Specialty Drug Pipeline Poised to Offer Many New Therapies in the Coming Year


There were a number of FDA approvals in 2019 that were either the first new therapies or first in class approved for certain conditions, and the specialty pipeline is continuing to grow at a rapid pace.

The specialty drug pipeline is coming off a strong year. There were a number of FDA approvals in 2019 that were either the first new therapies or first in class approved for certain conditions, and the specialty pipeline is continuing 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 and Expo Virtual Experience. During the session, presenter Ray Tancredi, RPh, MBA, CSP, divisional vice president, specialty pharmacy development and brand, RX/Vaccine purchase, 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 disease;1,3 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 conditions. 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 at least 1 copy of the F508del mutation;1,5 darolutamide (Nubeqa, Bayer) for men with nonmetastatic castration-resistant prostate cancer;1 and the oral medication selinexor (Xpovio), 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 last couple of years, the FDA has committed to responding to all of 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:

  • Maribavir (Camvia, Takeda), an oral drug indicated for cytomegalovirus infections.
  • Berotralstat (BCX-7353, BioCryst), an oral kallikrein inhibitor for hereditary angioedema. A New Drug Application has been filed and the product has been designated as an orphan drug by the FDA, 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.
  • VTS-270 (Adrabetadex, 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 (Tralokinumab, Leo Pharma), a subcutaneous (SC) injection in the anti-IL-13 monoclonal antibody 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 survival motor neuron 2 gene splicing modifier was approved by the FDA in August 2020 for SMA in adults and children aged 2 months and older.1,7
  • Avalglucosidase Alfa (GZ402666, Sanofi), an intravenous (IV) neo alpha glucosidase for treatment of Pompe disease. “We could see a launch in the next year or so,” Tancredi said.
  • Efgartigimod (Efgartigimod, Argenx), an SC IV monoclonal antibody biologic for myasthenia gravis (MG). Also is development is rozanolixizumab (Rozanolixizumab, UCB), an SC IV antineonatal Fc receptor MAb for treatment of MG.
  • Bis-choline-tetrathiomolybdate (Decuprate, Alexion), an oral superoxide dismutase 1 inhibitor for Wilson disease.
  • Fitusiran (Fitusiran, Sanofi & Alnylam), an antithrombin SC injection for hemophilia A and B. “It works be re-setting the clotting cascade to stop the bleeding by silencing antithrombin,” Tancredi said.
  • Gaboxadol (OV101, Ovid & Lundbeck), an oral capsule taken once a day 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 that 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 other products are in the pipeline for new indications. They include:

  • Ponesimod (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 (Bimekizumab, UCB), an SC IV anti-IL-17A & F Mab for the treatment of psoriasis and psoriatic arthritis.
  • Cenicriviroc mesylate (Cenicriviroc, Allergan & Takeda), an oral chemokine receptor type 2/5 dual antagonist for nonalcoholic steatohepatitis.
  • Apomorphine hydrochloride (Apomorphine Sublingual Film, Sumitomo Dainippon Pharma), a transmucosal dopamine D2 agonist for Parkinson disease.
  • Aducanumab (Aducanumab, Biogen) an IV infusion anti-beta amyloid monoclonal antibody for treatment of Alzheimer disease. “Biogen’s aducanumab will be the first disease-modifying Alzheimer therapy,” Tancredi said.
  • Mirikizumab (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 disease-causing 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 (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 (AMT-061, UniQure), an injection for hemophilia B. UniQure is currently enrolling patients in a phase 3 trial, according to Tancredi.
  • VY-AADC01 (VY-AADC01, Voyager Therapeutics), an intracerbral injection for Parkinson disease.
  • ABO-102 (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 (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.
  • Idasanutlin (Idasanutlin, Roche), an oral murine double minute 2 inhibitor for AML. “[This drug] can improve the efficacy of chemotherapy in patients with relapsed or refractory AML,” Tancredi said.
  • Napabucasin (Napabucasin, Sumitomo Dainippon), an oral STAT 3 inhibitor for colorectal cancer. “It targets malignant cancer stem cells that are responsible for malignancy metastatic growth and recurrence,” Tancredi said.
  • Relatimab (Relatimab, Bristol-Myers Squibb), an anti-lymphocyte activation gene 3 injection for melanoma, and renal cell cancer. This drug is also being investigated with other therapies for potential additional oncology indications, Tancredi said.

Additional drug products in the pipeline for all 4 categories also were addressed during Tancredi’s 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]. Silver Spring, MD; May 24, 2019: FDA. 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 Adakveo® (crizanlizumab) approved by FDA to reduce frequency of pain crises in individuals living with sickle cell disease [news release]. Basel, Switzerland; November 15, 2019: Novartis. 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]. Silver Spring, MD; November 20, 2019: FDA. 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]. Silver Spring, MD; October 21, 2019: FDA. 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]. Silver Spring, MD; July 3, 2019: FDA. 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]. South San Francisco, CA; August 7, 2020: Genentech. Accessed September 18, 2020. https://www.gene.com/media/press-releases/14866/2020-08-07/fda-approves-genentechs-evrysdi-risdipla.

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