Key Market Trends for the Current Specialty Drug Pipeline

Although specialty drug approvals have been on the rise, there is still work to be done in the specialty field, specifically in rare diseases and for nonalcoholic steatohepatitis (NASH). During the keynote session at the Academy of Managed Care Pharmacy Nexus 2023 conference, Aimee Tharaldson, PharmD, clinical program principal of Emerging Therapy at Evernorth, discussed key market trends in specialty drugs and medications that are currently in the pipeline.

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“Specialty trends are a lot higher than traditional trends at 8.7% compared to 3.4%, but it is important to know that less than 2% of [individuals] take specialty medications, but of course, they’re very expensive, costing on average $3500 a month,” Tharaldson said in the session.

Tharaldson identified the key market trends for specialty medications in 3 categories: biosimilars, cancer drugs, and orphan drugs. The leading classes for specialty therapies include inflammatory conditions, cancer, HIV, and multiple sclerosis (MS).

There are currently 43 biosimilars approved for 13 biologics with 37 for 10 biologics on the market, according to Tharaldson. There are also 8 biosimilars for adalimumab (Humira; AbbVie) currently on the market, and etanercept (Enbrel; Amgen) will have exclusivity until 2029. Etanercept will be a drug to watch in addition to tocilizumab (Actemra; Genentech), golimumab (Simponil; Janssen Immunology), and ustekinumab (Stelara; Janssen Immunology), according to Tharaldson.

Orphan drugs are another key specialty market trend, according to Tharaldson. She added that when looking at novel specialty drugs that are pending approval, two-thirds are for orphan conditions.

“Last year, the FDA approved 32 specialty drugs and 9 traditional medications, and so far, this year, as of yesterday, 35 novel specialty drugs have been approved, already so far this year, with 16 more expected before the end of the year. We can have 51 specialty approvals this year, which would beat the previous record by 11,” Tharaldson said in the session.

Tharaldson also discussed 10 specialty pipeline therapy classes of interest: inflammatory conditions, MS, HIV, NASH, Alzheimer disease (AD), paroxysmal nocturnal hemoglobinuria (PNH), Duchenne muscular dystrophy (DMD), hemophilia, and gene and cell therapies.

Following today’s approval of bimekizumab (UCB), other drugs for inflammatory conditions in the pipeline include infliximab (Celltrion), mirikizumab (Eli Lilly), and secukinumab (Cosentyx; Novartis) in quarter 4 of 2023. Risankizumab-rzaa (Skyrizil AbbVie), vedolizumab (Entyvio; Takeda), and lebrikizumab (Eli Lilly) are expected in 2024.

For MS, there are 6 drugs in the pipeline, with half expected in 2024 and half in 2025, including glatiramer acetate, GA depot (Viatris, Mapi Pharma), ocrelizumab (Ocrevus SC; Genentech), evobrutinib (EMD Serono), fenebrutinib (Genentech), orelabrutinib (Biogen), and tolebrutinib (Sanofi).

There are not a lot of drugs in the pipeline for HIV, according to Tharaldson, with only 2 in the pipeline currently: rilpivirine (Edurant; Jansen), expected in May 2024, and islatravir (Merck), expected as early as 2025.

Currently, NASH has limited treatment options and a variety of comorbidities, including high cholesterol, type 2 diabetes, insulin resistance, and obesity. However, the pipeline currently includes 11 drugs, with approval potentially ranging from 2024 to 2027. The drugs include resmetirom (Madrigal Pharmaceuticals), aramchol (Galmed Pharmacuticals), belapectin (Galectin therapeutics), denifanstat (Sagimet Biosciences), efinopegdutide (Merck), efruxifermin (Akero), semaglutide (Novo Nordisk), TERN-501 (Terns Pharmaceuticals), VK2809 (Viking Therapeutics), cilofexor (Gilead), and firsocostat (Gilead).

“NASH has the potential to be a huge market, but there’s still a lot of unknowns. We don’t yet have the data to know the long-term benefits by actually decreasing the risk that the patient will progress to cirrhosis of liver transplant,” Tharaldson said.

AD has 4 potential therapeutics in the pipeline with donanemab (Eli Lily) expected in December 2023, E2814 (Eisai) in 2026, and posdinemab (Janssen) and semorinemab (Genentech) expected in 2027. PNH has 3 drugs currently in the pipeline: iptacopan (Novartis) expected in December 2023, and crovalimab (Genentech) and danicopan (AstraZeneca) expected in 2024.

DMD also has 3 drugs in the pipeline with 2 expected by the end of 2023: vamorolone (Santhera, ReveraGen) and givinostate (Italfarmaco SpA). Fordadistrogene movaparvovec (Pfizer) is expected in 2024.

In the hemophilia pipeline, there are fidanacogene elaparvovec (Pfizer), concizumab (Novo Nordisk), diroctocogene samoparvovec (Genentech), marstacimab (Pfizer), and Mim8 (Novo Nordisk). Giroctocogene fitelparvovec (Sangamo, Pfizer) is expected in 2025. Fidanacogene elaparvovec, diroctocogene samoparvovec, and giroctocogene fitelparvovec are gene therapies.

Other gene and cell therapies in the pipeline expected in 2023 are exagamglogene autotemcel (Vertex, CRISPR) and lovotibeglogene autotemcel (bluebird bio), which are seeking indications for sickle cell disease. For 2024, gene and cell therapies include lifileucel (Iovance Biotherapeutics) for advanced melanoma and atidarsagene autotemcel (Orchard Therapeutic) for metachromatic leukodystrophy.

Tharaldson concluded the presentation with 4 select specialty drugs that are currently in the pipeline. Cinaxadamtase alfa (Takeda) for congenital thrombotic thrombocytopenic purpura, expected in November 2023; eplontersen (Ionis, AstraZeneca) for hereditary transthyretin-mediated amyloid polyneuropathy expected in December 2023; and sotatercept (Merck) for pulmonary arterial hypertension expected on March 26, 2024. Zilucoplan (UCB) was originally planned for approval in December 2023, but was approved early, on October 17, 2023.

Reference

Tharaldson A. Specialty Pharmaceuticals in Development. AMCP Nexus 2023. Orlando, Florida. October 18, 2023. Accessed October 18, 2023.