Session Details the Specialty Pharmacy Pipeline

The specialty pharmaceutical market is continuing to see increased competition, and in the past 10 years, there has been a significant number of new options to treat patients with several specialty conditions. During a session at AMCP Nexus 2020 Virtual, Aimee Tharaldson, PharmD, senior clinical pharmacist of emerging therapeutics at Express Scripts, highlighted new and emerging specialty medications and their potential impact on the managed care market.

There has been significant development of therapeutics to treat cancer. The cancer death rate has declined by 25% since 1991, and cancer is now be treated as a chronic condition. So far this. year, the FDA has approved 16 novel cancer drugs and five more are expected to be approved before the end of the year. The average annual cost of novel cancer drugs approved this year is $225,000. Orphan drug development is also growing, with more than half (52%) of novel drugs in development indicated for orphan designations.

Nearly half (48%) of per-member, per-year (PMPY) drug spend under the pharmacy benefit is for specialty medications. Last year, specialty trends were 11.6% compared with -5.0% for traditional drugs. “We expect that 2020 will be the year that spend on specialty medications will account for over half of pharmacy drug spend,” said Dr. Tharaldson, noting that only 3% of patients take specialty medications but they are much more expensive.

Leading specialty therapy classes, per 2019 PMPY spend, are inflammatory conditions, cancer, HIV, and multiple sclerosis. In 2018, the FDA approved a “record” number of specialty drugs—39 novel approvals—and this year, 28 novel specialty drugs have been approved, with 10 more expected before the end of the year, so “we’re going to come close to tying that 2018 record,” she said.

So far this year, some of the approved specialty approvals include:

  • Teprotumumab-trbw for thyroid eye disease (costs $340,000 for treatment course)
  • Istuximab-irfc for multiple myeloma (MM; costs $135,000 per year)
  • Ozanimod for multiple sclerosis (MS; costs $86,000 per year)
  • Tucatinib for breast cancer
  • Sacituzumab govitecan-hziy for breast cancer
  • Capmatinib for non-small cell lung cancer (NSCLC)
  • Selpercatinib for lung and thyroid cancer
  • Fostemsavir for HIV-1
  • Brexucabtagene autoleucel for mantle cell lymphoma (costs $373,000)
  • Tafasitamab-cxix for diffuse large B-cell lymphoma
  • Belantamab mafodotin-blmf for MM
  • Risdiplam for spinal muscular atrophy (costs $340,000 annually)
  • Viltolarsen for Duchenne muscular dystrophy (cost about $750,000 per year)

Dr. Tharaldson then gave snapshot of the specialty pharmacy pipeline. Some highlights include:

  • Inflammatory conditions
    • Upadacitinib for psoriatic arthritis: expected approval in April 2021
    • Filgotinib for rheumatoid arthritis: expected approval in late 2021
    • Ozanimod for ulcerative colitis: expected approval in 2021
    • Several drugs indicated for atopic dermatitis, including:
      • Four JAK inhibitors could be approved in 2021: abrocitinib, upadacitinib, baricitinib, and ruxolitinib
  • MS
    • Ponesimod: an oral once-daily agent with expected approval in March 2021
    • Ublituximab: an intravenous agent with expected approval in 2022
    • Evobrutinib: an oral twice-daily agent with expected approval in 2024
  • Cancer
    • Five more therapies are expected to be approved in 2020:
      • Lisocabtagene maraleucel for large B-cell lymphoma could be approved in Nov. 2020
      • Naxitamab for neuroblastoma could be approved in Nov. 2020
      • Margetuximab for breast cancer could be approved in Dec. 2020
      • Relugolix for prostate cancer could be approved in Dec. 2020
      • Melphalan flufenamide for MM could be approved in Dec. 2020
    • A number of therapies are expected to be approved in 2021:
      • Dostarlimab for endometrial cancer could be approved in Jan. 2021
      • Trilaciclib for small cell lung cancer could be approved in Feb. 2021
      • Tepotinib for NSCLC could be approved in Feb. 2021
      • Idecabtagene vicleucel could be approved for MM in March 2021
  • HIV
    • Two agents are expected to be approved in early 2021: cabotegravir (oral) and cabotegravir/rilpivirine (intramuscular)
    • Leronlimab, a subcutaneous agent is expected later in 2021
  • Nonalcoholic steatohepatitis
    • Obeticholic acid is expected in 2021 or beyond
    • Cenicriviroc, resmetirom, and tropifexor are expected in 2022 or beyond
    • Nine other agents are in development with expected approvals in 2023 and 2024 and beyond
  • Alzheimer’s disease
    • Three anti-amyloid beta antibodies (immunotherapies) are in development: aducanumab (expected in 2021), BAN2401 (expected in 2022), and gantenerumab (expected in 2023
    • Three anti-tau antibody agents are in developing, with expected approvals in 2024
  • High cholesterol
    • Two drugs are pending approval:
      • Inclisiran, expected in Dec. 2020
      • Evinacumab, expected in Feb. 2021
  • Duchenne muscular dystrophy
    • Casimersen, expected in Feb. 2021
    • Two gene therapies may reach the market: SRP-9001 (2021 and beyond) and fordadistrogene movaparvovec (2023 and beyond)
  • Hemophilia
    • A number of gene therapies (expected to cost between $2 million and $3 million) are in development (one-time intravenous infusions):
      • Etranacogene dezaparvovec (expected in 2021)
      • Fidanacogene elaparvovec (expected in 2022)
      • Giroctocogene fitelparvovec (expected in 2022)
  • Miscellaneous orphan drug categories
    • Sutimlimab for cold agglutinin disease could be approved in Nov. 2020
    • Lonafarnib for Hutchinson-Gilford progeria syndrome could be approved in Nov. 2020
    • Lumasiran for primary hyperoxaluria type 1 disease could be approved in Dec. 2020
    • Pegunigalsidase alfa for Fabry disease could be approved in Jan. 2021
    • Arimoclomol for Niemann–Pick disease type C could be approved in March 2021
    • Vosoritide for achondroplasia could be approved in Aug. 2021

Presentation: P1 Specialty Pharmaceuticals in Development.AMCP Nexus 2020 Virtual.