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  • HC bins Roche's injunction plea, allows generic version of only drug for SMA

    HC bins Roche's injunction plea, allows generic version of only drug for SMA

    • Published On Oct 11, 2025 at 12:32 PM IST
    • Read by 100 Professionals

    Times News Network

    A division bench of Delhi high court Thursday rejected the appeal of pharma giant, Roche, seeking an injunction against Indian pharma company Natco Pharma launching a generic version of Risdiplam, the only available drug to treat a rare genetic disorder, spinal muscular atrophy (SMA). This comes as a major relief for Indian SMA patients.

    Govt’s one-time aid of Rs 50 lakh for patients of rare diseases was not enough to cover even a year’s treatment using Roche’s patented version of Risdiplam, but at Natco’s price for the generic version of the drug, treatment cost could be covered for 10-16 years.

    In March this year, a single-judge bench of Delhi HC rejected Roche’s injunction plea against Hyderabad-based Natco Pharma in a patent infringement dispute related to Risdiplam. Roche’s patent expires only in March 2035. However, Natco applied for a patent for an improved process for the preparation of Risdiplam and intermediates in Sept 2022. The court had ruled Natco had prima facie raised credible challenges to the validity of Roche’s patent and ruled in favour of Natco.

    Several public health groups had opposed Roche seeking a permanent injunction against Natco, arguing that Roche’s patent infringement lawsuit against Natco prioritised profits over patients’ access to affordable treatment, potentially violating India’s National Rare Disease Policy.

    Roche’s patented medicine costs roughly Rs 6 lakh a bottle. An individual with a body weight of over 20kg needs roughly 2.5-3 bottles per month, or 30-36 bottles per year. Roche gives two bottles for free if you buy one, and so the patient would have to pay about Rs 60-72 lakh per year for treatment, unaffordable for most patients in India. Drug costing experts peg the cost of producing the drug at roughly Rs 3,000 for a year’s supply.

    In April, Natco revealed in a “legal update regarding Risdiplam launch in India” that the “company has decided to price the product (Risdiplam) at Rs 15,900” per 60mg bottle, reducing the price by as much as 97%. This would bring down the cost to less than Rs 5 lakh per year. Bulk buying by govt could bring down the price to Rs 10,000 per bottle, which could bring the cost down to Rs 3.1 lakh per year, said health activists. The financial assistance for patients with rare diseases was increased to Rs 50 lakh in Sept 2024. At Natco’s price, those living with SMA would be able to afford treatment for up to 16 years.

    The Working Group on Access to Medicines and Treatment welcomed HC’s decision and said it highlighted harmful effects of patent evergreening, which could delay access to affordable medicines and lead to unnecessary litigation. It reaffirmed that access to lifesaving drugs should not depend on one’s ability to pay.

    • Published On Oct 11, 2025 at 12:32 PM IST

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  • CDSCO to inspect cough syrup manufacturing units

    CDSCO to inspect cough syrup manufacturing units

    Teena Thacker

    New Delhi: The central drug regulator is set to launch nationwide inspections and auditing of cough syrup manufacturing units even as the World Health Organization sought clarification from India on recent child deaths allegedly after consuming toxic syrup.

    The Central Drugs Standard Control Organisation (CDSCO) has asked all states and union territories to provide a list of cough syrup manufacturers in their jurisdiction for audit and surveillance as part of a drive to ensure stricter quality control, people aware of the development said.

    At least 15 children aged below six have died in Madhya Pradesh and Rajasthan, allegedly due to toxic cough syrups. Government officials, however, said the deaths reported in Rajasthan are not linked to cough syrups.

    The WHO has, meanwhile, expressed concern regarding the regulatory gap in diethylene glycol and ethylene glycol screening for domestically marketed medicines in India.

    DEG has been detected in at least three oral syrup medicines tested so far, the CDSCO has informed the international health agency. The WHO said it is monitoring media reports regarding clusters of paediatric illnesses and deaths in Madhya Pradesh and Rajasthan.

    “These reports…describe symptoms consistent with acute renal failure and acute encephalitis syndrome, with suspected links to the use of oral syrup medicines. WHO is deeply saddened by the reported fatalities and extends its sincere condolences to the affected families and communities,” it said.

    “WHO communicated that it stood ready to support national authorities in investigating and responding to these tragic events,” one of the persons cited above said.

    The WHO had on October 1 contacted the CDSCO for clarification regarding potential links to contaminated medicines and whether the products were exported to other countries.

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  • US FDA approves first treatment for rare, life-threatening disease in males

    US FDA approves first treatment for rare, life-threatening disease in males

    • Published On Sep 20, 2025 at 06:49 AM IST
    • Read by 100 Professionals

    Bengaluru: The U.S. Food and Drug Administration said on Friday it had approved Stealth Biotherapeutics‘ once-daily injection for a rare, genetic disease that primarily affects males, making it the first treatment to be greenlit for the condition.

    Stealth’s Forzinity was granted accelerated approval for treating Barth syndrome, in patients weighing at least 30 kg.

    Barth syndrome is a rare, serious and life-threatening disease of the mitochondria, the energy-producing parts of cells.

    It typically starts with severe heart failure in infancy, and causes premature death. Patients who survive into adolescence and adulthood often have fatigue, poor stamina and exercise intolerance, according to the FDA.

    According to a study on the National Institutes of Health’s website, as of 2020, an estimated 230 to 250 males were identified with Barth syndrome worldwide.

    The under-the-skin or subcutaneous injection works by binding to the inner part of the mitochondria, improving its structure and function.

    The accelerated approval is based on improved strength of the muscle used to straighten the leg at the knee. The FDA said it considers this improvement reasonably likely to predict patient benefit, such as an ability to stand more easily or walk farther.

    Under the FDA’s accelerated approval pathway that allows earlier approval of medications that treat serious conditions and fill an unmet medical need, it mandates additional post-market trials that verify the drug’s benefits.

    Privately held Stealth will conduct a trial to confirm the changes seen on knee muscle strength translate into patient benefit.

    If data from the trial do not show the drug’s effectiveness, the regulator can ask the company to withdraw it from the market.

    (Reporting by Puyaan Singh in Bengaluru; Editing by Krishna Chandra Eluri)

    • Published On Sep 20, 2025 at 06:49 AM IST

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  • 4 dead in gas leak at pharma firm

    4 dead in gas leak at pharma firm

    • Published On Aug 23, 2025 at 11:44 AM IST
    • Read by 100 Professionals

    Mumbai: Four workers were killed and two others hospitalised after inhalation of nitrogen gas that leaked at a pharmaceutical company in Tarapur MIDC of Maharashtra’s Palghar district on Thursday, officials said.

    The incident occurred in the afternoon at Medley Pharma located in the industrial area of Boisar, around 130 km from here, they said.

    Chief of Palghar district disaster management cell, Vivekanand Kadam, said that between 2.30 pm and 3 pm, gas leaked from the nitrogen reaction tank at the pharma company, seriously affecting six persons working there.

    “The six workers were rushed to a hospital, where four of them died around 6.15 pm,” he said.

    Two others are currently undergoing treatment in the intensive care unit (ICU) at the hospital, he added. The deceased were identified as Kalpesh Raut, Bangali Thakur, Dheeraj Prajapati and Kamlesh Yadav, while Rohan Shinde and Nilesh Hadal are undergoing treatment, officials said.

    • Published On Aug 23, 2025 at 11:44 AM IST

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  • Recent Changes to References, Search Tools, Related Citations, and Sharing Features

    Recent Changes to References, Search Tools, Related Citations, and Sharing Features

    As part of our commitment to continuous improvement, PubMed recently introduced several updates designed to improve usability and consistency across the platform. These changes are informed by user feedback and aim to facilitate a more efficient and accessible experience for all PubMed users. Key changes include improved rendering of reference lists, the addition of date stamps in search history downloads, streamlined sharing options, and other usability updates.

    Formatting in reference lists

    Reference lists displayed on PubMed abstract pages now render formatting such as bold, italics, superscripts, and subscripts when provided by the publisher. This change ensures meaningful formatting such as superscript and subscript characters are preserved, and references retain the citation style provided to PubMed.

    Date stamps in search history download

    PubMed search history downloads from the Advanced Search page now include date stamps for each search. This feature can be used to help facilitate record keeping and reporting, for example when conducting systematic reviews. Please note that the date format may vary depending on the local settings configured in the software program you use to open CSV files.

    Usability improvement to “See all cited by articles” and “See all similar articles”

    Abstract pages include links to citations for similar articles and cited by articles, when available. You can now right click on the “See all cited by articles” and “See all similar articles” links to open these results in a new tab or window, and clicking these links now clear any applied filters so that the entire cited by or similar article set will be displayed.

    Searches related to [your query]

    The “Searches related to [your query]” feature located below the PubMed search results has been removed. These related searches continue to be auto-suggested as you enter terms in the PubMed search bar, consolidating this feature to where it is most used.

    Streamlined share functionality

    Platform-specific social media share buttons have been removed in favor of the universal permalink option that can be shared on any platform of your choice. This change helps to save space on the search results page for the most compact search results view and aligns PubMed’s abstract page with the updated PubMed Central (PMC) website, providing a consistent user experience.

    To get a citation permalink to share:

    • From the search results, right-click an article title and choose the copy link option; exact wording and functionality may change depending on your operating system and browser.
    • From an abstract page, click the Permalink button on the right sidebar and click “Copy”.
    • From a mobile device, use your device’s native share functionality to copy a link or share to the social media app of your choice.

    Please see the following Technical Bulletin articles for other recent updates:

  • Breast Cancer Risk in Younger Women May Be Influenced by Hormone Therapy

    Breast Cancer Risk in Younger Women May Be Influenced by Hormone Therapy

    Scientists at the National Institutes of Health (NIH) have found that two common types of hormone therapy may alter breast cancer risk in women before age 55. Researchers discovered that women treated with unopposed estrogen hormone therapy (E-HT) were less likely to develop the disease than those who did not use hormone therapy. They also found that women treated with estrogen plus progestin hormone therapy (EP-HT) were more likely to develop breast cancer than women who did not use hormone therapy. Together, these results could help to guide clinical recommendations for hormone therapy use among younger women.

    The two hormone therapies analyzed in the study are often used to manage symptoms related to menopause or following hysterectomy (removal of uterus) or oophorectomy (removal of one or both ovaries). Unopposed estrogen therapy is recommended only for women who have had a hysterectomy because of its known association with uterine cancer risk.

    “Hormone therapy can greatly improve the quality of life for women experiencing severe menopausal symptoms or those who have had surgeries that affect their hormone levels,” said lead author Katie O’Brien, Ph.D., of NIH’s National Institute of Environmental Health Sciences (NIEHS). “Our study provides greater understanding of the risks associated with different types of hormone therapy, which we hope will help patients and their doctors develop more informed treatment plans.”

    The researchers conducted a large-scale analysis that included data from more than 459,000 women under 55 years old across North America, Europe, Asia, and Australia. Women who used E-HT had a 14% reduction in breast cancer incidence compared to those who never used hormone therapy. Notably, this protective effect was more pronounced in women who started E-HT at younger ages or who used it longer. In contrast, women using EP-HT experienced a 10% higher rate of breast cancer compared to non-users, with an 18% higher rate seen among women using EP-HT for more than two years relative to those who never used the therapy.

    According to the authors, this suggests that for EP-HT users, the cumulative risk of breast cancer before age 55 could be about 4.5%, compared with a 4.1% risk for women who never used hormone therapy and a 3.6% risk for those who used E-HT. Further, the association between EP-HT and breast cancer was particularly elevated among women who had not undergone hysterectomy or oophorectomy. That highlights the importance of considering gynecological surgery status when evaluating the risks of starting hormone therapy, the researchers noted.

    “These findings underscore the need for personalized medical advice when considering hormone therapy,” said NIEHS scientist and senior author Dale Sandler, Ph.D. “Women and their health care providers should weigh the benefits of symptom relief against the potential risks associated with hormone therapy, especially EP-HT. For women with an intact uterus and ovaries, the increased risk of breast cancer with EP-HT should prompt careful deliberation.”

    The authors noted that their study is consistent with previous large studies that documented similar associations between hormone therapy and breast cancer risk among older and postmenopausal women. This new study extends those findings to younger women, providing essential evidence to help guide decision-making for women as they go through menopause.

    Reference: O’Brien KM, et al. 2025. Hormone therapy use and young-onset breast cancer: a pooled analysis of prospective cohorts included in the Premenopausal Breast Cancer Collaborative Group. Lancet Oncol 26: 911–23.


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  • US FDA approves Gilead's twice-yearly injection for HIV prevention

    US FDA approves Gilead's twice-yearly injection for HIV prevention

    By Deena Beasley and Julie Steenhuysen

    Los Angeles: The U.S. Food and Drug Administration on Wednesday approved Gilead Sciences lenacapavir, a twice-yearly injection, for preventing HIV infection in adults and adolescents at high risk of contracting the deadly virus.

    Investors and AIDS activists had been eagerly awaiting the regulatory decision for the drug seen as convenient enough to help end the 44-year-old HIV epidemic.

    It will be sold under the brand name Yeztugo in the U.S. at a list price of $28,218 a year.

    Lenacapavir, part of a class of drugs known as capsid inhibitors, proved nearly 100% effective at preventing HIV in large trials last year, raising new hope of interrupting transmission of the virus that infects 1.3 million people a year.

    Yeztugo “will only be as effective as it is accessible and affordable,” Kevin Robert Frost, CEO of the Foundation for AIDS Research, said in a statement, calling on Gilead and the U.S. government to make sure people who want lenacapavir can get it.

    Gilead said it is working to secure health insurer coverage. It said it will provide co-pay assistance for eligible insured people, and the drug may be available free of charge for some under its program for the uninsured.

    Medications to prevent HIV, known as pre-exposure prophylaxis, or PrEP, are widely available. But most are daily pills, including low-cost generic versions of Gilead’s older drug Truvada, that require strict adherence to be effective. Gilead said Yeztugo is priced in line with other branded drugs.

    “This is a milestone moment,” said Gilead Chief Executive Daniel O’Day of the approval.

    “We believe that lenacapavir is the most important tool we have yet to bend the arc of the epidemic and move this epidemic into the history books,” O’Day said.

    Availability of a twice-yearly HIV prevention tool is “a huge advance,” that could help change the course of the epidemic, Dr. Raphael Landovitz, director of the UCLA Center for Clinical AIDS Research & Education, said in an email. But he said the product’s high launch price “is almost certainly going to complicate payor coverage and access.”

    Gilead has plans for a rapid launch in the United States as well as a wider rollout of the drug in collaboration with global partners.

    Gilead’s chief commercial officer, Johanna Mercier, said the company’s “end game” is to normalize PrEP usage, both in the United States and other countries, including low-income African nations where the virus is most prevalent.

    Citi Research analyst Geoff Meacham said he expects Yeztugo’s launch to be slow and steady, reaching annual sales of $2.8 billion by 2030.

    Mercier said she expects around 75% of U.S. insurers, including government health plans, will cover lenacapavir for PrEP within about six months, with the number rising to 90% within 12 months of launch.

    The drug is currently sold as a treatment in the U.S. under the brand name Sunlenca for patients with advanced disease that has become resistant to other drugs.

    PEPFAR CUTS

    In December, the President’s Emergency Plan for AIDS Relief (PEPFAR) under then-President Joe Biden signed an agreement with the Global Fund to Fight AIDS, Tuberculosis and Malaria to provide the treatment to as many as 2 million people for three years if it won U.S. regulatory approval for prevention.

    That would allow for unprecedented early access to a state-of-the-art treatment, as six generic drugmakers that have licensed the product from Gilead gear up for production of low-cost versions in 120 resource-limited countries.

    AIDS activists have viewed the drug as a way to significantly slow the epidemic, but cuts to PEPFAR by the Trump administration have raised concerns about the U.S. government’s commitment to the rollout.

    O’Day acknowledged that the changes have been “challenging,” but said the company has continued to have discussions with both the Global Fund and PEPFAR.

    “I believe that there will be sources of funding for this, and that these organizations will prioritize this type of prevention,” he said.

    (Reporting by Deena Beasley in Los Angeles and Julie Steenhuysen in Chicago; Editing by Bill Berkrot and Leslie Adler)

    • Published On Jun 19, 2025 at 11:39 AM IST

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  • Weight-loss surgery more effective than drugs in real world study

    Weight-loss surgery more effective than drugs in real world study

    London: Bariatric surgeries led to about five-times more weight loss than weekly injections of popular GLP-1 drugs, according to data from a real-world comparison study presented on Tuesday at the American Society for Metabolic and Bariatric Surgery scientific meeting in Washington.

    “Clinical trials show weight loss between 15% to 21% for GLP-1s, but this study suggests that weight loss in the real world is considerably lower even for patients who have active prescriptions for an entire year,” study leader Dr. Avery Brown of NYU Langone Health said in a statement.

    Researchers reviewed records of 38,545 patients who were prescribed injectable semaglutide or tirzepatide between 2018 and 2024 and 12,540 patients who underwent bariatric surgery during the same period. Everyone started the study with a body mass index of at least 35, which is considered severe obesity.

    Semaglutide is the main ingredient in Novo Nordisk‘s Wegovy and Ozempic, while tirzepatide is the main ingredient in Eli Lilly‘s Zepbound and Mounjaro.

    At three years after undergoing sleeve gastrectomy or gastric bypass or starting the drugs, patients who underwent surgery had lost on average 24% of their starting weight, compared to about 5% for similar patients who used the drugs for at least six months and about 7% for those who took them for a year.

    Brown noted that as many as 70% of GLP-1 patients may discontinue treatment within one year.

    “While both patient groups lose weight, metabolic and bariatric surgery is much more effective and durable,” ASMBS President Dr. Ann M. Rogers, who was not involved in the study, said in a statement.

    “Those who get insufficient weight loss with GLP-1s or have challenges complying with treatment due to side effects or costs, should consider bariatric surgery as an option, or even in combination,” she said.

    NEW BLOOD TEST MAY REDUCE LIVER TRANSPLANT FAILURES

    An experimental blood test can help surgeons catch and identify problems with newly transplanted livers at early stages, researchers say.

    It’s not unusual for transplanted organs and recipients’ nearby tissues to sustain damage during the transplantation process. Hints of problems will show up later in routine blood tests, but identifying the precise site of the damage often requires costly imaging studies and surgical biopsies, according to a report published on Tuesday in Nature Communications.

    The new test works by picking up DNA fragments left behind in the blood by dying cells. The chemical signatures on these DNA fragments can be used to identify the original cell type and where it came from, with precise detail, the researchers found.

    If you can determine which part of the liver is injured – for example, the bile ducts, or the blood vessels – “you could provide a more personalized treatment approach that leads to better care for the patient,” study leader Dr. Alexander Kroemer of MedStar Georgetown University Hospital in Washington, DC said in a statement.

    In addition to being faster and less invasive than a traditional biopsy, the blood test is also potentially more accurate, because biopsies only sample a few spots in the liver and might miss the site of the problem, he added.

    Georgetown has filed patent applications on the technology, and the research team is seeking partners to commercialize the test.

    DIABETES PRECURSOR SHOWS UP ON MUSCLE ULTRASOUND

    Ultrasound exams of thigh or shoulder muscles can detect insulin resistance at its earliest stages, researchers reported in the Journal of Ultrasound in Medicine.

    “We perform a large number of shoulder ultrasounds and noticed that many patients’ muscles appear unusually bright,” study leader Dr. Steve Soliman of the University of Michigan said in a statement.

    His team discovered in earlier studies that most of these patients have type 2 diabetes. But some had bright muscles on ultrasound even with no signs of diabetes or prediabetes.

    Subsequently, upon short-term follow-up, these patients often also developed prediabetes or type 2 diabetes.

    To test the potential for noninvasive muscle ultrasound as a predictive tool for detecting the development of pre- or type 2 diabetes – potentially earlier than current methods – the researchers performed muscle ultrasounds on 25 patients who were also being evaluated for insulin resistance.

    Although muscle ultrasound could detect insulin resistance and impaired insulin sensitivity, in this small study the level of brightness was not directly correlated with the degree of the condition. The researchers are recruiting more participants to continue the analysis.

    The exact reason why muscle brightness on ultrasound might indicate insulin resistance is less clear than the finding that it does, the researchers said.

    “Clinicians increasingly use these point-of-care and handheld ultrasound devices, sometimes called ‘the stethoscope of the future,’ for rapid diagnosis of various conditions,” Soliman said.

    “A medical assistant or clinician with little to no training could easily use this device on a patient’s upper arm or thigh, as routinely as checking weight or blood pressure, and potentially flag patients as ‘high risk’ or ‘low risk’ for further testing.”

    (Reporting by Nancy Lapid; editing by Bill Berkrot)

    • Published On Jun 18, 2025 at 05:13 PM IST

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  • FTP Improvements Available for Testing

    FTP Improvements Available for Testing

    In response to user feedback, the National Center for Biotechnology Information (NCBI) is announcing an upcoming update to the data provided through PubMed’s public FTP servers.

    Starting with the annual baseline release in December 2025, the content of PubMed data distributed via FTP will be generated using the same technology as the PubMed website and E-utilities API; this update means the FTP data will match the PubMed website and API.

    Test Site Available Now

    To help users prepare for this transition, a test FTP site is available now with daily update files generated by the new process. Please note, the test files may differ from the current FTP update files (see Expected Data Differences below).

    A complete baseline will not be available on the test site; only update files will be posted.

    We encourage users to review the sample files, test their workflows, and provide feedback via the National Library of Medicine (NLM) Help Desk.

    Expected Data Differences

    FTP users will notice some differences in data formatting and content. Most of these changes improve standardization and data richness without affecting the underlying meaning of the data. These differences include:

    Availability of Additional Data – PubMed data may be augmented with additional data from PubMed Central (PMC) when available. This additional data was already integrated on the PubMed website and API and will now be included in the FTP files. This change will ensure that all users have access to consistent, up-to-date PubMed data.

    Examples include:

    • More robust reference data supplied by PMC.
    • Inclusion of Conflict of Interest (COI) statements and additional content from PMC.
    • Inclusion of PMC Release Date even after the release date has passed.
    • will include and in addition to when available from PMC.

    Due to this augmented data, daily update files on the test site may contain additional PMIDs that are not included in the current FTP files since the current files are not updated with the PMC data.

    Formatting, Normalization, and Ordering – Some data may be formatted or ordered differently from the current FTP files. The structure of files and meaning of the data are not changed. Examples include:

    • Differing order of attributes or child elements.
    • Differences in capitalization of labels and some text.
    • Single digit dates will not be preceded by a zero (e.g., “1” instead of “01”).
    • Author identifiers will not be preceded by the URL. The ID source will remain as an attribute in .

    Schedule of Updates – Please note that daily update files for the current FTP and the test FTP site are generated using different processes on separate schedules. This means updated PMIDs may appear on different days between the two sites.

    Share Your Feedback

    Please reach out to us if you have questions or would like to provide feedback.

    For more information about accessing PubMed data via FTP, please see: Download PubMed Data.

    NLM will share additional details as the transition date approaches. Stay tuned to the NLM Technical Bulletin for updates.

  • Changes to Email Feature

    Changes to Email Feature

    PubMed Update: Changes to Email Feature. NLM Tech Bull. 2025 May-Jun;(464):e4.

    May 30, 2025 [posted]

    PubMed will soon reintroduce the ability to customize subject lines and body text when emailing search results or citations. With this update, the email feature will also require My NCBI login, and PubMed emails will only be sent to the email address associated with the My NCBI account.  

    In addition to restoring functionality to customize the subject and body text based on user feedback, these changes will strengthen the security of the email feature.

    Screenshot of the PubMed website showing a banner that will soon be added to the email feature interface notifying users of the forthcoming changes.
    Figure 1: Screenshot of the PubMed website showing a banner that will soon be added to the email feature interface notifying users of the forthcoming changes.

    The changes to email are currently expected to go into effect on or after July 28. To continue using PubMed’s email feature, please register for a free My NCBI account and see the My NCBI Help to learn more about My NCBI and the additional features it supports for NCBI databases.

    If you would like to save or share PubMed search results without logging in to My NCBI, use the Save feature to download results to your computer, copy and share a direct link to your results, or create an RSS feed.