An Overview of the Specialty Therapy Pipeline in 2023

To kick off the first business session of Asembia 2023, Ray Tancredi, divisional vice president at Walgreens, noted 6 major trends in the 2023 specialty therapy pipeline:

• Innovation
• Gene and cell therapy
• Oncology
• Patient centricity
• Biosimilars
• Rare disease

Tancredi noted that specialty pharmacies grew by 315% between 2015 and 2021, with hospital or health system-owned specialty pharmacies accounting for about a third of this total growth—and that the specialty drug pipeline is projected to grow by another 8% each year through 2025.

“Specialty pharmacy continues to carry the FDA pipeline year over year,” Tancredi said. “What I saw was about 39 more drugs that could potentially be approved by the end of 2023, and of those 39, I think 28 of them could be considered specialty.”

He first listed 8 major recent specialty drug approvals that “have the potential to change the standard of care in a given disease and/or have a large economic impact to payer.” These were:

• Spesolimab-sbzo injection (Spevigo) for pustular psoriasis flares
• Efgartigimod alfa-fcab (Vyvgart) for myasthenia gravis
• Olipudase alfa-rpcp (Xenpozyme) for non-central nervous system manifestations of acid sphingomyelinase deficiency
• Deucravacitinib (Sotyktu) for moderate-to-severe plaque psoriasis
• Fam-trastuzumab deruxtecan-nxki (Enhertu) for unresectable or metastatic HER2-low breast cancer
• Mavacamten (Camzyos) for class II or III obstructive hypertrophic cardiomyopathy
• Lecanemab-irmb (Leqembi) for Alzheimer disease
• Zanubrutinib (Brukinsa) for chronic lymphocytic leukemia or small lymphocytic lymphoma

Looking to the rest of 2023, there are several more specialty drugs anticipated to receive FDA approval. Here is a wrapped-up version of these anticipated approvals.

Rare and Orphan Disease

Tancredi focused on 23 drugs in the pipeline to treat a large variety of rare and orphan diseases. There are more than 7000 rare diseases, which affect fewer than 200,000 individuals per condition and 30 million Americans overall. Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of treatments for rare diseases, though most rare diseases still do not have FDA-approved treatments.

This list included 11 oral drugs and 12 drugs delivered via injection. All drugs are in phase 2 or 3 of research and development, or filed for FDA approval.

The orally administrated drugs on this list include:

• Mavorixafor for WHIM syndrome
• Nirogacestat for desmoid tumors
• Sodium oxybate for narcolepsy
• Alvelestat for alpha 1 antitrypsin congenital deficiency
• Vamorolone for Duchenne muscular dystrophy
• Govorestat for galactosemia
• Elafibranor for primary biliary cirrhosis
• Sparsentan for IgA nephropathy
• Paltusotine for acromegaly
• Leniolisib for activated phosphoinositide 3-kinase delta syndrome
• Trofinetide for Rett syndrome

The drugs delivered via injection on this list include:

• Concizumab for hemophilia A and B
• Tilavonemab for Alzheimer disease
• Cendakimab for eosinophilic esophagitis
• Magrolimab for myelodysplastic syndrome
• Acaziolcept for systemic lupus erythematosus
• Tegoprubart for amyotrophic lateral sclerosis (ALS)
• Fitusiran for hemophilia A and B
• Tofersen sodium for ALS
• Sotatercept for pulmonary arterial hypertension
• Narsoplimab for thrombosis
• Diazoxide choline for Prader-Will syndrome
• Nipocalimab for hemolytic disease of the fetus and newborn

Specialty Drugs

Tancredi focused on 19 traditional specialty drugs in the pipeline for FDA approval, including 10 oral drugs, 8 drugs delivered via injection, and 1 delivered via inhalation.

The orally-administrated drugs on this list include:

• Resmetirom for nonalcoholic steatohepatitis
• Ritlecitinib for alopecia
• Danicopan for paroxysmal nocturnal hemoglobinuria
• Islatravir hydrate for HIV
• Tolebrutinib for relapsing remitting multiple sclerosis, with potential indication for myasthenia gravis
• Etrasimod arginine for ulcerative colitis
• Remibrutinib for chronic spontaneous urticaria
• Bemnofosbuvir for hepatitis C
• Omecamtiv mecarbil for chronic heart failure
• Evobrutinib for multiple sclerosis

The drugs delivered via injection on this list include:

• Ziltivekimab for cardiovascular disease
• Nirsevimab for respiratory syncytial virus
• Mirikizumab for ulcerative colitis
• Brazikumab for Crohn’s disease
• Itepekimab for chronic obstructive pulmonary disease
• Lebrikizumab for eczema and atopic dermatitis
• Avacincaptad pegol for geographic atrophy
• Donanemab for Alzheimer disease

Additionally, treprostinil sodium was the only drug on this list delivered via inhalation.

Oncology

Tancredi noted that about 13 oncology drugs are approved each year. In 2023, there are 16 oral drugs and 6 drugs delivered via injection in the pipeline, with 6 drugs in the pipeline for breast cancer and 3 for non-small cell lung cancer (NSCLC).

The orally-administrated drugs on this list include:

• Veliparib for breast cancer
• Tovorafenib for breast cancer
• Ipatasertib for prostate cancer
• Giredestrant for breast cancer
• Momelotinib for myelofibrosis
• Repotrectinib for NSCLC
• Imlunestrant for breast cancer
• Navitoclax dihydrochloride for myelofibrosis
• Elacestrant for breast cancer
• Bezuclastinib for gastrointestinal stromal tumors
• Capivasertib for breast cancer
• Milademetan for soft tissue sarcoma
• Bemcentinib for myelodysplastic syndrome
• Pirtobrutinib for non-Hodgkin lymphoma
• Sitravatinib for NSCLC
• Xl092 for colorectal cancer

The drugs delivered via injection on this list include:

• Vusolimogene oderparepvec for cutaneous squamous cell carcinoma
• Talquetamab for multiple myeloma
• Sabatolimab for chronic myelomonocytic leukemia
• Epcoritamab for non-Hodgkin lymphoma
• Tiragolumab for esophageal cancer
• Tusamitamab ravtansine for NSCLC

Gene and Cell Therapies

There are 13 injectable gene and cell therapies in the pipeline, with 1 drug delivered topically.

The gene and cell therapies delivered via injection on this list include:

• Tabelecleucel for Epstein-Barr virus
• Factor VIII for hemophilia A, developed by Bayer
• Dcvax-L for glioblastoma
• Valoctocogene roxaparvovec for hemophilia A
• Delandistrogene moxeparvovec for Duchenne muscular dystrophy
• Fidanacogene elaparvovec for hemophilia B
• FCR001 for scleroderma
• DTX401 for glycogen storage disease type 1a
• Posoleucel for Epsteinn-Barr virus
• Exagamglogene autotemcel for sickle cell disease
• Fordadistrogene movaparvovec for Duchenne muscular dystrophy
• Aglatimagene besadenovec for prostate cancer
• Factor VIII for hemophilia A, developed by Roche

Beremagene geperpavec is also in the pipeline for topical treatment of dystrophic epidermolysis bullosa.