Top 8 Most Anticipated Drug Approvals to Watch for in 2025

These include Pfizer's Xalkori for non-small cell lung cancer, Keytruda for cancer immunotherapy developed by Merck and Novo Nordisk's Ozempic, a blockbuster drug for diabetes and anti-obesity treatment. This will likely bring a surge in demand for generics as well as biosimilars, bringing avenues for drug manufacturers. New drugs, developed by some of the leading pharmaceutical companies, can transform treatment for a wide range of conditions, including rare genetic disorders, chronic pain, and cardiovascular diseases. By addressing critical unmet needs, these innovative drugs can help provide new hope for patients worldwide.

Latest News On Major Drugs to Watch for in 2025 That Could Transform Healthcare:

March 2025: After acquiring its new drug application (ANDA) from Aspen Pharma USA, Glenmark Pharmaceuticals introduced injectable acetylcysteine in the United States. SBI Securities claims that the move will benefit the Indian pharmaceutical company in the long run.

March 2025: Sun Pharmaceutical, the biggest pharmaceutical company in India in terms of revenue, announced its goal to introduce Utreglutide, an experimental medication that fights obesity and type 2 diabetes, over the next four to five years. The medication, a GLP-1 receptor agonist, has demonstrated encouraging outcomes in preliminary testing. Sun Pharma is concentrating on introducing the medication to the Indian and international markets.

March 2025: The drug regulator CDSCO has given AstraZeneca Pharma India Ltd permission to import and distribute Durvalumab solutions, a treatment for cancer, within the nation. Tremelimumab and durvalumab together are recommended for the treatment of patients with incurable hepatocellular cancer.

November 2024: AstraZeneca Pharma India Limited declared that Breztri Aerosphere would be introduced in India. Adult patients with chronic obstructive pulmonary disease (COPD) may benefit from Breztri Aerosphere as a maintenance medication to reduce symptoms and avoid flare-ups.

Let us explore some of the important drugs to watch for in 2025.

Groundbreaking Treatments for Hereditary Angioedema Coming in 2025

KalVista Pharmaceuticals' Sebetralstat is under FDA review for the on-demand treatment of hereditary angioedema. With its approval expected by June 17, 2025, the drug will be the first oral treatment for hereditary angioedema. With an estimated prevalence of 1 in 50,000, hereditary angioedema causes life-threatening attacks. It affects over 20,000 people in the United States and Europe. Sebetralstat's Phase 3 trial demonstrated rapid symptom relief, offering a promising new option for patients of all ages.

In addition, Ionis Pharmaceuticals has announced FDA acceptance of the New Drug Application for Donidalorsen, an RNA-targeted treatment aimed at preventing attacks in patients with hereditary angioedema patients. Donidalorsen, which showed promising results in Phase 3, may offer a significant reduction in attack frequency. The FDA's decision is expected by August 21, 2025.

New Treatment Possibilities for Uncomplicated UTIs with GSK’s Gepotidacin

Gepotidacin, an innovative oral antibiotic developed by GSK, is currently under priority review by the United States FDA for treating uncomplicated urinary tract infections in women and adolescents. This potential first-in-class antibiotic could address the growing challenge of drug-resistant uncomplicated UTIs, affecting over 50% of women. With the FDA's decision expected by March 26th, 2025, Gepotidacin may provide a significant advancement in UTI treatment after 20 years without a new antibiotic class.

Elamipretide to Emerge as a Promising Drug for Barth Syndrome Treatment

Stealth BioTherapeutics has received a positive vote from the FDA Advisory Committee for its potential approval of Elamipretide, intended for the treatment of Barth syndrome. It is a rare mitochondrial disease affecting approximately 1 in 1,000,000 male births. Elamipretide has shown promising results in clinical trials, offering hope for patients with this devastating, progressive disease. If approved, it will be the first-ever therapy for this life-threatening condition. 

Suzetrigine, a Non-Opioid Pain Management Breakthrough

Vertex Pharmaceuticals’ Suzetrigine has been granted a priority review by the United States Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA). Suzetrigine is a selective sodium voltage-gated channel NaV1.8 inhibitor, which plays a crucial role in delivering pain signals. This innovative drug is expected to revolutionize the management of severe, debilitating chronic pain by targeting pain pathways directly. Unlike traditional pain medications, Suzetrigine reduces chronic pain intensity while minimizing the common adverse effects. It holds the potential to significantly improve the quality of life for over 20 million Americans living with incapacitating chronic pain.

Cardamyst, a Promising Treatment for Paroxysmal Supraventricular Tachycardia Management to be Approved by March 2025

Cardamyst (etripamil) nasal spray, an investigational product by Milestone Pharmaceuticals for paroxysmal supraventricular tachycardia treatment is anticipated to be approved by the FDA by March 2025. The disease impacts approximately 0.2% of the population, with an incidence rate of 1 to 3 cases per 1,000 individuals in the United States, triggering unpredictable episodes of rapid heartbeats, severe symptoms, and anxiety. Cardamyst (etripamil) will provide a convenient and non-invasive option for self-management. The trials demonstrated exceptional efficacy, addressing the critical need for innovative therapies beyond current emergency treatments and invasive procedures.

Vutrisiran, a Revolutionary Treatment for ATTR Amyloidosis with Cardiomyopathy

Alnylam Pharmaceuticals' investigational drug, Vutrisiran, has received the United States FDA’s supplemental New Drug Application (sNDA) to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM). Set for approval by March 23, 2025, Vutrisiran, also known as Amvuttra, targets both hereditary and wild-type forms of ATTR. It significantly improved cardiovascular outcomes, survival, and quality of life in clinical trials, offering hope for patients suffering from this fatal, progressive disease.

CUTX-101, a Breakthrough Drug for Menkes Disease

CUTX-101, developed by Fortress Biotech and Cyprium Therapeutics, is currently under U.S. FDA review for treating Menkes disease, a rare pediatric condition caused by mutations in the ATP7A gene mutations. Affecting 1 in 50,000 to 1 in 360,000 births, Menkes disease often results in death during early childhood. With promising clinical results showing up to an 80% reduction in mortality, approval is expected by June 30, 2025. This could be the first FDA-approved treatment for this devastating condition.

Several promising treatments are on track for approval in 2025, offering hope for patients with rare and chronic diseases. While developing drugs for chronic illnesses like cancer, diabetes and cardiovascular conditions, there is a significant emphasis on managing rare diseases as well. Elamipretide could be the first therapy for Barth syndrome, while Cardamyst may revolutionize the treatment of paroxysmal supraventricular tachycardia treatment. The approval of such drugs indicates the dedication of leading companies and academic institutions to provide effective treatment alternatives to patients. With major FDA decisions expected in 2025, these therapies are set to transform the lives of millions.