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Rare Orphan, Oncology, and Specialty Drugs in Development: Review of Therapeutic Areas, Designations, and Launch Projections
The specialty pharmacy pipeline was the subject of one of many informative sessions at the annual Asembia Summit this year. Ray Tancredi, DVP, Pharma Relations & Specialty Pharmacy Development, Walgreens, offered key insights about current drugs in the pipeline according to their drug designations, the phase of each drug’s development, and projected first launch dates after receiving FDA approval. Mr Tancredi’s focus was echoed in his presentation, with him stating, “…it became really apparent to me that [drug development and approvals are] really evolving very quickly. And never before have I realized how important the pursuit of innovation is.”
Mr Tancredi’s insights provided payers, pharmacists, and health care professionals alike with a preview of upcoming drugs in the pipeline and the anticipated timeline for their approval and treatment considerations.
Mr Tancredi separated each drug into 3 key categories: rare orphan drugs, oncology drugs, and traditional specialty drugs. He focused on the most promising drugs in the pipeline in these categories according to their drug designations and first launch dates.
One constant that Mr Tancredi emphasized is that rare diseases and rare drugs are not truly rare anymore. Mr Tancredi said, “When you look at it, a rare disease is any condition that impacts 200,000 people or less, but statistics show that 1 in 10 Americans actually have a rare disease.” He also highlighted that the cost of care for patients with rare diseases is now nearly 3 or 5 times more expensive than the typical patient. Referring to statistics, Mr Tancredi supported this statement by saying “the total cost of care [for rare diseases] is about $400 billion,” which rivals cancer, heart failure, and even Alzheimer disease. He notes that despite that there are about 10,000 rare and orphan diseases, the FDA only approved drugs for about 5% of them.
“There’s a lot of opportunity and innovation in the rare and orphaned disease space,” Mr Tancredi said.
Rare Orphan Drugs
Focusing on the most notable rare disease and orphan drugs discussed during Mr Tancredi’s session, here’s what the industry can expect for 2024 and into 2025.
Drug Name |
Therapeutic Area(s) |
Drug Designation(s) |
Expected First Launch |
Glepaglutide (Zealand Pharma) |
Short Bowel Syndrome |
Orphan Drug |
6/30/2024 |
Cemdisiran (Alnylam Pharmaceuticals) |
Myasthenia Gravis (MG) |
Phase III |
12/31/2024 |
Marstacimab (Pfizer) |
Hemophilia A and B |
Orphan Drug, Fast Track |
12/31/2024 |
Nipocalimab (J&J) |
MG |
Phase III |
Expected in 2025 |
Acoramidis (AstraZeneca) |
Amyloidosis |
Currently in PEDUFA as of November 29, 2024, Orphan Drug |
Expected in 2024 |
Elafibranor (Ipsen) |
Primary Biliary Cholangitis |
PEDUFA slated for June 2024, Orphan Drug, Breakthrough Therapy |
Expected in 2024 |
Latozinemab (GSK/Alector) |
Frontotemporal Dementia |
Orphan Drug, Breakthrough Therapy |
12/31/2024 |
Rilzabrutinib (Sanofi) |
Autoimmune Hemolytic Anemia, Immune Thrombocytopenic Purpura, Asthma, Chronic Spontaneous Urticaria, Atopic Dermatitis, Purigo Nodularis, IgG4-Related Disease |
Orphan Drug, Fast Track |
12/31/25 |
Paltusotine (Crinetics) |
Acromegaly |
Orphan Drug |
12/31/25 |
Garadacimab (CSL Behring) |
Hereditary Angioedema (HM) |
Orphan Drug |
6/30/25 |
Vanzacaftor (Vertex) |
Cystic Fibrosis |
Orphan Drug, Fast Track |
12/31/24 |
Sebetralstat (KalVista) |
HM |
Orphan Drug, Fast Track |
12/31/24 |
Specialty Drugs
Mr Tancredi also shared notable specialty drugs in the pipeline. Of the several drugs awaiting approval in the specialty pipeline, there were about 6 designated for multiple sclerosis. While the therapeutic already has established drugs on the market, “there are more improvements” coming that are expecting approval soon. “The pipeline remains pretty fertile in traditional specialty pharmacy,” he said.Mr Tancredi also noted that the same can be said about drugs slated for approval for metabolic dysfunction-associated steatohepatitis (MASH), with 11 products for those disease states coming soon. The drugs identified in the table below are for other therapeutic areas.
Drug |
Therapeutic Area |
Drug Designation(s) |
Expected First Launch |
Valiltramiprosate (Alzheon) |
Alzheimer Disease |
Fast Track |
12/31/25 |
Bepirovirsen (GSK/Ionis Pharmaceuticals) |
Hepatitis B |
Fast Track |
12/31/25 |
Deuruxolitinib (Sun Pharmaceuticals) |
Alopecia Areata |
In review with the FDA, Breakthrough Therapy |
May be approved by June 2024 |
Remibrutinib (Novartis) |
Chronic Spontaneous Urticaria |
None |
12/31/25 |
Resmetirom (Madrigal Pharmaceuticals) |
Metabolic Dysfunction Associate Steatohepatitis |
Breakthrough Therapy, Priority Review |
TBD |
Atrasentan (Novartis) |
IgA Nephropathy |
None |
12/31/24 |
Frexalimab (Sanofi) |
Multiple Sclerosis |
None |
12/31/25 |
Oncology Drugs
Though the oncology pipeline continues to remain strong, rising demand is expected due to the earlier detection of cancers compared to previous years.
“Even though cancer death rates were on a steady decline over the last 30 years, I think in 2024 it’s expected, for the first time, [we’ll see] new cancer diagnoses are expected to exceed 2 million,” said Mr Tancredi. “That is approximately 5,500 diagnoses per day.”
The cancers most often detected in men are prostate, lung, colon, and rectum cancer. These account for almost half of all cancers found in men. For women, the most often detected cancer types are breast, lung, colon, and rectum, which account for almost 51% of cancer in women.
Mr Tancredi highlighted that FDA drug approvals have remained constant, but there have been numerous questions about why the oncology drug pipeline may be slowing down. At the end of 2023, there were 16 oncology drugs approved. Mr Tancredi reviewed what to expect for 2024 and 2025.
Drug |
Therapeutic Area |
Drug Designation(s) |
Expected First Launch |
Tarlatamab (Amgen) |
Small-Cell Lung Cancer |
Breakthrough Therapy, Priority Review |
Sometime in 2024 |
Teliso-v (AbbVie) |
Non-Small-Cell Lung Cancer |
Breakthrough Therapy |
TBD |
Tevimbra (BeiGene) |
Esophageal Cancer |
Orphan Drug |
June 2024 |
What’s Next?
Despite only intending to highlight key drugs in the specialty pipeline, Mr Tancredi's inability to discuss all of them in a single conference session emphasizes the wide range of efforts to develop new treatment options across different therapeutic fields. Numerous promising drugs are currently in the pipeline, and attendees were advised to monitor expected approvals through the rest of 2024.
Reference
Tancredi R. The Specialty Pipeline. Presented at: Asembia Summit 2024; April 29, 2024; Las Vegas, NV.