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Conference Coverage

Rare Orphan, Oncology, and Specialty Drugs in Development: Review of Therapeutic Areas, Designations, and Launch Projections

Danielle Sposato

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.