Drugs for Alzheimer Disease, Hemophilia, NASH, and More Pending Approval in Specialty Pipeline


Attendees at an AMCP Nexus 2023 session received an overview of FDA approvals granted in 2023 as well as a preview of nononcology drugs to be released over the next 5 years.

At the Academy of Managed Care Pharmacy (AMCP) Nexus meeting, Aimee Tharaldson, PharmD, previewed the major nononcology drugs likely to be approved by the FDA over the next 5 years, including more medications for Alzheimer disease, hemophilia, and nonalcoholic steatohepatitis (NASH).

Tharaldson began her session with an overview of the market, including the growth of the biosimilar market in 2023, with the launch of 8 products referencing Humira (adalimumab) and the approval of the first tocilizumab and natalizumab biosimilars. She also provided an overview of the drugs the FDA has approved so far this year, which included 2 products for diffuse large B-cell lymphoma, Leqembi (lecanemab-irmb) for Alzheimer disease, 2 medications and a stem cell transplant for multiple myeloma, and several products for orphan conditions (Table).


In the inflammatory space, 7 products are expected over the next couple of years, including Celltrion Healthcare’s subcutaneous infliximab product (Remsima SC) for ulcerative colitis (UC) and Crohn disease (CD), which is expected to be approved by the end of October 2023. Additionally, the companies’ behind risankizumab-rzaa (Skyrizi), vedolizumab (Entyvio), and secukinumab (Cosentyx) are seeking expanded indications for UC, CD, and hidradenitis suppurativa, respectively.

Multiple Sclerosis and HIV

Tharaldson noted several medications coming for multiple sclerosis, including evobrutinib, fenebrutinib, orelabrutinib, and tolebrutinib. There is also a subcutaneous version of ocrelizumab (Ocrevus) in the pipeline.

Rilpivirine and islatravir are 2 HIV treatments pending FDA approval.


Eleven medications are currently in the pipeline for NASH, including resmetirom, belapectin, efinopegdutide, and firsocostat. Semaglutide, which is marketed as Wegovy for obesity and Ozempic for diabetes, is also in the pipeline.

TERN-501 is a thyroid hormone receptor β-selective agonist currently being investigated in phase 2 trials. Most of the NASH pipeline is expected to be approved in 2026.

Alzheimer Disease

Although 1 product has already been approved this year, donanemab is expected to receive FDA approval in December 2023. Three other products (posinemab, semorinemab, and E2814) are projected to be approved between 2026 and 2027.


Paroxysmal nocturnal hemoglobinuria (PNH) and Duchenne muscular dystrophy (DMD) each have 3 products in the pipeline. PNH is a rare blood disorder involving the immune system attacking red blood cells and platelets, and DMD is an inherited disorder characterized by progressive muscular weakness.


Tharaldson noted 6 major hemophilia products coming down the pipeline, including fidanacogene elaparvovex, which is a cell and gene therapy. The list also contains concizumab, dirloctocogene samoparvovec, marstacimab, Mim8, and giroctocogene fitelparvovec.

Gene Therapies

So far, the FDA has approved 17 gene and cell therapies, about half of which are indicated for cancer. In addition to fidanacogene elaparvovec, 4 other gene therapies are pending FDA approval, including:

  • Exagamglogene autotemcel—sickle cell disease and β-thalassemia
  • Lovotibelglogene autotemcel—sickle cell disease
  • Lifileucel—advanced melanoma
  • Atidarsagene autotemcel—metachromatic leukodystrophy

Other Specialty Drugs Pending Approval

Tharaldson ended her presentation noting 4 products that are expected to be approved by the end of 2023. If approved, cinaxadamtase alfa will be used to treat congenital thrombotic thrombocytopenia purpura, zilucoplan will treat generalized myasthenia gravis, eplontersen will help patients with hereditary transthyretin-mediated amyloid polyneuropathy, and sotatercept will treat pulmonary arterial hypertension.

This article originally appeared on AJMC.

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