.3%. Alkermes' earnings beat estimates in one of the trailing four quarters, while missing the same on the remaining three occasions, the negative average surprise being 8.24%. In the past 60 days, BioXcel Therapeutics' 2025 loss per share estimates have improved from $16.44 to $8.60. Loss per share estimates for 2026 have narrowed from $23.76 to $7.02 during the same period. BTAI stock has declined 67.1% year to date. BioXcel Therapeutics' earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 42.84%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Alkermes plc (ALKS) : Free Stock Analysis Report Capricor Therapeutics, Inc. (CAPR) : Free Stock Analysis Report BioXcel Therapeutics, Inc. (BTAI) : Free Stock Analysis Report Arvinas, Inc. (ARVN) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research
South Wales Argus
03-07-2025
- Entertainment
- South Wales Argus
Risca fundraiser for British Heart Foundation
The 'Have a Heart Charity Event' will take place on Friday, November 28 at the Workingman's Top Club and will feature Darren Graceland Jones and award-winning singer Savannah Mountain. "This charity is extremely dear to my heart as both my father and younger brother have been diagnosed with the condition Hypertrophic Cardiomyopathy (HCM)," said Danielle Haddigan. "On February 10 this year my father was called to London to undergo a heart transplant as a donor was found, but my world came crashing down on the March 24 when he passed away due to many complications. "During my father's life, in fact also my brother's life, the British Heart Foundation have supported them both. "With this all in mind we as a family would like to give something back to the charity." The family are asking for help with prizes for a raffle and donations as they set a fundraising target of £2,500. "We want to stress that every donation, no matter how small, is deeply appreciated and will go a long way towards helping us achieve our goal," said Ms Haddigan. Tickets cost £10 with doors opening at 6pm.
The Hindu
29-06-2025
- Health
- The Hindu
After 18-year battle with genetic heart disease, 41-year-old Bengaluru resident gets transplant
For 18 long years, life for a 41-year-old Bengaluru resident was a battle against a failing heart. Diagnosed with a genetically inherited cardiomyopathy in his early twenties, his struggle of nearly two decades ended with a life-saving heart transplant at a private hospital in the city. A marketing manager by profession, Mohammed Rafi — who hails from Kerala — was diagnosed with Hypertrophic Cardiomyopathy (HCM) at the age of 23, a disease that has been running in his family for ages. From then on, his life took an unexpected turn from being unable to go to gym and or play in cricket tournaments to a daily battle for survival. Runs in family His mother, sister, grandmother, uncle, cousin, and even his young daughter had the same condition. However, it was not until his daughter passed away, at the age of four, that they began to explore the genetic factor. Doctors at Manipal Hospital Old Airport Road, where the transplant was done, said the patient, who otherwise led an active life, experienced unusual fatigue while playing cricket during an office excursion. However, he ignored it to be a normal consequence of skipping breakfast. Other symptoms of dizziness, breathlessness, and bloating persisted for days. Worried about this sudden change in his health, he visited a doctor in Kerala, only to discover that he was in the early stages of HCM — a serious, often hereditary condition where the heart muscle becomes abnormally thick. Although he continued to lead a normal life, the physical toll began to affect his professional and personal life over time. 'Because my job included business travel to different places, I had to ask people to help push my bike through traffic. At one point, I realised I could not even lift my laptop,' he said. Initially treated by Sridhara G, Consultant - Interventional Cardiology and Cardiac Electrophysiology at Manipal Hospital Old Airport Road, for over a decade, he underwent the implantation of an AICD (Automated Implantable Cardioverter Defibrillator). While this intervention gave some relief for a couple of years, his health gradually worsened with symptoms, such as swelling in the body, constant fatigue, and reduced mobility. This was when he was diagnosed with heart failure, with a heart transplantation being the last resort. Wait for organ donors The waiting period for a heart transplant was lengthy, with a severe shortage of compatible organ donors. Registered on the transplant list since 2019, it was only three months ago that he finally underwent the transplant, on March 21, 2025. A team of doctors led by Devananda N.S., HOD and Consultant – Cardiothoracic Vascular Surgery, and Heart and Lung Transplant Surgery, conducted the surgery. After two weeks of hospital stay, he was discharged on April 5, 2025. His recovery process is still ongoing, with doctors anticipating another 3-4 months for full recovery. 'If families like Mr. Rafi's are screened early, disease modification to some extent is possible, and other asymptomatic members of the family can also be helped. This is high time we work on creating a national genetic database on such rare cardiac disorders,' said Dr. Devananda.
Yahoo
13-05-2025
- Health
- Yahoo
Innovations in Echocardiography, Cardiac MRI, and Genetic Testing Reshape Dilated Cardiomyopathy Treatment Landscape
Dublin, May 13, 2025 (GLOBE NEWSWIRE) -- The "Dilated Cardiomyopathy Market - A Global and Regional Analysis: Focus on Country and Regional Analysis - Analysis and Forecast, 2025-2035" report has been added to global dilated cardiomyopathy (DCM) market is expected to grow substantially due to increasing prevalence, advancements in diagnostic technologies, and the development of novel therapeutic options. The rising burden of heart failure, aging populations, and increasing awareness about cardiovascular diseases are key drivers of this rising prevalence of cardiovascular diseases is a major driver of the dilated cardiomyopathy (DCM) market. Conditions such as heart failure, obesity, hypertension, and diabetes are on the rise globally, significantly contributing to the increasing incidence of DCM. According to the World Health Organization, heart disease remains the leading cause of death worldwide, with DCM comprising a substantial portion of heart failure cases. This growing burden is fueling demand for effective diagnostic and therapeutic advances in diagnostic technologies, including echocardiography, cardiac MRI, and genetic testing, are improving the early detection and diagnosis of DCM, enabling timely intervention and better disease management. Additionally, the development of targeted therapies based on genetic insights and personalized medicine is opening new avenues for treatment. Drugs such as beta-blockers, ACE inhibitors, ARBs, and anticoagulants are gaining traction, especially as they address the underlying mechanisms of heart ongoing advancements, the dilated cardiomyopathy (DCM) market faces several key restraints that limit its growth potential. One of the primary challenges is the limited availability of effective treatment options for advanced-stage DCM. While medications such as ACE inhibitors and beta-blockers can help manage symptoms and slow disease progression in earlier stages, treatment options for severe cases remain scarce. Heart transplantation is often the only viable solution for advanced DCM, but donor availability is low, and the procedure is costly and associated with significant medical high cost of emerging therapies including gene therapies, biologics, and transplant procedures further restricts access, particularly in low- and middle-income regions. Moreover, the expense and complexity of genetic testing for identifying familial forms of DCM create additional barriers, especially for uninsured or underinsured patients. The absence of standardized treatment protocols also complicates clinical decision-making, making it difficult for healthcare providers to consistently deliver optimal care. Additionally, current medications can produce adverse effects such as kidney dysfunction, electrolyte imbalances, and fatigue, which pose ongoing challenges in patient management and adherence. These factors collectively hinder the broader adoption and accessibility of DCM Topics Covered1. Markets Overview1.1 Introduction1.2 Market Trends1.3 Regulatory Framework1.4 Pricing Analysis1.5 Pipeline Analysis1.6 Market Dynamics1.6.1 Impact Analysis1.6.2 Market Drivers1.6.3 Market Challenges1.6.4 Market Opportunities2. Global Dilated Cardiomyopathy Market (Region), ($Million), 2023-20352.1 North America2.1.1 Key Findings2.1.2 Market Dynamics2.1.3 Market Sizing and Forecast2.1.3.1 North America Dilated Cardiomyopathy Market, by Country2.1.3.1.1 U.S.2.1.3.1.2 Canada2.2 Europe2.2.1 Key Findings2.2.2 Market Dynamics2.2.3 Market Sizing and Forecast2.2.3.1 Europe Dilated Cardiomyopathy Market, by Country2.2.3.1.1 Germany2.2.3.1.2 U.K.2.2.3.1.3 France2.2.3.1.4 Italy2.2.3.1.5 Spain2.2.3.1.6 Rest-of-Europe2.3 Asia Pacific2.3.1 Key Findings2.3.2 Market Dynamics2.3.3 Market Sizing and Forecast2.3.3.1 Asia Pacific Dilated Cardiomyopathy Market, by Country2.3.3.1.1 China2.3.3.1.2 Japan2.3.3.1.3 India2.3.3.1.4 South Korea2.3.3.1.5 Australia2.3.3.1.6 Rest-of- Asia Pacific2.4 Rest-of-the-World2.4.1 Key Findings2.4.2 Market Dynamics2.4.3 Market Sizing and Forecast3. Global Dilated Cardiomyopathy Market: Competitive Landscape and Company Profiles3.1 Key Developments3.1.1 Mergers and Acquisitions3.1.2 Synergistic Activities3.1.3 Business Expansions and Funding3.1.4 Product Launches and Approvals3.1.5 Other Activities3.2 Company Profiles3.2.1 Bristol Myers Squibb3.2.2 Cumberland Pharmaceuticals3.2.3 Berlin Cures3.2.4 AstraZeneca3.2.5 Rocket Pharmaceuticals, Inc. (Renovacor)3.2.6 Tenaya Therapeutics4. Research MethodologyFor more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
