Most Anticipated New Drug Launches of 2025
The pharmaceutical industry is set for another groundbreaking year in 2025, with several highly anticipated drug launches poised to reshape treatment landscapes across various therapeutic areas. As biotech and pharma companies continue to push the boundaries of innovation, next year’s approvals could bring transformative therapies for conditions ranging from rare diseases to widespread chronic illnesses.
According to the latest Evaluate Vantage 2025 report, a number of potential blockbuster drugs are on the horizon, each with the potential to generate significant revenue while addressing critical unmet medical needs. From novel biologics to first-in-class small molecules, these upcoming therapies could redefine standards of care and open new doors for patients worldwide.
Vanza Triple
Company | Vertex |
Indications | Cystic fibrosis |
One of the most highly anticipated approvals of 2025 is vanzacaftor/tezacaftor/deutivacaftor (vanza triple)—a therapy for cystic fibrosis (CF). With a target FDA approval in Q1 2025, this once-daily treatment could offer a new standard of care for CF patients aged 6 and older with at least one F508del or another responsive CFTR mutation.
Cystic fibrosis is a genetic disorder caused by mutations in the CFTR gene, leading to faulty or insufficient CFTR protein channels. This disrupts the movement of salt and water across cell membranes, causing thick mucus buildup in the lungs and other organs.
The vanza triple addresses this issue by improving both CFTR protein levels and function: Vanzacaftor and tezacaftor help transport more functional CFTR protein to the cell surface. Deutivacaftor increases the protein’s activity, improving salt and water balance in the body. By targeting the root cause of CF, this therapy has the potential to significantly reduce disease progression and improve quality of life.
Recognizing its potential, the FDA granted Fast Track and Orphan Drug Designations to vanza triple, expediting the review process. Vertex Pharmaceuticals also leveraged a priority review voucher, cutting the FDA’s decision time from 10 months to 6 months.
Beyond the U.S., the treatment is also under review in Canada, Australia, Switzerland, the U.K., and the European Union. The European Medicines Agency (EMA) has already validated its application, paving the way for a global rollout in 2025.
A major factor that could help with the launch of Vanza Triple is its reduced royalty obligation, making it a more cost-effective option compared to previous CF treatments from Vertex. This could help increase access to life-changing therapy for more patients.
Datopotamab deruxtecan
Company | AstraZeneca and Daiichi Sankyo |
Indications | Locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) |
AstraZeneca and Daiichi Sankyo have received acceptance from the U.S. Food and Drug Administration (FDA) for their Biologics License Application (BLA) submission for datopotamab deruxtecan (Dato-DXd). The application seeks approval for the treatment of adult patients with locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) who have previously undergone systemic therapy.
The application is supported by findings from the TROPION-Lung01 Phase III clinical trial. In this study, datopotamab deruxtecan demonstrated a statistically significant advantage in progression-free survival (PFS), one of the trial's dual primary endpoints, compared to docetaxel, the current standard treatment for this patient group.
Regarding overall survival (OS), the second primary endpoint, preliminary data suggested a numerical benefit in favor of datopotamab deruxtecan over docetaxel; however, statistical significance had not been reached at the time of the data analysis.
In patients with nonsquamous NSCLC, the drug exhibited a notable PFS advantage and a positive trend in OS outcomes. The study remains ongoing, with final OS data to be assessed later.Datopotamab deruxtecan is an antibody-drug conjugate (ADC) designed to target TROP2 and incorporates DXd technology. It is being co-developed by AstraZeneca and Daiichi Sankyo.
Suzetrigine
Company | Vertex |
Indications | Moderate-to-severe acute pain |
A newly developed oral medication designed to manage moderate-to-severe acute pain in adults has gained approval from the U.S. regulatory agency overseeing food and drug safety. This non-opioid treatment works by selectively inhibiting the NaV1.8 sodium channel, a key component in transmitting pain signals. The medication is both effective and well-tolerated, with no signs of addiction risk, and is suitable for various types of acute pain.
Suzetrigine represents a novel category of pain management therapies. As an orally administered, non-opioid compound, it specifically targets NaV1.8 while avoiding other sodium channels. This particular channel is found in peripheral nerve cells responsible for detecting pain and plays a crucial role in transmitting pain signals. Because this treatment blocks pain signals at their source rather than within the brain, it offers relief without the drawbacks of existing options, such as the dependency concerns associated with opioids.
The newly authorized pain reliever is intended for adult patients and is to be taken twice daily. The manufacturer has set a baseline cost of $15.50 per 50mg dose in the U.S. market.
Further studies are underway to assess the drug’s potential in addressing peripheral neuropathic pain. Clinical trials are currently in progress for individuals experiencing painful diabetic nerve damage, and pending regulatory discussions, research may expand to include those suffering from lower back nerve-related pain.
Aficamten
Company | Cytokinetics |
Indications | Obstructive hypertrophic cardiomyopathy |
This experimental oral medication is a small-molecule inhibitor designed to regulate cardiac myosin activity. Its primary function is to decrease excessive muscle contraction linked to hypertrophic cardiomyopathy (HCM) by limiting myosin’s ability to generate force, thereby reducing the intensity of contractions. Studies conducted in laboratory models suggest that this treatment may help reverse or diminish thickening and rigidity of the heart muscle.
A large-scale Phase 3 study, aimed at assessing its safety and effectiveness in individuals with symptomatic obstructive HCM, has been completed. The drug is also being investigated in additional late-stage trials. One ongoing study compares its use as a standalone treatment against metoprolol in patients experiencing left ventricular outflow tract obstruction. Another trial focuses on its impact in those with non-obstructive HCM by comparing it to a placebo. Additionally, a separate clinical evaluation is exploring its potential benefits for pediatric patients diagnosed with symptomatic obstructive HCM.
Brensocatib
Company | Insmed |
Indications | Bronchiectasis and chronic rhinosinusitis without nasal polyps |
Brensocatib is an investigational oral therapy and a reversible inhibitor of dipeptidyl peptidase 1 (DPP1), developed as a potential treatment for neutrophil-driven conditions such as bronchiectasis and chronic rhinosinusitis without nasal polyps. It remains under study and has not yet received regulatory approval in any country.
A regulatory agency in the United States has granted an expedited review process for this therapy as a potential treatment for bronchiectasis, setting a target decision date of August 12, 2025.
The approval application is supported by findings from a pivotal late-stage clinical trial, which successfully met its primary goal. Both tested dosage levels demonstrated statistically and clinically meaningful reductions in the annual rate of lung flare-ups compared to a placebo over the course of a year. Additionally, both dosage levels showed significant benefits on pre-specified secondary measures, such as delaying the onset of the first exacerbation and increasing the likelihood of remaining free from flare-ups during treatment. The 25 mg dose was also linked to significantly slower lung function decline after 52 weeks, as assessed by post-bronchodilator forced expiratory volume in one second.
Tolebrutinib
Company | Sanofi |
Indications | Non-relapsing secondary progressive multiple sclerosis |
Tolebrutinib is designed to cross the blood-brain barrier and actively inhibit Bruton’s tyrosine kinase (BTK). It reaches levels in cerebrospinal fluid that are expected to influence B cells and microglia linked to disease activity.
Currently, it is undergoing late-stage clinical trials to assess its potential in treating different types of multiple sclerosis. Its effectiveness and safety have not yet been reviewed or approved by any regulatory agency worldwide.
The U.S. regulatory agency responsible for drug approvals has granted an expedited development pathway to an investigational therapy for adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS). This decision is based on encouraging findings from a late-stage clinical trial, which showed that the treatment reduced the risk of confirmed disability progression over six months by 31% compared to a placebo. Additional analysis of secondary measures indicated that the proportion of patients experiencing confirmed improvement in disability was nearly twice as high with the investigational drug (10%) compared to placebo (5%).
This regulatory designation is intended to accelerate the development and review of therapies for serious or life-threatening illnesses. To qualify, a drug must show early clinical evidence suggesting it may offer significant benefits over currently available treatments based on clinically meaningful outcomes.
Mazdutide
Company | Eli Lilly |
Indications | Obesity |
Mazdutide, also referred to as IBI362 or LY3305677, is a synthetic version of mammalian oxyntomodulin that includes a fatty acid side chain. It is currently being developed as a dual agonist targeting both the GLP-1 and glucagon receptors for once-weekly administration, aimed at managing obesity and type 2 diabetes.
Clinical trial results demonstrated that at both the 32-week and 48-week marks, individuals receiving mazdutide at doses of 4 mg and 6 mg exhibited significantly greater reductions in body weight compared to those given a placebo. The percentage of participants who achieved weight loss of at least 5%, 10%, and 15% was also notably higher in the treatment groups (P<0.001). By week 48, the difference in average body weight reduction between the placebo group and those receiving 6 mg of mazdutide was -14.31% based on the treatment-policy estimand and -14.37% according to the efficacy estimand.
Depemokinab
Company | GSK |
Indications | Asthma |
Depemokimab is an advanced biologic therapy designed for long-lasting effects, offering a significant improvement in the treatment of asthma.
This ultra-long-acting drug works by binding with high affinity to interleukin-5, a key cytokine involved in eosinophilic inflammation. Its unique mechanism of action allows for extended dosing intervals, with administration required only once every six months. This could provide a major convenience for patients, reducing the frequency of injections compared to current treatments while maintaining effective symptom control.
Recent Phase III clinical trial data presented at the European Respiratory Society highlighted depemokimab’s impressive efficacy in reducing asthma exacerbations. Patients receiving the treatment experienced more than a 50% reduction in severe asthma attacks, alongside a 72% decrease in episodes requiring hospitalization or emergency care. These promising results suggest that depemokimab could become a game-changing therapy in asthma management.
Nipocalimab
Company | J&J |
Indications | Generalized myasthenia gravis, chronic inflammatory demyelinating polyneuropathy, warm autoimmune hemolytic anemia, and idiopathic inflammatory myopathies. |
Nipocalimab is an experimental monoclonal antibody engineered for high affinity and lacks glycosylation and effector functions. It is designed to selectively inhibit the neonatal Fc receptor (FcRn), thereby reducing circulating immunoglobulin G (IgG) antibodies, including those responsible for autoimmune and alloimmune disorders. By blocking FcRn, this therapy aims to lower harmful antibody levels while preserving overall immune function without inducing widespread immunosuppression.
This investigational treatment is currently being evaluated across three major categories of autoantibody-related diseases. These include rare autoimmune disorders such as generalized myasthenia gravis, chronic inflammatory demyelinating polyneuropathy, warm autoimmune hemolytic anemia, and idiopathic inflammatory myopathies. It is also being studied for maternal-fetal conditions caused by maternal alloantibodies, including hemolytic disease of the fetus and newborn (HDFN).
Additionally, it is being explored for widespread rheumatologic conditions like rheumatoid arthritis, Sjögren’s disease, and systemic lupus erythematosus. Another key aspect of FcRn inhibition is its potential to block the transfer of maternal alloantibodies to the fetus by preventing IgG binding in the placenta, which could help mitigate complications arising from maternal-fetal antibody interactions.
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