Asembia 2025: The Specialty Pharma Pipeline

The first day of Asembia’s AXS25 Summit started off strong with a multitude of sessions to choose from, and being that the conference revolves around specialty pharmacy, it might come as no surprise that one of the seminars on the docket focused on the “Specialty Pharmacy Pipeline: A Glimpse at What’s Ahead.”

Being led by Brad McElya, PharmD, Walgreens’ senior director of clinical pharmacy strategy, the goal of the session was to investigate the top trends pertaining to specialty drug and therapy development, with a closer look at drug development landscape.

“At the core of this discussion is patient care and innovation, and I believe the pipeline offers hope for patients with rare and common, debilitating diseases, signaling progress or we still have opportunities to make difference in patients’ lives,” he said. “This topic is vital because it provides insights into market dynamics, competitive landscapes, and helps us anticipate market shifts, identify emerging trends, and strategically position products for success. Additionally, it aligns our objectives with patients’ needs, fostering a patient-centric approach that resonates with stakeholders and regulators.”

Although the broader pipeline includes over 140 drugs across various therapeutic areas, it’s important to note that McElya focused on a select few for the sake of providing a deeper dive—during the presentation, he listed all of the drugs for the audience, but explored some of what he believed were the key therapies in greater detail. Many of these drugs represent years of research investment and collaboration, with the potential to transform patients’ lives.

The specialty therapeutic area revolves around a multitude of trends including rare and orphan growth; oncology breakthroughs; cell and gene therapies; immunology expansion; neurology focus; diabetes and obesity; and biosimilars growth. As a result, this niche sector continues to be powered by growth.

“The specialty pharmaceutical market is rapidly growing and now makes up 51% of total market sales. This growth is driven by advancements in biotechnology, personalized medicine and treatments for rare diseases,” McElya explained. “In 2024, the Center for Drug Evaluation and Research (CDER) approved 50 novel drugs, and 24 of them—or 48%—were for first-in-class drugs, which are unique because these produce new pharmacologic effects targeting specific biological mechanisms and diseases. We've seen that the specialty pharmaceutical market is evolving quickly, and is driven by innovation and the need for advanced treatments.”

From the specialty therapeutic category for instance, Aficamten by Cytokinetics is indicated for obstructive hypertrophic cardiomyopathy; there's also a phase II trial ongoing for chronic heart failure. It is considered a next-in-class cardiac myosin inhibitor designed to reduce myocardial hyper contractility, presenting significant improvements in exercise capacity, symptom burden, as well as cardiac biomarkers in patients with Arrhythmogenic cardiomyopathy or ACM. The targeted PDUFA date—also known as the targeted date for the FDA to make a decision on the approval—is Sept. 26 of this year.

Contrary to popular belief, rare and orphan diseases are not as rare as one might believe. Although, it’s defined as any condition affecting less than 200,000 people in the United States, approximately 1 in 10 Americans have a rare disease. Further, according to data from the National Organization for Rare Diseases, these are predominantly due to genetic disorders, and from a financial perspective, generate medical costs three to five times higher than non-rare diseases.

One drug that could be making a splash sooner rather than later is Elamipretide by Stealth BioTherpeutics for cardiomyopathy and Barth syndrome. It stabilizes mitochondrial structure, reduces oxidative stress, and enhances ATP production, has shown potential in improving mitochondrial function and reducing symptoms in clinical trials. There are currently no FDA-approved treatments for the ultra-rare genetic condition that’s characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth.

However, the targeted PDUFA date is tomorrow, April 29, with a potential first launch by June 30, 2025.

McElya also touched on the oncology pipeline—cancer’s current projections represent two million diagnosed cases and 1,700 deaths per day, with a predicted 50% of those being breast, prostate, lung, and coon/rectum cancers.

Reference

McElya B. Specialty Pharmacy Pipeline: A Glimpse at What’s Ahead. April 28, 2025. Asembia AXS25 Summit. Las Vegas.