Specialty Drug Pipeline Is Poised to Offer Myriad New Therapies Next Year

Specialty Pharmacy TimesOctober 2020
Volume 3
Issue 3

The specialty pipeline continues to grow at a rapid pace with a number of FDA approvals in 2019 for either new therapies or first in class for certain conditions, and many more drugs in development.

The specialty drug pipeline is coming off a strong year. A number of FDA approvals in 2019 were for either the first new therapies or the first in class approved for certain conditions, and the specialty pipeline continues to grow at a rapid pace.1

These recent developments for specialty pharmacy were discussed during a session of the National Association of Specialty Pharmacy (NASP) 2020 Annual Meeting & Expo Virtual Experience. During the session, presenter Ray Tancredi, MBA, RPh, CSP, divisional vice president, specialty pharmacy development and brand, RX/vaccine purchase at Walgreen Company, said 27 of the 49 new drugs and biologics approved by the FDA in 2019 were considered specialty products.1

“We saw 59 in 2017 and 63 new drugs in 2018, and there’s no signs of slowing. We’re going to see an even greater explosion of drugs over the next few years,” Tancredi said.1

“It’s important to look back at some of the blockbusters and game-changing therapies that were approved that came to market in 2019,” he added.

New medications were approved as the first treatments for some rare diseases, according to Tancredi.1 These drugs included the gene therapy onasemnogene abeparvovec-xioi (Zolgensma; AveXis) for the treatment of children aged less than 2 years with spinal muscular atrophy (SMA)1,2; crizanlizumab (Adakveo; Novartis) for reducing the frequency of vaso-occlusive crises, or pain crises associated with sickle cell disease1,3; and givosiran (Givlaari; Alnylam) for adult patients with acute hepatic porphyria, a genetic condition that results in the buildup of toxic porphyrin molecules.1,4

Other medications approved in 2019 provided new therapeutic options for hard-to-treat diseases. These first-in-class drugs included the triple combination of elexacaftor, ivacaftor, and tezacaftor (Trikafta; Vertex) for patients aged 12 years and older with cystic fibrosis and the F508 del mutation in at least 1 copy of the CFTR gene;1,5 darolutamide (Nubeqa; Bayer) for men with nonmetastatic castrationresistant prostate cancer;1 and the oral medication selinexor (Xpovio; Karyopharm), which is indicated in combination with the corticosteroid dexamethasone for treating adult patients with relapsed/refractory multiple myeloma who have received at least 4 prior therapies and whose disease is resistant to several other forms of treatment.1,6

In his presentation, Tancredi discussed several specialty drugs that are still in the pipeline. He broke these drugs into 4 categories: rare and orphan, specialty pipeline, gene therapy, and oncology.

Rare and Orphan

“Over the past couple of years, the FDA has committed to responding to all the orphan drug applications within 90 days,” Tancredi said. “There are a number of products in the rare and orphan space.”

Many of the pending applications for rare and orphan therapies are in phase 3 of development and hold orphan drug designation from the FDA. They include as follows:

  • Maribavir (Camvia; Takeda), an oral drug indicated for cytomegalovirus infections.
  • Berotralstat (BCX-7353; BioCryst), an oral kallikrein inhibitor for hereditary angioedema. The manufacturer has filed a new drug application and the FDA has given the product orphan drug designation, according to Tancredi.
  • Ataluren (Translarna; PTC Therapeutics), an oral transcription modulator for Duchenne muscular dystrophy (DMD), a condition with a progressive loss of muscle strength.
  • Adrabetadex (VTS-270; Mallinckrodt), an intrathecal injection and cholesterol modulator for the treatment of Niemann-Pick type C (NPC) disease. “Manifestations of this disease are typically onset in childhood. NPC is typically fatal, and the majority of cases lead to death before the age of 20,” Tancredi said.
  • Tralokinumab (Leo Pharma), a subcutaneous (SC) injection in the anti—IL-13 monoclonal antibody (mAb) class, for atopic dermatitis. “Leo entered into an agreement with AstraZeneca in 2016 to secure global rights to tralokinumab,” Tancredi said. “Leo anticipates a potential 2021 launch for their product that will compete against Dupixent [dupilumab] by Sanofi.”
  • Risdiplam (Evrysdi; Genentech), an oral SMN2 splicing modifier, received FDA approval in August 2020 for SMA in adults and children aged 2 months and older.1,7
  • Avalglucosidase alfa (GZ402666; Sanofi), an intravenous (IV) neo α-glucosidase for treatment of Pompe disease. “We could see a launch in the next year or so,” Tancredi said.
  • Efgartigimod (Argenx), an SC IV mAb biologic for myasthenia gravis (MG). Also in development is rozanolixizumab (UCB), an SC IV antineonatal Fc receptor mAb for treatment of MG.
  • Bis-choline-tetrathiomolybdate (Decuprate; Alexion), an SOD1 inhibitor for Wilson disease.
  • Fitusiran (Fitusiran; Sanofi and Alnylam), an antithrombin SC injection for hemophilia A and B. “It works by resetting the clotting cascade to stop the bleeding by silencing antithrombin,” Tancredi said.
  • Gaboxadol (OV101; Ovid and Lundbeck), an oral capsule taken once daily at bedtime for Angelman syndrome (AS), a rare, genetic neurodevelopmental disease. This drug is an extrasynaptic GABAA receptor agonist that could be the first FDA-approved medication for AS, according to Tancredi.
  • Edasalonexent (Edasalonexent; Catabasis), another oral drug for Duchenne muscular dystrophy (DMD). “It was designed to benefit all adolescent males who are affected, regardless of the mutation type,” Tancredi said.

Specialty Pipeline

These drugs represent some of the more traditional specialty products. According to Tancredi, some of these products are being developed for disease states that are familiar to pharmacists, and others are in the pipeline for new indications. They include as follows:

  • Ponesimod (Johnson & Johnson), an oral sphingosine-1-phosphate agonist for multiple sclerosis (MS). “There are probably about 20 products in the marketplace today for MS, but we still have some interesting drugs that continue to come to market,” Tancredi said.
  • Bimekizumab (UCB), an SC IV anti—IL-17A/F mAb for the treatment of psoriasis and psoriatic arthritis.
  • Cenicriviroc mesylate (Allergan and Takeda), an oral CCR2/5 dual antagonist for nonalcoholic steatohepatitis.
  • Apomorphine hydrochloride (Apomorphine Sublingual Film; Sumitomo Dainippon Pharma), a transmucosal dopamine D2 agonist for Parkinson disease.
  • Aducanumab (Biogen), an IV infusion anti—β- amyloid mAb for treatment of Alzheimer disease. “Biogen’s aducanumab will be the first disease-modifying Alzheimer therapy,” Tancredi said.
  • Mirikizumab (Eli Lilly), an SC IV IL-23 antibody with investigational indications for psoriasis, ulcerative colitis, and Crohn disease.

Gene Therapy

Gene therapy mechanisms replace a diseasecausing gene with a healthy copy of the gene, inactivate a disease-causing gene that is not properly functioning, and introduce a new or modified gene into the body to treat a disease. “The pipeline for gene therapy continues to advance with potential cures for diseases in a single treatment,” Tancredi said. “There are a number of products on the horizon.” These potential therapies include :

  • RGX-501 (Regenxbio), an injection currently in phase 2 of development for the treatment of familial hypercholesterolemia.
  • Micro-dystrophin program (SRP-9001; Sarepta Therapeutics), a gene therapy injection for DMD. “Hopefully, we’ll see this product in the next couple of years,” Tancredi said.
  • AMT-061 (UniQure), an injection for hemophilia B. UniQure is currently enrolling patients in a phase 3 trial, according to Tancredi.
  • VY-AADC01 (Voyager Therapeutics), an intracerebral injection for Parkinson disease.
  • ABO-102 (Abeona Therapeutics), an injection currently in phase 2 for Sanfilippo syndrome. “It involves a 1-time therapy delivery of a normal copy of defective gene cells to the central nervous system,” Tancredi said.


Drug products are in the oncology pharmacy pipeline for various forms of cancer, such as breast cancer, acute myeloid leukemia (AML), non—small cell lung cancer (NSCLC), and biliary cancer. These investigational products include:

  • Guadecitabine (Otsuka Holdings), a DNA methyltransferase inhibitor for AML and myelodysplastic syndrome with an SC injection delivery. The drug is currently in phase 3 development for these indications as well as phase 2 trial stage for ovarian cancer, according to Tancredi.
  • Napabucasin (Sumitomo Dainippon), an oral STAT3 inhibitor for colorectal cancer. “It targets malignant cancer stem cells that are responsible for malignancy metastatic growth and recurrence,” Tancredi said.
  • Relatlimab (Bristol Myers Squibb), an anti- LAG3 injection for melanoma, and renal cell cancer. This drug is also being investigated with other therapies for potential additional oncology indications, Tancredi said.

Tancredi also addressed additional drug products in the pipeline for all 4 categories during the presentation.


  • Tancredi R. Presented at: National Association of Specialty Pharmacy (NASP) 2020 Annual Meeting & Expo Virtual Experience; [online], September 17, 2020.
  • FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. News release. FDA. May 24, 2019. Accessed August 18, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease
  • New Novartis medicine o (crizanlizumab) approved by FDA to reduce frequency of pain crises in individuals living with sickle cell disease. News release. Novartis. November 15, 2019. Accessed August 18, 2020. https://www.novartis.com/news/media-releases/new-novartis-medicine-adakveo-crizanlizumab-approved-fda-reduce-frequency-pain-crises-individuals-living-sickle-cell-disease
  • FDA approved first treatment for inherited rare disease. News release. FDA. November 20, 2019. Accessed August 18, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-inherited-rare-disease
  • FDA approved new breakthrough therapy for cystic fibrosis. News release. FDA. October 21, 2019. Accessed September 18, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-new-breakthrough-therapy-cystic-fibrosis
  • FDA approves new treatment for refractory multiple myeloma. News release. FDA. July 3, 2019. Accessed September 18, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-refractory-multiple-myeloma
  • FDA Approves Genentech’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older. News release. Genentech. August 7, 2020. Accessed September 18, 2020. https://www.gene.com/media/press-releases/14866/2020-08-07/fda-approves-genentechs-evrysdi-risdipla

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