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Category: Pharma & Biotechnology

  • 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
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    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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  • 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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  • Trump administration proposes cutting FDA budget by 5.5%

    Trump administration proposes cutting FDA budget by 5.5%

    • Updated On May 25, 2025 at 07:19 PM IST
    • Read by 100 Professionals

    By Ahmed Aboulenein and Mariam Sunny

    Washington: The Trump administration is proposing a $6.8 billion budget request for the U.S. Food and Drug Administration for the 2026 fiscal year, its Commissioner Martin Makary said on Thursday, a cut of around 5.5% from its $7.2 billion budget this year.

    The White House had on May 2 proposed reducing U.S. health spending by more than a quarter next year, with the National Institutes of Health and Centers for Disease Control and Prevention facing the brunt of billions of dollars in cuts.

    The proposed budget requested $93.8 billion for the Department of Health and Human Services – a cut of $33.3 billion, or 26.2% – from this year’s budget of $127 billion, but did not mention the FDA. Makary’s comments on Thursday were the first official outlining of the agency’s 2026 budget request.

    “The Trump administration is proposing a $6.8 billion budget for the FDA,” Makary told the Senate Appropriations Committee’s Subcommittee on Labor, Health and Human Services, Education, and Related Agencies at a hearing on the budget.

    The FDA in 2025 had a budget of $7.2 billion, including $3.5 billion in user fees, payments made by pharmaceutical and medical device manufacturers to fund the staff resources needed to review their products quickly, conduct inspections, and ensure the safety of clinical trials.

    Makary said that nearly 1,900 people were laid off at the FDA as part of a massive restructuring of federal health agencies under Secretary of Health and Human Services Robert F. Kennedy Jr. to align with President Donald Trump’s goal of dramatically shrinking the federal government.

    No scientific reviewer or inspector was cut as a part of the reduction in force, he added. Democratic senators pushed back, asking Makary about media reports of the FDA firing and later rehiring scientists. He said the scientists were not fired but rather scheduled for firing before the decision was reversed.

    A further 1,200 took early retirement packages and the agency is hiring more scientists to replace them, Makary said.

    The staffing changes have not impacted approval schedules and the agency is on track to meet its treatment review targets under the Prescription Drug User Fee Act, known as PDUFA, he said. (Reporting by Ahmed Aboulenein in Washington and Mariam Sunny in Bangalore; Editing by Mark Porter, Conor Humphries and Aurora Ellis)

    • Published On May 25, 2025 at 06:11 PM IST

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  • Vertex misses quarterly results on weaker-than-expected demand for cystic fibrosis drug

    Vertex misses quarterly results on weaker-than-expected demand for cystic fibrosis drug

    • Updated On May 6, 2025 at 11:55 AM IST
    • Read by 100 Professionals

    Bengaluru:: Vertex Pharmaceuticals missed Wall Street estimates for quarterly results on Monday, due to lower-than-expected sales of its cystic fibrosis (CF) drug Trikafta.

    However, the drugmaker raised the lower end of its revenue forecast as it looks to its acute pain drug, Journavx, and newer CF drugs to drive growth beyond its established line of treatments.

    In December, the U.S. Food and Drug Administration approved Vertex’s next-generation treatment, Alyftrek. This once-daily treatment for a rare and progressive genetic disease further strengthens Vertex’s market dominance in CF treatments.

    CF is an inherited disorder resulting from the absence of a specific protein, which disrupts the movement of salt and water in and out of cells in various organs.

    In the first quarter ended March 31, sales of the company’s older CF treatment, Trikafta, rose 2% to $2.53 billion but missed analysts’ average expectation of $2.58 billion, according to LSEG data.

    Vertex has activated more than 65 authorized treatment centers globally and 90 patients have begun cell collection for its gene therapy Casgevy, which treats a rare blood disorder that requires regular blood transfusions.

    Vertex, sees 2025 revenue between $11.85 billion and $12 billion, compared to the previously projected range of $11.75 billion to $12 billion.

    Total quarterly revenue rose 2.7% to $2.77 billion, missing analysts’ estimates of $2.85 billion.

    On an adjusted basis, the company reported a profit of $4.06 per share for the quarter, compared with analysts’ expectations of a profit of $4.32.

    (Reporting by Sriparna Roy and Christy Santhosh in Bengaluru; Editing by Mohammed Safi Shamsi)

    • Published On May 6, 2025 at 11:52 AM IST

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  • Pharma companies must file compliance strategy

    Pharma companies must file compliance strategy

    Teena Thacker

    New Delhi: Drugmakers that want to extend the timeline for implementation of Schedule M must carry out a gap analysis and file an application with the drug regulator, detailing their strategy to comply with the revised manufacturing standards.

    Small and medium manufacturers with turnover of less than ₹250 crore may seek extension of the timeline by submitting a plan of upgradation within three months from now, the drug regulatory authority said in a notification issued on Tuesday.

    “These companies will have to comply with the revised Schedule M requirements as per the plan submitted by them,” a senior official told ET.

    As per the notification, a company’s director, proprietor or partner will have to submit a gap analysis and give an undertaking to initiate upgradation within three months from the date of the application. The submission should also justify the time period they seek to comply with the requirements.

    As reported by ET, Tuesday’s notification gave small drug companies time until December 31 this year to adhere to the revised Schedule M. The application should be submitted to the Central Licence Approving Authority online, it said.

    The drug rules will be amended to provide the extension of time for another year.

    The revised Schedule M-which outlines tighter quality norms for manufacturing-was notified in January last year. While units with annual turnover of more than ₹250 crore had to comply from July 1, 2023, the implementation date for MSMEs was January 1 this year.

    However, MSMEs sought more time to make the necessary changes in their manufacturing processes.

    The ministry then issued a draft notification, proposing to extend the deadline for the MSMEs. In this regard, it had invited public comments by January 11.

    “The feedback was compiled, and the final draft was sent to the legal affairs department for vetting,” a person cited above told ET.

    The country’s pharma lobby groups had been seeking a two-year extension to the deadline, saying much time is needed for improving infrastructure, increasing manpower and training.

    Federation of Pharma Entrepreneurs (FOPE) wrote to the drug controller, seeking an extension of the notification implementation.

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