From cancer vaccines to COPD treatments, these 10 companies are developing innovative medicines

In 2024, drug makers delivered breakthrough treatments in conditions that had seen little progress for decades—and helped make existing therapies more accessible. Approved in July, Eli Lilly’s Kisunla is the first limited-duration treatment shown to slow the progression of early-stage Alzheimer’s disease.

In September, Bristol Myers Squibb got the agency’s greenlight for Cobenfy, the first major new drug for schizophrenia in 70 years. With the landmark approval of its epinephrine nasal spray, Neffy, ARS Pharmaceuticals has taken the needle—and needle-phobia—out of the equation for people who need epinephrine auto-injectors to treat severe allergic reactions.

Cancer immunotherapy remains a focus of innovation across the industry, with notable 2024 wins Iovance’s and Adaptimmune’s first-ever approvals for T-cell therapies targeting solid tumors. Addressing the burden of patients living with chronic disease, Pfizer built on its pipeline of innovative hemophilia therapies, while Verona’s dual-action treatment for COPD offers a breakthrough in maintenance therapy that will help some 16 million adults U.S. living with respiratory disease to breathe easier.

1. ARS Pharma

For developing a needle-free way to treat life-threatening allergic reactions

Roughly 1.5 million patients in the U.S. have a prescription for an Epipen or another epinephrine auto-injector, for the emergency treatment of allergic reactions. But many of them don’t regularly carry the drug with them, or they hesitate to use it because of the large needle. They got an alternative with the August 2024 Food and Drug Administration approval of Neffy, the first a nasal spray formulation of epinephrine, made by ARS Pharmaceuticals. The approval, which covers Neffy for adults and children who weigh at least 66 pounds, makes Neffy the first non-injectable epinephrine product for the treatment of life-threatening anaphylaxis. Neffy uses a novel chemical-compound technology called Intravail that ensures the drug can be absorbed through the nose at levels similar to injectable forms. The spray container requires no priming—you just insert and press—and its 30-month shelf life at temperatures up to 122°F is a big advantage over auto-injectors, which have 18-20 month shelf lives and stricter temperature requirements. Neffy could be especially useful for emergency medical units, first responders, and airlines, for whom needle-based therapies may not be permitted. The drug officially hit the U.S. market in late September, and by February 2025 insurance coverage was available for about 50% of people covered by commercial plans. A European version, EURneffy, will be available starting in early 2025, distributed by Denmark’s ALK-Abelló, and licensing partners have filed for approval of Neffy in China, Japan, and Australia. 

Read more about ARS Pharma, honored as No. 34 on Fast Company’s list of the World’s 50 Most Innovative Companies of 2025.

2. Bristol Myers Squibb

For bringing a new approach to schizophrenia treatment—at last

In September 2024, the FDA approved Cobenfy (xanomeline and trospium chloride) as the first major new schizophrenia drug in 70 years. Cobenfy uses an entirely different mechanism than existing treatments; it works by triggering muscarinic receptors, rather than dopamine receptors, and is the first schizophrenia treatment to do so. Cobenfy has been generally well tolerated in clinical trials and unlike the commonly used atypical antipsychotics, does not have a boxed warning of increased mortality in certain older patients. A rare breakthrough in a chronically difficult and underfunded illness, Cobenfy was acquired when it was in development from Karuna Therapeutics and successfully brought to market by BMS, which is now working to expand indications for the drug: the company could present data from its ADEPT program for Cobenfy in Alzheimer’s disease in 2026. BMS also plans to start three additional phase 3 trials in bipolar I disorder and Alzheimer’s disease.

3. Eli Lilly

For helping to slow Alzheimer’s-related cognitive decline

The FDA approved Lilly’s Alzheimer’s drug, Kisunla, a monoclonal antibody treatment for early symptomatic Alzheimer’s disease, in July. Developed internally at Lilly, Kisunla is the first and only amyloid plaque-targeting therapy that uses a limited-duration treatment regimen. In phase 3 trials of 1,736 patients, Kisunla slowed cognitive and functional decline by up to 35% compared to placebo at 18 months and reduced the risk of progressing to the next clinical stage of disease by up to 39%. Kisunla differs from its competitor therapy, Leqembi, manufactured by Biogen and Eisai, in that it needs to be administered every four weeks vs. every two weeks. Lilly has also continued to build on its lucrative GLP-1 franchise of Mounjaro and Zepbound. To compete against online compounders of knockoff drugs, in January, the company launched a direct-to-consumer channel for discount Zepbound prescriptions with home delivery. Continuing trials in new indications is also paying off, most notably with the December 23, 2024 FDA approved the weight-loss drug Zepbound as the first prescription medication specifically for treating obstructive sleep apnea, which affects an estimated 22 million Americans or more.

4. Aurion Biotech

For designing cell therapy that prevents blindness

Approved in Japan in May 2023 and commercially launched there in September 2024, Aurion’s Vyznova (AURN001) is the first clinically validated cell therapy for corneal care. The drug treats corneal endothelial disease, a progressive condition that affects millions of people throughout the world and which, if left untreated, leads to blindness. Prior to Vyznova, the only definitive treatment for bullous keratopathy, one form of corneal endothelial disease, was corneal transplantation using donor tissue, which is scarce—for every 70 diseased eyes, there is only one donor cornea available. Aurion has developed a manufacturing process that can produce up to 1,000 doses of Vyznova from the corneal endothelial cells of a single donor. In June 2024 the U.S. Food and Drug Administration granted Vyznova coveted Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) designations, giving the cell therapy an expedited path to approval. Data released in December from U.S. phase 1/2 clinical trials showed that the highest dose of Aurion’s cell therapy saw a statistically significant vision improvement.

5. Iovance Biotherapeutics

For fighting melanoma with a tumor-infiltrating immunotherapy

In February 2024, the FDA gave accelerated approval for Iovance’s Amtagvi to treat advanced melanoma (a Phase 3 trial is underway to confirm the clinical benefit seen in earlier trials). Amtagvi is the first one-time, individualized T-cell therapy approved for a solid tumor cancer. While CAR T-cell therapies can be powerfully effective in blood cancers, they haven’t performed as well in solid tumors. A one-time treatment, Amtagvi uses tumor infiltrating lymphocytes (TILs), naturally occurring immune cells that fight cancer, to durably treat some of these solid tumors. Some treated patients have been cancer-free for a decade or more. Amtagvi is manufactured in a proprietary process that collects and expands natural TIL cells that unique to each patient, but are usually depleted when they are sick. Then, billions of these killer cells are put back into their body to fight the cancer. The approval of Amtagvi is promising for TILs as a new class of drugs for solid tumors, with trials ongoing for lung cancer, cervical cancer, and more. The one-time cell has a cost $515,000 per patient, before rebates and discounts. The company expects that many insurance companies will cover the treatment at the same levels as CAR T-cell therapies.

6. Adaptimmune

For engineering T-cells to target solid tumors

On August 1, 2024, Adaptimmune received accelerated FDA approval of Tecelra, a gene therapy for the treatment of adults with a specific type of synovial sarcoma who have previously undergone chemotherapy. This approval made Tecelra the first engineered cell therapy for a solid tumor cancer approved in the U.S., and the first new therapy option in more than a decade for synovial sarcoma, a rare soft tissue cancer that most commonly impacts young adults. Tecelra is an “autologous” T cell immunotherapy, meaning that it is composed of a patient’s own T cells that are modified in a lab to express a receptor that targets the MAGE-A4 protein on cancer cells. When these modified T cells are infused back into the patient., they are “pre-trained” to find and attack cancer cells that express the MAGE-A4 protein, helping to stop the cancer from growing or spreading.

Trial data of Tecelra showed an overall response rate of 43%, with a complete response rate of 4.5% and a median duration of response of 6 months. Among patients who were responsive to the treatment, 39.% had a duration of response of 12 months or longer. In December, Adaptimmune announced that the first patient has been treated with Tecelra, at Moffitt Cancer Center, one of Adaptimmune’s Authorized Treatment Centers. The one-time treatment is launching with a list price of $727,000, the most expensive per-dose cost of any cellular medicine for cancer in the U.S. A second experimental T cell therapy called lete-cel has achieved its primary endpoint in a phase 2 trial. Adaptimmune estimates the two therapies combined could generate $400 million in peak yearly sales in the U.S.

7. Verona Pharma

For helping COPD patients breathe easier

On June 26, 2024, the U.S. Food and Drug Administration (FDA) approved Ohtuvayre (ensifentrine), Verona Pharma’s novel therapy for chronic obstructive pulmonary disease (COPD). The treatment is the first inhaled maintenance therapy with a novel mechanism of action in more than 20 years to treat the condition, and offers a breakthrough for some 16 million adults living with COPD in the US. A single, small-molecule drug, Ohtuvayre has a unique dual-action mechanism that selectively inhibits PDE3 and PDE4 enzymes, simultaneously enhancing bronchodilation (opening small airways in the lungs) and reducing inflammation associated with COPD—without the use of steroids. Verona is now conducting phase 2 clinical trials of ensifentrine in the treatment of other respiratory diseases with unmet medical needs, including chronic non-cystic fibrosis bronchiectasis, cystic fibrosis, and asthma.

8. Pfizer

For offering hemophiliacs once-weekly prophylactic treatment

In 2024 Pfizer bolstered its growing portfolio of innovative therapies for hemophilia, which inhibits the blood’s ability to clot properly and impacts more than 800,000 people globally. In October, the FDA approved Pfizer’s Hympavzi, the first once-weekly injection (administered with a pre-filled auto-injector pen) to prophylactically control excess bleeding in people with either hemophilia A or B. In other therapeutic areas, Pfizer has charged into the market for antibody drug conjugate drugs—ADCs— with the full FDA approval in April 2024 for Tivdak, which had previously won accelerated approval in 2021 for the treatment of patients with recurrent or metastatic cervical cancer. Tivdak, which Pfizer acquired through its acquisition of Seagen in December 2024, is the first ADC to have positive overall survival data for patients with previously treated recurrent or metastatic cervical cancer. The disease is typically untreatable and devastating, but the clinical trial for Tivdak showed a 30% reduction in risk of death compared to chemotherapy.

9. ImmunityBio

For activating cells to attack bladder cancer

Up to 40% of patients with non-muscle-invasive bladder cancer do not respond to traditional immunotherapy for the condition, known as BCG treatment, which uses a weakened bacteria. In April 2024, the FDA approved Anktiva, a first-in-class treatment and rare breakthrough in BCG-unresponsive non-muscle invasive bladder cancer. Anktiva, which is approved for maintenance therapy alongside the standard of care BCG therapy, to boost its effectiveness. Anktiva works in a novel way: It is a an “IL-15 receptor agonist” that activates natural killer (NK) and T-cells to attack tumors and cancer cells. Then, by stimulating memory T cells, it facilitates a lasting immune response, with some patients showing complete responses lasting over 47 months. After reaching the first patients this summer, ImmunityBio had secured coverage of treatment coverage from insurers who collectively cover some 200 million people. In the third quarter of 2024, Anktiva generated roughly $6 million in net product revenue. According to the company, as of November 2024, 100 patients had been treated with the Anktiva-BCG combination therapy, achieving a 71% complete response (CR) rate. Data released from phase 2 trials of Anktiva in patients with non-small-cell lung cancer, who did not respond to checkpoint inhibitors, showed median overall survival that was almost double that of standard-of-care chemotherapy.

10. Zevra Therapeutics

In September 2024, the US FDA approved Zevra Therapeutics Miplyffa, an oral medication for the treatment of Niemann-Pick disease, type C (NPC), a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. Miplyffa is the first drug approved by the FDA to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. NPC prevents the body from moving and using cholesterol and other lipids in cells, in turn causing a buildup of fats in the liver, spleen or lungs. The disease can take a toll on patients’ speech, cognition, swallowing, movement and fine motor skills, and people with the disease live about 13 years on average. The new drug was cleared in combination with the enzyme inhibitor miglustat, which has long been used as the primary, albeit off-label, treatment for NPC patients.

Explore the full 2025 list of Fast Company’s Most Innovative Companies, 609 organizations that are reshaping industries and culture. We’ve selected the companies making the biggest impact across 58 categories, including advertising, applied AI, biotech, retail, sustainability, and more.

https://www.fastcompany.com/91269637/medicines-therapeutics-pharmaceuticals-most-innovative-companies-2025?partner=rss&utm_source=rss&utm_medium=feed&utm_campaign=rss+fastcompany&utm_content=rss