Pharmaceutical Research and Approvals: What's Trending in 2024

In the ever-evolving field of pharmaceutical research and development, staying abreast of the latest trends and advancements is paramount. This year, the pharmaceutical industry is marked by groundbreaking innovations, regulatory shifts and unprecedented challenges. From cutting-edge technologies to novel therapeutic approaches, pharmaceutical research, and pharmaceutical approvals in 2024 are redefining how we perceive health care.

The 2024 Pharmaceutical Landscape

Unprecedented innovation and transformation are expected in pharmaceuticals this year. With the increasing integration of artificial intelligence, machine learning, precision medicine, and big data analytics into drug discovery and development processes, the pace of scientific breakthroughs is accelerating like never before.

This convergence of technology and biology is expected to yield a surge in the development of novel therapeutics, particularly in areas of high unmet medical need, such as rare diseases and neurodegenerative disorders like Alzheimer's and certain types of cancers.

Fueled by genomic and precision medicine improvements, personalized medicine continues to reshape how pharmaceutical companies develop and deliver treatments.

Exciting Pharmaceutical Approvals in 2024

Regulatory bodies have approved several exciting drugs for 2024. These groundbreaking therapies have received regulatory approval and are poised to impact patient care and disease management significantly. Here are the most anticipated pharmaceutical approvals in 2024:

KarXT by Karuna Therapeutics

KarXT, a novel treatment developed by Karuna Therapeutics, is one of the most eagerly awaited pharmaceutical approvals. KarXT represents a pioneering approach in neuropsychiatric disorder management, particularly schizophrenia.¹

This innovative therapy modulates key neurotransmitter pathways in the brain associated with psychotic symptoms. Following rigorous clinical trials demonstrating its efficacy and safety, KarXT received Food and Drug Administration (FDA) approval, marking a significant milestone in psychiatry.¹

Sotatercept by Merck & Co.

Sotatercept represents another cutting-edge advancement in treating pulmonary arterial hypertension, a type of high blood pressure affecting the arteries in the heart and lungs. This therapy targets the pathway responsible for regulating cell growth and differentiating bone marrow. The drug was granted FDA approval following a phase 3 STELLAR trial.²

Tryvio by Idorsia Pharmaceuticals

Tryvio treats hypertension, a prevalent cardiovascular condition affecting millions worldwide. Unlike conventional antihypertensive medications primarily targeting blood vessel constriction or fluid balance, Tryvio targets the endothelin (ET) pathway.³

As the first antihypertensive drug in over four decades to target a different therapeutic pathway, Tryvio demonstrates the potential to achieve superior blood pressure control with fewer adverse effects. Idorsia aims to use Tryvio to treat hypertension patients who’ve had little to no success from other antihypertensive treatments.³

Donanemab by Eli Lilly

Donanemab represents a significant breakthrough in the treatment of early-stage Alzheimer’s disease. Unlike previous treatments that have primarily targeted symptomatic relief, Donanemab slows down disease progression by selectively targeting and clearing toxic amyloid plaques from the brain.⁴

Donanemab aims to slow cognitive decline and preserve brain function, potentially leading to improved outcomes and enhanced quality of life for those living with Alzheimer’s. In phase three of the study, patients on Donanemab had a 39% lower risk of disease progression.⁵

Datopotamab deruxtecan by Daiichi Sankyo and AstraZeneca

Datopotamab deruxtecan marks a milestone in the oncology field, particularly in the treatment of metastatic breast and lung cancers — a challenging and aggressive form of cancer. This innovative therapy delivers a targeted approach to destroying cancer cells while minimizing damage to healthy tissues.⁶

The FDA has accepted the US biologics license application (BLA) and is set to make a decision in the fourth quarter of the year. The application requests the approval of the drug for locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) in adults who have previously undergone systemic therapy.⁷

Technologies Transforming Drug Discovery

A wave of new technologies is reshaping the pharmaceutical industry, accelerating the pace of innovation and opening new frontiers for researchers. At the forefront of this revolution are several key technologies that are revolutionizing the way scientists identify, develop, and bring new drugs to the market.

AI and Machine Learning

AI and machine learning are transforming drug discovery by enabling researchers to sift through vast amounts of data more efficiently and identify potential drug candidates with greater precision. AI algorithms can analyze complex biological datasets, predict drug-target interactions and even design novel compounds with specific therapeutic properties. This efficiency allows for a much faster drug discovery pipeline.⁸

As these technologies continue to evolve and integrate with other cutting-edge tools, they promise to improve drug discovery, accelerate the development of safer, more effective therapies, and ultimately improve patient outcomes across a wide range of diseases. 

The Rise of Precision Medicine

Scientists and researchers believe 2024 to be the breakout year for precision medicine, offering patients tailored treatments with fewer adverse effects.

“We’re seeing more and more investment in the genetic editing field and the targeting of specific proteins and biomarkers. This is because of the ability to target specific disease states and molecular or cellular targets to offer the best chance for an optimized response that leads to disease regression. For investors and for companies, tailoring an approach that you have a higher probability of success and more clear success criteria for versus throwing something against the wall and seeing what sticks will drive a lot of interest. It’s better for the patient as well because it could mean less side effects and tailored, specific treatments that give more hope and better progress overall. For the medical industry, it won’t be one-size-fits-all anymore.” says Mark Melton, vice president of scientific operations and development at Slope on precision medicine. [SOURCE: https://www.pharmexec.com/view/precision-medicine-breakout-year-2024-trends]  

By leveraging advances in genomics, proteomics, and other technologies, precision medicine will tailor treatments to the individual characteristics of each patient. This approach recognizes that different patients may respond differently due to variations in their genetic makeup, lifestyle, and environmental factors.⁹

By identifying biomarkers and molecular signatures associated with disease susceptibility and treatment response, precision medicine enables health care providers to make more informed decisions about which therapies are likely to be most effective for individual patients.⁹

Additionally, ensuring high-quality patient care has become essential. Hospitals are increasingly turning to outsourcing companies specialized in enhancing the patient experience. Approximately 80% of health care clients express confidence that these outsourcing partnerships improve the quality of patient care. This shift reflects a growing recognition of the importance of patient-centered care and the valuing of leveraging external expertise to optimize health care delivery.

Biopharmaceutical Advancements

Biopharmaceuticals — which include biologics such as monoclonal antibodies, therapeutic proteins, and gene therapies — are derived from biological sources and offer unique advantages over small-molecule drugs. These advantages include:

• Greater specificity
• Higher potency
• Reduced toxicity
• Long-lasting effects
• Less likely to elicit immune responses
• Flexibility due to various formats
• Personalized medicine

These benefits make biopharmaceuticals particularly well-suited for targeting diseases such as cancer, autoimmune disorders, and rare genetic conditions.

Recent biopharmaceutical innovations have propelled the field forward, offering new possibilities for treating a wide range of diseases. One significant advancement is the emergence of next-generation antibody-based therapies, such as bispecific antibodies and antibody-drug conjugates (ADCs). Bispecific antibodies are engineered to simultaneously target two different antigens or cell types, enhancing their specificity and efficacy.¹⁰

These novel molecules hold promise for treating complex diseases like cancer by engaging multiple pathways involved in tumor growth and immune evasion.¹⁰

Similarly, ADCs combine the targeting specificity of antibodies with the cytotoxic properties of chemotherapy drugs, allowing for precise delivery of potent anticancer agents directly to tumor cells while minimizing systemic toxicity. These advancements represent a shift in oncology and other therapeutic areas, offering new treatment options for patients with challenging-to-treat diseases.¹⁰

Another notable biopharmaceutical advancement is the development of gene therapies and nucleic acid-based therapeutics. Gene therapy aims to treat or prevent disease by delivering genetic material directly into cells to correct or replace faulty genes.

Recent breakthroughs in gene editing technologies, such as CRISPR-Cas9, have enabled precise and efficient genome editing, opening up new possibilities for treating genetic disorders, cancer, and infectious diseases.¹¹

Additionally, mRNA vaccines and RNA interference (RNAi) therapies are transforming how we prevent and treat diseases. mRNA vaccines, such as those developed for COVID-19, harness the body’s own cellular machinery to produce antigens and stimulate immune responses, offering rapid and scalable vaccine production platforms. Biopharmaceutical companies now aim to extend mRNA efforts beyond COVID-19.¹²

RNAi therapies use small RNA molecules to silence or modulate the expression of specific genes, offering potential treatments for conditions ranging from rare genetic disorders to viral infections and neurodegenerative diseases.¹³

Emerging Regulatory Considerations Influencing Drug Approval Processes

Current and emerging regulatory considerations are shaping the landscape of drug approval processes, reflecting the nature of health care and the need for more robust oversight of novel therapies. Several key factors are driving these changes and influencing how regulators approach the evaluation and approval of new drugs.

Advanced Therapies

The rise of advanced therapies, including gene therapies, cell-based therapies, and tissue-engineered products, has prompted regulators to develop specialized frameworks to ensure their safety, efficacy, and quality. These therapies often present unique challenges related to manufacturing and administration, necessitating tailored regulatory approaches to facilitate their development and approval. The Center for Biologics Evaluation and Research (CBER) regulates these therapies in the U.S.¹⁴

Real-World Evidence (RWE)

There is a growing recognition of the value of real-world evidence in complementing traditional clinical trial data and informing regulatory decisions. Regulators are exploring ways to incorporate RWE, including data from electronic health records, claims databases, and patient registries, in the drug approval process to provide additional insights into a drug’s effectiveness and safety in real-world settings.¹⁵

Patient-Centered Drug Development

Regulatory agencies increasingly prioritize patient input and engagement throughout drug development and approval. This includes soliciting patient perspectives on treatment outcomes, preferences, and tolerability, as well as involving patients in the clinical trial design and benefit-risk profile evaluation. By incorporating patient-centric approaches, regulators aim to ensure that new therapies address unmet patient needs and align with patient priorities.¹⁶

Digital Health Technologies

The proliferation of digital health technologies, such as wearable devices and digital biomarkers, reshapes how health care is delivered and monitored. Regulators are exploring how to evaluate and regulate these technologies to ensure their safety, effectiveness, and data privacy while fostering innovation and facilitating their integration into clinical practice.¹⁷

Global Harmonization

As drug development becomes increasingly globalized, regulators are collaborating more closely to harmonize regulatory standards and streamline the drug approval process across jurisdictions. Initiatives such as the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use (ICH) aim to promote mutual recognition of regulatory decisions, reduce duplication of efforts, and facilitate timely access to safe and effective therapies for patients worldwide.¹⁸

Challenges of Bringing New Drugs to Market

Bringing new drugs to market is a challenging and resource-intensive process involving numerous hurdles and uncertainties. Some of the key challenges in this process include:

Research and Development (R&D) Costs

Developing a new drug from discovery to market approval requires substantial financial investment, often costing billions of dollars. On average, it costs approximately $2 billion to develop a new medication. High R&D costs contribute to the expense of bringing a new drug to market, and the failure rate of drug candidates during clinical trials further compounds these expenses.¹⁹

Regulatory Hurdles

Regulatory approval is crucial, and navigating the regulatory landscape can be challenging. Regulatory agencies such as the FDA and the European Medicines Agency (EMA) have rigorous safety and quality standards that drugs must meet before they can approve them for market entry.

Clinical Trial Complexity

Conducting clinical trials is complex and time-consuming. Recruiting suitable candidates, ensuring compliance, and managing logistics all pose challenges, often slowing down the process and halting many trials. Moreover, designing clinical trials that generate robust and clinically meaningful data adds another layer of complexity to the process.²⁰

Market Access and Reimbursement

Even after receiving regulatory approval, gaining market access and securing reimbursement for a new drug can be difficult. Demonstrating the value of a new treatment relative to existing treatments and negotiating pricing agreements with payers are critical to ensuring market uptake.

New Trends Improving Health Care in 2024

It’s evident that the pharmaceutical industry is on the brink of transformative change. From AI and machine learning and navigating the complexities of regulatory pathways to several pharmaceutical approvals in 2024, the journey toward advancing health care will continue to be defined by innovation, perseverance, and a commitment to improving patient outcomes.

References

1. Karuna Therapeutics announces U.S. Food and Drug Administration accepts New Drug Application for KARXT for the treatment of schizophrenia. Karuna Therapeutics. November 29, 2023. Accessed April 11, 2024. https://investors.karunatx.com/news-releases/news-release-details/karuna-therapeutics-announces-us-food-and-drug-administration.
2. FDA approves Merck’s Winrevair (sotatercept-csrk), a first-in-class treatment for adults with pulmonary arterial hypertension (PAH, who* group 1). Merck.com. March 26, 2024. Accessed April 11, 2024. https://www.merck.com/news/fda-approves-mercks-winrevair-sotatercept-csrk-a-first-in-class-treatment-for-adults-with-pulmonary-arterial-hypertension-pah-who-group-1/.
3. US FDA approves Idorsia’s once-daily tryvio (aprocitentan) - the first and only endothelin receptor antagonist for the treatment of high blood pressure not adequately controlled in combination with other antihypertensives. PR Newswire. March 20, 2024. Accessed April 11, 2024. https://www.prnewswire.com/news-releases/us-fda-approves-idorsias-once-daily-tryvio-aprocitentan--the-first-and-only-endothelin-receptor-antagonist-for-the-treatment-of-high-blood-pressure-not-adequately-controlled-in-combination-with-other-antihypertensives-302094474.html.
4. Donanemab for treatment of early alzheimer’s disease - news pending FDA Review. Alzheimer’s Disease and Dementia. Accessed April 11, 2024. https://www.alz.org/alzheimers-dementia/treatments/donanemab. 
5. Lilly’s Donanemab significantly slowed cognitive and functional decline in phase 3 study of early alzheimer’s disease. Eli Lilly and Company. May 3, 2023. Accessed April 11, 2024. https://investor.lilly.com/news-releases/news-release-details/lillys-donanemab-significantly-slowed-cognitive-and-functional.
6. Datopotamab deruxtecan biologics license application accepted in the US for patients with previously treated advanced nonsquamous non-small cell lung cancer. February 19, 2024. Accessed April 11, 2024. https://www.astrazeneca.com/media-centre/press-releases/2024/fda-accepts-dato-dxd-bla-for-nonsquamous-nsclc.html.
7. Saraceno N. FDA to evaluate bla for Datopotamab Deruxtecan in non-small cell lung cancer. PharmExec. February 20, 2024. Accessed April 11, 2024. https://www.pharmexec.com/view/fda-to-evaluate-bla-for-datopotamab-deruxtecan-in-non-small-cell-lung-cancer. 
8. Dashpute, Sakshi & Pansare, Jagruti & Deore, Yashashri & Pansare, Mayur & Sonawane, Poonam & Jadhav, Shivraj & Jadhav, Shivraj. (2023). Artificial Intelligence and Machine Learning in the Pharmaceutical Industry INTRODUCTION. International Journal of Pharmacy and Pharmaceutical Research. 28. 111-131.
9. Akhoon N. Precision Medicine: A New Paradigm in Therapeutics. Int J Prev Med. 2021 Feb 24;12:12. doi: 10.4103/ijpvm.IJPVM_375_19. PMID: 34084309; PMCID: PMC8106271.
10. Crescioli S, Kaplon H, Chenoweth A, Wang L, Visweswaraiah J, Reichert JM. Antibodies to watch in 2024. mAbs. 2024;16(1). doi:10.1080/19420862.2023.2297450
11. Li Z-H, Wang J, Xu J-P, Wang J, Yang X. Recent advances in CRISPR-based genome editing technology and its applications in Cardiovascular Research. Military Medical Research. 2023;10(1). doi:10.1186/s40779-023-00447-x
12. MRNA technology transfer programme moves to the next phase of its development. World Health Organization. April 20, 2023. Accessed April 11, 2024. https://www.who.int/news/item/20-04-2023-mrna-technology-transfer-programme-moves-to-the-next-phase-of-its-development.
13. Traber GM, Yu A-M. RNAi-based therapeutics and novel RNA Bioengineering Technologies. Journal of Pharmacology and Experimental Therapeutics. 2022;384(1):133-154. doi:10.1124/jpet.122.001234
14. Center for Biologics Evaluation and Research. Cellular & Gene Therapy Products. U.S. Food and Drug Administration. March 20, 2023. Accessed April 11, 2024. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products.
15. Vaghela, S., Tanni, K.A., Banerjee, G. et al. A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases. Orphanet J Rare Dis 19, 117 (2024). https://doi.org/10.1186/s13023-024-03111-2
16. CDER’s patient-focused drug development. U.S. Food and Drug Administration. February 14, 2024. Accessed April 11, 2024. https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development.
17. Digital Health Technologies. U.S. Food and Drug Administration. December 26, 2023. Accessed April 11, 2024. https://www.fda.gov/science-research/science-and-research-special-topics/digital-health-technologies-dhts-drug-development.
18. Global and regional regulatory harmonization initiatives. World Health Organization. Accessed April 11, 2024. https://www.who.int/teams/regulation-prequalification/regulation-and-safety/regulatory-convergence-networks/harmonization.
19. The unbearable cost of drug development: Deloitte Report shows 15% jump in R&D to $2.3 billion. GEN. February 28, 2024. Accessed April 11, 2024. https://www.genengnews.com/gen-edge/the-unbearable-cost-of-drug-development-deloitte-report-shows-15-jump-in-rd-to-2-3-billion/.
20. Markey N, Howitt B, El-Mansouri I, Schwartzenberg C, Kotova O, Meier C. Clinical trials are becoming more complex: a machine learning analysis of data from over 16,000 trials. Sci Rep. 2024 Feb 12;14(1):3514. doi: 10.1038/s41598-024-53211-z. PMID: 38346965; PMCID: PMC10861486.