Top Specialty Pharmaceutical Pipeline Developments Health Execs Should Watch


Expiring patents and biosimilar developments are creating big market opportunities within the specialty drug space.

That’s according to Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts, who discussed the specialty pharmaceutical pipeline at the Academy of Managed Care Pharmacy Managed Care and Specialty Pharmacy Annual Meeting in Boston April 24.

Tharaldson defined specialty medications as those that require frequent dosing adjustments and/or intensive clinical monitoring. Such drugs also require intensive patient training and/or compliance assistance and there are limits to their distribution. Further, these medications require specialized handling and administration by healthcare providers.

Patent expirations

Specialty generics is a $25 billion market, said Tharaldson. It’s also a market where 64 patents will expire through 2022.

She also noted that, in 2018, seven specialty pharmaceuticals (non-generic) lose patient protection, which represents $3.4 billion in U.S. market opportunity. These are:

  • Adcirca (tadalafil) for erectile dysfunction, enlarged prostate, and high blood pressure in the lungs
  • Ampyra (dalfampridine) for multiple sclerosis
  • Letairis (ambrisentan) for pulmonary arterial hypertension
  • Makena (hydroxyprogesterone caproate injection) for reducing premature births
  • Norvir (ritonavir) for HIV
  • Viread (tenofovir disoproxil) for hepatis B and HIV infection
  • Zytiga (abiraterone acetate) for prostate cancer

Looking ahead to 2021, eight specialty pharmaceuticals (non-generic) will lose patents, which alone represents $9.6 billion in opportunity in the U.S. market. Pharmaceuticals in this grouping are:

  • Atripla (efavirenz/emtricitabine/tenofovir disoproxil fumarate) for HIV
  • Intelence (etravirine) for HIV
  • Jevtana (cabazitaxel) for prostate cancer
  • Northera (droxidopa) for neurogenic orthostatic hypotension, a rare condition that causes a drop in blood pressure when standing
  • Selzentry (maraviroc) for HIV
  • Sutent (sunitinib malate) for a variety of cancers
  • Tecfidera (dimethyl fumarate) for multiple sclerosis
  • Truvada (emtricitabine/tenofovir disoproxil fumarate) for HIV


The session also included an overview of the future opportunities for biosimilars, which present a $54.4 billion opportunity within the United States.

The nine approved biosimilars for the U.S. market include:

  • Zarxio (filgrastim-sndz) for preventing infection
  • Inflectra (infliximab-dyyb) for inflammatory diseases
  • Erelzi (etanercept-szzs) for inflammatory diseases
  • Amjevita (adalimumab-atto) for inflammatory diseases
  • Renflexis (infliximab-abda) for inflammatory diseases 
  • Cyltezo (adalimumab-adbm) for inflammatory diseases
  • Mvasi (bevacizumab-awwb) for certain colorectal, lung, brain, kidney, and cervical cancers
  • Ogivri (trastuzumab-dkst) for breast or metastatic stomach cancer
  • Ixifi (infliximab-qbtx) for inflammatory diseases

Ten other biosimilars are pending approval by the FDA. These biosimilars, which Tharaldson says should be approved this year, are for the treatment of anemia, breast cancer, neutropenia (abnormally low white blood cell count), rheumatoid arthritis, non-Hodgkin's lymphoma, chronic lymphocytic leukemia, and psoriasis.

She said that eventually biosimilars will lead to cost savings, but they face significant legal hurdles and challenges capturing market share.

Next: Pipeline Developments to Watch 

Pipeline developments

Cancer drugs comprise 21% of all specialty drugs in development, said Tharaldson. But that makes sense, since there are approximately 1.7 million new cases diagnosed each year in the United States, according to the American Cancer Society, which also finds there are nearly 610,000 deaths due to cancer each year. Still, the death rate has dropped 25% since 1991, and there may be a time when cancer can be treated as a chronic condition.

Tharaldson also highlighted that orphan drug development represents 44% of specialty drug development. (The remaining 35% of specialty drugs in development cover other specialties.)

Specialty drugs represented 41% of all pharmacy drug spending in 2017, whereas the remaining 59% was on traditional drugs, said Tharaldson. Inflammatory disease is the leading specialty therapy in terms of spend per patient, followed by oncology, multiple sclerosis, HIV, and hepatitis C.

In 2017, the FDA approved 12 specialty drugs for use in the treatment of cancer, angioedema, hemophilia A, and inherited retinal dystrophy, she said. As of April 2018, the federal agency approved six specialty drugs that treat HIV, cystic fibrosis, prostate cancer, neuroendocrine tumors, psoriasis, X-linked hypophosphatemia (an inherited disorder characterized by low levels of phosphate in the blood), and chronic immune thrombocytopenic purpura (an autoimmune disease that can lead to easy or excessive bruising and bleeding).

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