Specialty Drug Growth Continues: A Look at the Pipeline

Key specialty pharmaceuticals market trends include increased competition along with cancer and orphan drug development, according to Aimee Tharaldson, PharmD, senior clinical consultant of emerging therapeutics at Express Scripts in Woodbury, Minn.

In the past four years, a number of specialty drug generic options have been approved, representing $14 billion in overall U.S. spending opportunities, with the potential for $24 billion in expected specialty generics in the next five years. Through 2023, 71 specialty therapy patents will expire, representing a $55 billion market opportunity. In addition, the biosimilar market keeps growing, as seven more agents are projected to be approved this year. There has also been extensive oncology drug development over the past seven years, with 2017 and 2018 seeing increasing growth after a slowdown in 2016.

The orphan drug pipeline, however, is experiencing extensive growth, as these agents represent more than half (53%) of the current specialty pharmacy pipeline. Food and Drug Administration approvals for the agents continue to grow in comparison with traditional drugs. In quarter one of 2019, four specialty drugs have already been approved: caplacizumab‐yhdp (for acquired thrombotic thrombocytopenic purpura), turoctocog alfa pegol (for hemophilia A), esketamine (for depression), and brexanolone (for depression).

Dr. Tharaldson then provided an extensive look at projected approvals for the top specialty pharmaceutical drug classes.

Inflammatory conditions are the leading therapy class based on per-member, per-year spending ($153 billion). The pipeline includes:

  • Risankizumab, an interleukin (IL)‐23 inhibitor for psoriasis (expected 2019)
  • Upadacitinib, a JAK‐1 inhibitor for rheumatoid arthritis (RA; 2019)
  • Ustekinumab, an IL‐12 and ‐23 inhibitor for ulcerative colitis (2019)
  • Bimekizumab, an IL‐17A and ‐17F inhibitor for psoriasis (2020)
  • Filgotinib, a JAK‐1 inhibitor for RA, ulcerative colitis, and Crohn’s disease (2020)

Various multiple sclerosis (MS) medications are in development, including:

  • Cladribine, a nucleoside analog that depletes B and T lymphocytes for relapsing MS (RMS; 2019)
  • Siponimod, a sphingosine 1‐phosphate receptor modulator for secondary progressing MS (2019)
  • Diroximel fumarate, a monomethyl fumarate prodrug for RMS (2019)
  • Ozanimod, a sphingosine 1‐phosphate receptor modulator for RMS (2019)
  • Dimethyl fumarate, a monomethyl fumarate prodrug for RMS (2020)

A number of oncology products are in development and are projected to be approved before the end of the year, including:

  • Erdafitinib for urothelial cancer
  • Quizartinib for acute myeloid leukemia
  • Selinexor for multiple myeloma
  • Pexidartinib for tenosynovial giant cell tumor
  • Entrectinib for NTRK‐fusion+ solid tumors
  • Polatuzumab for diffuse large B-cell lymphoma
  • Darolutamide for prostate cancer
  • Fedratinib for myelofibrosis

In addition, four new HIV medications are expected to be approved within the next two years, including dolutegravir/lamivudine, cabotegravir/rilpivirine, fostemsavir, and leronlimab (PRO‐140).

The nonalcoholic steatohepatitis pipeline for 2020 and beyond is extensive as well, with 11 phase II and phase III drugs pending approval. This is particularly important, as current treatment options are limited; most recommendations for treatment are focused on weight, diet, and exercise. However, these new treatment options come with many unknowns, including a steep projected price of $3,000 to $70,000 per year. It remains to be determined which patients will be prescribed the medications and which will remain on a diet and exercise-focused regimen.

There are nine medications pending approval for Alzheimer’s disease, which are projected for approval in 2021 to 2024 and beyond. The hemophilia market has seven medications in the pipeline, which could result in approvals between 2020 and 2022.

Lastly, Dr. Tharaldson discussed some unique pipeline drugs, including:

  • Romosozumab for postmenopausal osteoporosis
  • Onasemnogene for spinal muscular atrophy
  • Bremelanotide for hypoactive sexual desire disorder
  • Celiprolol for vascular Ehlers‐Danlos syndrome
  • Tafamidis for transthyretin cardiomyopathy
  • Afamelanotide for erythropoietic protoporphyria
  • Fenfluramine for Dravet syndrome
  • Golodirsen for Duchenne muscular dystrophy

Presentation P1: Specialty Pharmaceuticals in Development. AMCP Annual Meeting 2019.