• UCLA Health investigators’ newly published report presents a groundbreaking alternative for thyroid cancer in children, suggesting that administering targeted therapy before standard treatment may improve outcomes.
• Vertex Pharmaceuticals’ newly approved non-opioid pain medication, Journavx, offers a promising alternative for managing moderate-to-severe acute pain without the risk of addiction.
• GSK’s new Penmenvy vaccine, a 5-in-1 shot protecting ages 10 to 25 from the five most common meningococcal strains, has received FDA approval, simplifying immunization and strengthening defenses against this disease.

Recent News

• Genetic therapy gives infants life-changing improvements in sight
  Medical Xpress – February 20, 2025
  Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic medicine through UCL Institute of Ophthalmology and Moorfields Eye Hospital, with the support of MeiraGTx. The children were born with a severe impairment to their sight due to a rare genetic deficiency that affects the AIPL1 gene. The condition, a form of retinal dystrophy, means those affected are born with only sufficient sight to distinguish between light and darkness. The gene defect causes the retinal cells to malfunction and die, with children affected being legally certified as blind from birth.
• FDA signs off on Sanofi’s insulin biosimilar, the first for Novo Nordisk’s rapid-acting NovoLog
  Fierce Pharma – February 18, 2025
  More than three years after approving two biosimilars referencing Sanofi’s long-acting insulin Lantus, the FDA has signed off on a rapid-acting insulin biosimilar for the first time. The agency has given a thumbs up to Sanofi’s Merilog (insulin-aspart-szjj) as the first biosimilar to Novo Nordisk’s NovoLog for patients with diabetes. Insulin aspart is delivered by a subcutaneous shot 5 to 10 minutes before mealtime to help patients control glucose and limit blood sugar spikes. Merilog will be provided by prefilled pen in a 3 mL dose or in a multiple-dose 10 mL vial. It is for adults and pediatric patients aged 6 and older.
• Researchers identify DNA changes, biological pathways associated with inherited cancer risk
  Stanford Medicine – February 17, 2025
  Thousands of single changes in the nucleotides that make up the human genome have been associated with an increased risk of developing cancer. But until now, it’s not been clear which are directly responsible for the uncontrolled cellular growth that is the hallmark of the disease and which are simply coincidences or minor players. Stanford researchers have conducted the first large-scale screen of these inherited changes, called single nucleotide variants, and homed in on fewer than 400 that are essential to initiate and drive cancer growth.
• US FDA approves GSK’s combo meningococcal vaccine
  Reuters – February 14, 2025
  The U.S. Food and Drug Administration approved British drugmaker GSK’s, combination vaccine to protect against meningococcal infection for use in people aged 10 through 25 years. Meningococcal infections, caused by bacteria called Neisseria meningitidis, can lead to severe, sometimes deadly, bloodstream infections as well as severe swelling in the brain and spinal cord. The vaccine, branded as Penmenvy, combines the immune response-generating components of two of the company’s approved vaccines, Bexsero and Menveo, to protect against the five most common strains of meningococcal bacteria, GSK said in a statement.
• AnaptysBio’s PD-1 drug reduces rheumatoid arthritis in phase 2 trial
  Fierce Biotech – February 12, 2025
  AnaptysBio may have ended last year on a low, but 2025 is looking up with the news that its PD-1 agonist rosnilimab has hit the goals of a phase 2 rheumatoid arthritis (RA) trial. The phase 2b RENOIR study assessed three doses of subcutaneous rosnilimab—100 mg every four weeks, 400 mg every four weeks or 600 mg every two weeks—versus placebo in 424 patients with moderate-to-severe RA who were also receiving standard antirheumatic drugs such as methotrexate. All three doses hit the trial’s primary endpoint of demonstrating a statistically significant change from baseline in disease activity after 12 weeks, the company said in a release.
• AbbVie Gets FDA Approval for New Antibiotic in the Face of Rising Resistance
  BioSpace – February 10, 2025
  Emblaveo, a new combination antibiotic developed in a partnership between AbbVie and Pfizer, received FDA approval to address difficult-to-treat intra-abdominal bacterial infections, AbbVie announced. Embalveo’s development partnership was originally between Pfizer and Allergan Inc., the latter of which was acquired by AbbVie in June 2019 for a whopping $63 billion. Under the terms of the partnership, AbbVie holds Emblaveo’s rights in the U.S. and Canada, while Pfizer has the rights for the drug in the rest of the world. Antimicrobial resistance (AMR) is a leading global public health threat, according to the World Health Organization (WHO), with nearly 5 million associated deaths per year—and that number is set to double by 2050 if more treatment options are not able to address the problem.
• Case report highlights promising approach for treating pediatric thyroid cancer
  UCLA Health – February 7, 2025
  A case report led by UCLA Health investigators suggests children with advanced papillary thyroid cancer driven by RET gene mutations may benefit from incorporating targeted therapy before starting standard treatment. The study highlights the use of selpercatinib, a type of tyrosine kinase inhibitor, prior to radioactive iodine therapy, the cornerstone treatment for thyroid cancer. Traditionally, agents like selpercatinib have been reserved as a last resort when other treatments fail. The case focused on a 10-year-old girl with metastatic papillary thyroid cancer caused by a RET gene mutation. Her disease had spread extensively to her lymph nodes, neck muscles and lungs.
• Cancer vaccine prevents recurrence of advanced kidney cancer in small trial
  Fierce Biotech – February 6, 2025
  After their advanced kidney tumors were surgically removed, nine patients were protected from the cancer returning thanks to an experimental, personalized cancer vaccine, researchers reported. The small, phase 1 trial was carried out by the Dana-Farber Cancer Institute, with the results published in Nature. Even more than three years after surgery, none of the patients saw their tumors recur. The vaccines boosted cancer-targeting T cells 166-fold, the scientists wrote, and these immune cells persisted in the body throughout the study’s duration.
• AI Accelerates the Search for New Tuberculosis Drug Targets
  UC San Diego – February 6, 2025
  Tuberculosis is a serious global health threat that infected more than 10 million people in 2022. Spread through the air and into the lungs, the pathogen that causes “TB” can lead to chronic cough, chest pains, fatigue, fever and weight loss. While infections are more extensive in other parts of the world, a serious tuberculosis outbreak currently unfolding in Kansas has led to two deaths and has become one of the largest on record in the United States. While tuberculosis is typically treated with antibiotics, the rise of drug-resistant strains has led to an urgent need for new drug candidates.
• Pfizer Sees Path to Braftovi’s Full Approval With Phase III CRC Data
  BioSpace – February 3, 2025
  Pfizer released topline data from the Phase III BREAKWATER study, demonstrating that a combination regimen featuring Braftovi significantly improves survival in certain colorectal cancer patients. The pharma did not provide specific data in its press release, only revealing that patients treated with the Braftovi combo saw a “statistically significant and clinically meaningful improvement” in progression-free survival (PFS), one of the two primary endpoints of BREAKWATER. Overall survival, a key secondary outcome, was also significantly and clinically better in the Braftovi arm.
• D.A. Approves Drug to Treat Pain Without Opioid Effects
  The New York Times – January 30, 2025
  The Food and Drug Administration approved a new medication to treat pain from an injury or surgery. It is expensive, with a list price of $15.50 per pill. But unlike opioid pain medicines, it cannot become addictive. That is because the drug, suzetrigine, made by Vertex Pharmaceuticals and to be sold as Journavx, works only on nerves outside the brain, blocking pain signals. It cannot get into the brain. Researchers say they expect it to be the first of a new generation of more powerful nonaddictive drugs to relieve pain. To test the drug, Vertex, which is based in Boston, conducted two large clinical trials, each with approximately 1,000 patients who had pain from surgery.