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.

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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	To treat 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	To treat uncomplicated urinary tract infections in female adult & pediatric patients ≥12 y of age weighing ≥40 kg	Targets and 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 adults and children ≥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	To treat adult & pediatric (≥12y) solid tumors	Activates the immune system, allowing T-cells to detect and attack cancer cells
Cardamyst (etripamil)	Self-administered nasal spray	To treat 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	To treat adults having 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	Used to 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	Treatment of non-cystic fibrosis bronchiectasis in adults and pediatric 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	Treatment of 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)	Used for prophylaxis 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	Treatment of 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	Treatment of 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)]	Used in combination with other antiretroviral agents to treat HIV-1 infection in treatment-naïve adults & children ≥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 treatment of 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	Used to treat 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	Used to treat 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	Treatment of 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)	Used in the treatment of 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)	Used to heal 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.