An Overview of the Specialty Remedy Pipeline in 2023

To kick off the primary enterprise session of Asembia 2023, Ray Tancredi, divisional vp at Walgreens, famous 6 main developments within the 2023 specialty remedy pipeline:

• Innovation
• Gene and cell remedy
• Oncology
• Affected person centricity
• Biosimilars
• Uncommon illness

Tancredi famous that specialty pharmacies grew by 315% between 2015 and 2021, with hospital or well being system-owned specialty pharmacies accounting for a few third of this complete development—and that the specialty drug pipeline is projected to develop by one other 8% annually via 2025.

“Specialty pharmacy continues to hold the FDA pipeline yr over yr,” Tancredi mentioned. “What I noticed was about 39 extra medication that would doubtlessly be authorized by the tip of 2023, and of these 39, I believe 28 of them may very well be thought-about specialty.”

He first listed 8 main latest specialty drug approvals that “have the potential to alter the usual of care in a given illness and/or have a big financial affect to payer.” These have been:

• 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 most cancers
• Mavacamten (Camzyos) for sophistication II or III obstructive hypertrophic cardiomyopathy
• Lecanemab-irmb (Leqembi) for Alzheimer illness
• Zanubrutinib (Brukinsa) for power lymphocytic leukemia or small lymphocytic lymphoma

Seeking to the remainder of 2023, there are a number of extra specialty medication anticipated to obtain FDA approval. Here’s a wrapped-up model of those anticipated approvals.

Uncommon and Orphan Illness

Tancredi targeted on 23 medication within the pipeline to deal with a big number of uncommon and orphan ailments. There are greater than 7000 uncommon ailments, which have an effect on fewer than 200,000 people per situation and 30 million People total. For the reason that Orphan Drug Act was signed into regulation in 1983, the FDA has authorized a whole bunch of therapies for uncommon ailments, although most uncommon ailments nonetheless should not have FDA-approved therapies.

This record included 11 oral medication and 12 medication delivered by way of injection. All medication are in section 2 or 3 of analysis and growth, or filed for FDA approval.

The orally administrated medication on this record embody:

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

The medication delivered by way of injection on this record embody:

• Concizumab for hemophilia A and B
• Tilavonemab for Alzheimer illness
• 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 illness of the fetus and new child

Specialty Medication

Tancredi targeted on 19 conventional specialty medication within the pipeline for FDA approval, together with 10 oral medication, 8 medication delivered by way of injection, and 1 delivered by way of inhalation.

The orally-administrated medication on this record embody:

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

The medication delivered by way of injection on this record embody:

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

Moreover, treprostinil sodium was the one drug on this record delivered by way of inhalation.

Oncology

Tancredi famous that about 13 oncology medication are authorized annually. In 2023, there are 16 oral medication and 6 medication delivered by way of injection within the pipeline, with 6 medication within the pipeline for breast most cancers and three for non-small cell lung most cancers (NSCLC).

The orally-administrated medication on this record embody:

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

The medication delivered by way of injection on this record embody:

• Vusolimogene oderparepvec for cutaneous squamous cell carcinoma
• Talquetamab for a number of myeloma
• Sabatolimab for power myelomonocytic leukemia
• Epcoritamab for non-Hodgkin lymphoma
• Tiragolumab for esophageal most cancers
• Tusamitamab ravtansine for NSCLC

Gene and Cell Therapies

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

The gene and cell therapies delivered by way of injection on this record embody:

• Tabelecleucel for Epstein-Barr virus
• Issue 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 illness sort 1a
• Posoleucel for Epsteinn-Barr virus
• Exagamglogene autotemcel for sickle cell illness
• Fordadistrogene movaparvovec for Duchenne muscular dystrophy
• Aglatimagene besadenovec for prostate most cancers
• Issue VIII for hemophilia A, developed by Roche

Beremagene geperpavec can be within the pipeline for topical remedy of dystrophic epidermolysis bullosa.