10 Remarkable Breakthroughs in Medicine and Health in 2025

From gene therapies and the development of next-generation drugs to devices and transplants that highlight fundamental changes in personalized and effective treatment, several discoveries in 2025 have given hope for improving quality of life. These breakthroughs matter because they have transformed the means of healthcare delivery, making it more accessible and efficient for mankind. They have shifted the focus from managing symptoms to prevention and cure, potentially saving millions of lives by tackling some of the deadliest diseases effectively.

Here are 10 medical breakthroughs of 2025:

1. Rare genetic disorder treated using CRISPR-based gene therapy

After birth, Baby KJ was diagnosed with a rare, fatal disorder called severe carbamoyl-phosphate synthetase 1 deficiency (CPS1), which affects one in a million babies. The ammonia levels in the blood increase and lead to vomiting, hypothermia, brain swelling, and coma. Scientists used modified CRISPR treatment to repair faulty genes in KJ’s liver. The experimental procedure was successful and enhanced baby KJ’s life expectancy. KJ became the world’s first patient to receive customized gene-editing therapy, developed by the Children’s Hospital of Philadelphia and Penn Medicine.

2. Human embryo implantation in 3D

Researchers have captured real-time 3D images of human embryo implantation into an artificial uterus for the first time. The uterine matrix was created using a collagen-based gel, rich in uterine tissue and proteins required for embryo development. The experiment was conducted on both human and mouse embryos to study the implantation process. Mouse embryos adhered to the surface, whereas human embryos penetrated deeply into the uterine tissue for implantation.

3. Male contraceptive pill YCT-529 passes safety trials

Researchers have long explored the possibility of a safe and reversible male oral contraceptive. They have tested a novel nonhormonal oral contraceptive pill for males, the first of its kind to pass early-phase clinical safety trials in humans in 2025. The daily pill, YCT-529, blocks a vitamin A metabolite from binding to receptors in the testes, which stops sperm production. The effects of the pill are reversible. However, further efficacy studies and larger trials may perhaps open doors for approval.

4. US FDA clears blood tests to help diagnose Alzheimer’s disease

In May 2025, the US FDA approved the first blood test to help diagnose Alzheimer’s disease. This new blood test (Lumipulse G pTau217/ß-Amyloid 1-42 Plasma Ratio) measures the ratio of two proteins in the human plasma - pTau217 and β-amyloid 1-42. The ratio is then linked to the presence or absence of amyloid plaques in the brain and helps in the diagnosis of Alzheimer’s disease. The test is rapid, highly accurate, and less expensive and invasive compared to the previously available tests. The test can be made highly accessible as primary care providers can now start the diagnostic process even at centers where healthcare providers are scarce. In October 2025, the US FDA approved a second blood test, Elecsys pTau181 plasma test, developed by Roche, that helps rule out the presence of amyloid plaque.

5. Gene therapy for Huntington’s disease shows promise

Huntington’s disease is a genetic neurodegenerative disorder where the brain cells are destroyed persistently, eventually leading to paralysis, dementia, or even death. This condition is caused by a single inherited gene mutation, which causes misfolded proteins to gather in brain cells. But a new gene treatment to disable the toxic protein, injected into the most affected part of the brain, has proven to slow down the progression of the disease. This paves the way for the first-ever treatment for this rare inherited brain degeneration.

6. Vaccines for dementia show potential

Large-scale studies in 2025 from the University of Oxford, NIHR, Stanford, and others suggest some vaccines, such as those for shingles, respiratory syncytial virus (RSV), influenza, pneumococcus, and Tetanus-Diphtheria-Pertussis (Tdap or DPT), can lower the risk for developing dementia. Researchers emphasize that more controlled trials are required to confirm the efficacy of these vaccines.

7. The first successful bladder transplant in a human

In a groundbreaking achievement, a 41-year-old became the first human to receive a urinary bladder transplant. After a tumor removal, his bladder was too small and functionally compromised, and both his kidneys had to be removed due to renal cancer. A donor’s kidney and bladder were transplanted. Postoperative evaluation confirmed adequate urine drainage of the transplanted bladder. Bladder transplantation involves joining a complex network of blood vessels and nerves. The surgery was done to test the viability of bladder transplants in people suffering from terminal kidney and bladder-related diseases. This surgery marks a major milestone in urology.

8. Gene-edited pig liver transplant functions inside a human for 10 days

In 2025, the world witnessed the first-ever xenotransplantation into a brain-dead human. The modified pig liver functioned for 10 days without rejection, which opened doors for a new era of organ transplantation. This achievement also brings hope for solving the organ-shortage crisis. Researchers used a Bama miniature pig liver with six-gene edits, with the recipient’s left liver intact, and simultaneously monitored the vitals, like bile production, for 10 days. The result showed potential compatibility of animal organs as a “bridge” or support organ for patients awaiting human donors.

 9. New advancements in mRNA technology

mRNA vaccines became well-known during the COVID-19 pandemic. These are now being tested in hundreds of clinical trials for conditions such as the flu, HIV, genetic disorders, and cancer. mRNA vaccines use a synthetic immunization strategy using genetic codes rather than live-attenuated or inactive viruses to trigger an immune response.  The main advantages include rapid design, no need for cell cultures, high immunogenicity, strong safety profiles, and adaptability to various pathogens. Early results from several studies are encouraging. A recent study confirmed that mRNA-based HIV vaccines can trigger the production of neutralizing antibodies, which are essential for protecting cells from infection. While these innovations suggest that mRNA technology could be a powerful weapon against various diseases, larger-scale clinical trials are still necessary to determine its long-term effectiveness and safety.

10. Smallest artificial heart implanted in a 7-year-old

In a breakthrough for pediatric medicine, Chinese surgeons successfully implanted the world’s smallest artificial heart into a 7-year-old boy suffering from end-stage heart failure.  Donor hearts for children are extremely rare, and adult-sized implants are often too large to be safe. This miniature device, weighing only 45 grams, fills a critical gap in care. The 2.9-centimeter device uses magnetic equipment to help the heart pump blood. The boy showed immediate signs of improvement after the surgery, proving that these tiny, high-tech devices can provide a vital lifeline for children who have run out of other options.

Read more here:

https://www.forbes.com/sites/juergeneckhardt/2025/12/18/five-medical-breakthroughs-of-2025-cancer-heart-and-genetic-diseases/

https://www.fda.gov/news-events/press-announcements/fda-clears-first-blood-test-used-diagnosing-alzheimers-disease

https://www.alz.org/news/2025/fda-clearance-blood-test-primary-care-rule-out-alzheimers-related-amyloid-pathology

https://www.scientificamerican.com/article/10-transformational-health-discoveries-of-2025/

https://www.sciencenews.org/article/medical-miracle-breakthroughs-hope-2025

https://qazinform.com/news/world-medical-breakthroughs-in-2025-a-year-in-review-d1f974.

https://www.popsci.com/health/health-breakthroughs-2025/

https://www.euronews.com/health/2025/12/25/health-in-2025-the-5-medical-breakthroughs-that-gave-us-hope

Novel drug therapies approved in 2025

As the year comes to an end, let us take a look at some of the new drugs approved in 2025 for the United States (US) and Indian markets.

Image credit: stockmedia.cc

1. The US – new drugs approved by the US Food and Drug Administration (FDA)

‘Novel’ drugs are drugs that have never been approved for the US market before. As of the 24^th of December, this year, 44 novel drugs have been approved for the US market, spanning multiple therapeutic areas including oncology, cardiology, immunology, infectious disease, and rare diseases.

Here is a selective list of new drugs approved by the US FDA.

Drug Name (Active Ingredient)	Type	US FDA-approved uses	Mechanism
Journavx (suzetrigine)	Non-opioid analgesic	Moderate to severe acute pain in adults	Blocks pain signals (sodium channels) in the peripheral nervous system  before they reach the brain
Blujepa (gepotidacin)	Triazaacenaphthylene antibiotic	Uncomplicated urinary tract infections in female patients ≥12 y of age weighing ≥40 kg	Targets & blocks bacterial enzymes essential for DNA replication, which kills the bacteria
Qfitlia (fitusiran)	Small interfering RNA (siRNA) therapy	Prophylactic care for hemophilia A & hemophilia B in patients ≥12y	Reduces the production of antithrombin, a protein that inhibits blood clotting, increasing thrombin
Keytruda Qlex (pembrolizumab & perahyaluronidase alfa-pmph)	PD-1 inhibitor and monoclonal antibody	Adult & pediatric (≥12y) solid tumors	Activates the immune system, allowing T-cells to detect and attack cancer cells
Cardamyst (etripamil)	Self-administered nasal spray	Paroxysmal supraventricular tachycardia	Blocks L-type calcium channels in the heart, which interrupts abnormal heart rhythm and restores normal heartbeat
Voyxact (sibeprenlimab-szsi)	Monoclonal antibody	Adults with primary immunoglobulin A nephropathy (IgAN) to reduce proteinuria and kidney damage	Binds to A Proliferation-Inducing Ligand (APRIL) & blocks signaling receptors, to reduce levels of pathogenic immunoglobulin A1 (IgA1)
Redemplo (plozasiran)	Small interfering RNA (siRNA) therapy	Reduce triglycerides in adults with chylomicronemia syndrome	Targets & degrades the apoC-III mRNA by RNA interference mechanism, resulting in reduced levels of hepatic & serum apoC-III protein.
Brinsupri (brensocatib)	DPP1 inhibitor	Non-cystic fibrosis bronchiectasis in  patients ≥12y	Blocks DPP1, which prevents activation of pro-inflammatory enzymes in neutrophils. This slows down the progression of lung infection.
Dawnzera (donidalorsen)	RNA-targeted therapy (prekallikrein-directed antisense oligonucleotide)	To avert attacks of hereditary angioedema	Targets and reduces the production of prekallikrein protein in the liver that prevents excessive formation of bradykinin
Komzifti (ziftomenib)	Menin inhibitor	Relapsed or refractory acute myeloid leukemia with susceptible nucleophosmin 1 mutation in adults	Blocks the interaction of menin and lysin [K]-specific methyltransferase 2A

 

India – New drugs approved by the Central Drugs Standard Control Organization (CDSCO)

Here are some new drugs that have been approved by the CDSCO, India, in 2025 :

Drug Name	Type	CDSCO-approved uses	Mechanism
Letermovir	Antiviral (Quinazoline Derivative)	Prevention of cytomegalovirus infection and disease in adult seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT) or adult recipients of kidney transplant	Disrupts the process of cleaving and packaging viral DNA into mature virus particles.
Edoxaban	Oral anticoagulant (DOAC)	· Prevention of stroke & systemic embolism in adult patients with nonvalvular atrial fibrillation with ≥1 risk factor, such as congestive heart failure, hypertension, age ≥ 75y, diabetes mellitus, prior stroke or transient ischemic attack(TIA). · Treatment of deep vein thrombosis (DVT) & pulmonary embolism (PE), and · Prevention of recurrent DVT & PE in adults	Blocks clotting protein Factor Xa in blood, to reduce the risk of clot formation.
Sodium zirconium cyclosilicate powder for oral suspension 5g/10g	Inorganic, selective monovalent cation exchanger	Hyperkalemia in adults	Binds potassium ions in the gastrointestinal tract, which are removed from the body in stool
Rimegepant	Calcitonin gene-related peptide (CGRP) receptor antagonist	Acute & episodic migraine with/without aura in adults with a previous insufficient response to triptans	Blocks the CGRP receptor to stop the release of pain-inducing signals and vasodilation associated with migraines
Doravirine	Antiretroviral [non-nucleoside reverse transcriptase inhibitor (NNRTI)]	In combination with other antiretroviral agents to treat HIV-1 infection in treatment-naïve patients ≥35 kg	Blocks activity of HIV-1 reverse transcriptase (RT) enzyme to stop replication of the virus
Tucatinib	Tyrosine kinase inhibitor (TKI)	In combination with trastuzumab &  capecitabine, for adults with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received ≥1 prior anti-HER2-based regimens in the metastatic setting	Blocks signals from the HER2-positive protein inside cancer cells and stops tumor growth
Zanubrutinib	Bruton's tyrosine kinase (BTK) inhibitor	Adults with different types of B-cell malignancies	Irreversibly inhibits the BTK enzyme in the B-cell receptor signaling, which halts B-cell proliferation and leads to tumor cell death
Linaclotide	Synthetic 14-amino acid peptide	Chronic idiopathic constipation (CIC) in adults	Binds to GC-C receptors on intestinal epithelial cells, which promotes the secretion of chloride and bicarbonate ions and inhibits sodium absorption. This leads to increased fluid and relieves constipation.
Ivosidenib	Small-molecule inhibitor and an enzyme inhibitor	Newly diagnosed acute myelogenous leukemia & advanced or metastatic cholangiocarcinoma with isocitrate dehydrogenase-1 (IDH1) R132 mutation in adults	Inhibits mutant IDH-1, which reduces cancer-promoting 2-HG and restores normal cell differentiation
Datopotamab deruxtecan	Antibody-drug conjugate (ADC)	Unresectable or metastatic HR+ve, HER2-ve breast cancer cases who have received prior endocrine-based therapy and chemotherapy	Delivers topoisomerase I inhibitor directly to cancer cells, causing DNA damage, blocking replication and leading to apoptosis
Fexuprazan hydrochloride	Potassium-competitive acid blocker (P-CAB)	Erosive esophagitis	Reversibly blocks the H+/K+ ATPase enzyme (proton pump) in parietal cells of the stomach

 

Take a moment to thank all the participants in clinical trials, who made it possible for newer drugs to be available this year...the regulators, companies that sponsored these trials, the ethics committees, the investigators and site staff, and overall the participants/patients who agreed to have a new drug tested on them.