The 10 Most Exciting Drugs in the 2019 Pipeline

While the development and approval of new therapies is never a sure thing, looking at the current drug pipeline is a useful way to peek into the future of healthcare.

What are the next blockbuster drugs going to be? Where will big expenses be coming from? What disease states are going to get the most attention?

Predicting the pipeline is not a perfect science, but we looked at what could be some exciting releases this year. Here are 10 of the most important expected drugs of 2019, in order of their expected Prescription Drug User Fee Act (PDUFA) date. The first was approved at press time.

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Ravulizumab (Ultomiris)

• Company: Alexion
• Approved: 12/21/2018
• Indication: Long-acting anti-C5 monoclonal antibody for treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Why it’s exciting: PNH is a rare bone marrow failure disorder that usually begins in a person’s early 30s. It results in uncontrolled complement activation which causes hemolysis and other issues.

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For patients with severe PNH, the only current treatment is eculizumab (Soliris), also made by Alexion. For patients who do not respond well to treatment, the only cure is a blood marrow transplant. Ravulizumab needs less frequent doses, every 8 weeks, than eculizumab, which is administered every 2 weeks.

Sacituzumab govitecan

• Company: Immunomedics
• PDUFA: 1/18/2019
• Indication: Antibody drug conjugate for treatment of metastatic triple-negative breast cancer (mTNBC) with at least two prior therapies for metastatic disease.

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Why it’s exciting: According to Arash Sadeghi, PharmD, clinical pharmacist of pipeline and drug surveillance at Optum, this would be the first treatment for mTNBC-which accounts for 10% to 20% of breast cancer cases. Jonathan R. Ptachcinski, PharmD, BCPS, BCOP, clinical pharmacist practitioner at the University of North Carolina Medical Center, says that data from clinical studies (one showed an objective response rate of 34% with a median duration of response of 7.6 months and progression-free survival of 5.5 months) holds promise for patients who progressed through multiple lines of treatment and may not be candidates for other therapies.

Siponimod

• Company: Novartis
• PDUFA: 3/2019
• Indication: A selective modulator of specific subtypes of the sphingosine-1-phosphate receptor for treatment of secondary progressive multiple sclerosis (SPMS).

Why it’s exciting: The National Multiple Sclerosis Society estimates that around 1 million Americans have MS. Of these, 85% will have relapsing-remitting MS, and 80% of those people will develop SPMS.

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If the FDA approves siponimod, it would provide another treatment option as the condition progresses from the relapsing forms of MS. No other drug has been consistently able to slow disease progression with in patient with SPMS, but clinical trials have demonstrated some ability for siponimod to achieve those results. Sadeghi says this drug could generate between $1 and $3 billion in peak revenue.

Esketamine nasal spray

• Company: Janssen
• PDUFA: 3/4/2019
• Indication: Nasal spray for treatment-resistant severe depression.

Why it’s exciting: About 9.5% of the population experiences depression during a given year. About half of patients with depression do not respond to one therapy, and up to 20% do not respond to multiple treatments. 

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Esketamine is touted as a fast-acting alternative to selective serotonin reuptake inhibitor and serotonin and norepinephrine reuptake inhibitors antidepressants. It could be the first new drug to treat depression in 30 years and could reach more than $2.3 billion in sales by 2024.

Zynquista (sotagliflozin)

• Company: Sanofi
• PDUFA: 3/22/2019
• Indication: SGLT-1 and SGLT-2 inhibitor used in addition to insulin therapy to improve blood sugar control in adults with type 1 diabetes.

Why it’s exciting: If sotagliflozin is approved, it would be the first oral treatment for type 1 diabetes on the market. The market for type 1 diabetes is a $5-billion market this year.

Joshua D. Miller, MD, MPH, assistant professor of endocrinology and metabolism at Stony Brook University in Stony Brook, New York, says while the potential market effects-and potential patient side effects-of this drug remain to be seen, the drug is “newsworthy because all we’ve had is insulin.” Diabetologists have long used medications off-label to treat diabetes, he says, though they have never used an SGL-2 inhibitor. This drug could be a game changer-if its benefits outweigh potential problems (especially concerning diabetic ketoacidosis).

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Sotagliflozin is also currently in phase 3 trials for type 2 diabetes, and could receive a PDUFA for that indication later this year.

Risankizumab

• Company: Boehringer Ingelheim/AbbVie
• PDUFA: 4/25/2019
• Indication: An interleukin-23 inhibitor for treatment of patients with moderate to severe plaque psoriasis.

Why it’s exciting: The psoriasis market is huge-according to Pharma Intelligence, it’s projected to be a nearly $7.5 billion U.S. market this year. In its first year on the market, risankizumab could capture $12 million in sales and $31 million by 2020.

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One reason for its predicted success is its efficacy. Efficacy is measured with a psoriasis area and severity index (PASI). Patients with PASI90 improved 90% or more during the treatment period. According to Sadeghi, 75% of patients achieved PASI90 with risankizumab, compared with 48% for Stelara (ustekinumab).

The drug is also currently in phase 3 trials for Crohn’s disease and ulcerative colitis.

NKTR-181

• Company: Nektar
• PDUFA: 5/28/2019
• Indication: A selective mu-opioid agonist for pain relief.

Why it’s exciting: With the opioid crisis ongoing, it seems odd to bring another opioid into the market. But NKTR-181 promises to be an opioid with a lower risk of dependence. According to Nektar, this is because it does not produce the same high levels of euphoria present with other opioids. It has a low permeability across the blood/brain barrier.

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Unlike many other addiction resistant opioids-which are often merely different formulations of existing drugs with a tamper-evident mechanism added or a long-acting formulation-NKTR-181 is a new molecular entity, a first-in-class opioid.

AVXS-101

• Company: Novartis
• PDUFA: 6/18/2019
• Indication: Gene therapy for the treatment of spinal muscular atrophy (SMA).

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Why it’s exciting: SMA affects around 20,000 people in the United States. While there is currently a treatment for SMA (nusinersen, Spinraza), it is wildly expensive, with costs of $750,000 for the first year and $375,000 per year after that. According to Sadeghi, AVXS-101 would be a direct competitor to nusinersen at a lower price tag even though it would still be wildly expensive. Novartis announced that it believes AVXS-101 would be cost-effective with a $4 to $5 million price tag for a one-time treatment, which could be a record price for a drug.

Viaskin Peanut

• Company: DBV Technologies
• PDUFA: 6/22/2019
• Indication: Immunotherapy for treatment of peanut hypersensitivity in children aged 4 to 11 years.

Why it’s exciting: Peanuts are one of the most common food allergies-a National Institute of Allergy and Infectious Diseases 2010 report lists the prevalence as 0.6% of Americans, or approximately 2 million people.

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Currently, there is no definitive treatment for peanut allergy-the only actions available are keeping patients with allergies away from their allergens.

Evenity (romosozumab)

• Company: Amgen and UCB
• PDUFA: 7/12/2019
• Indication: Monoclonal antibody that inhibits the protein sclerostin for treatment of osteoporosis in postmenopausal women at increased risk of fracture.

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Why it’s exciting: According to Sadeghi, there are 10 million with osteoporosis in the U.S. currently, with another 18 million people at risk. Those numbers add up to a large market for osteoporosis treatments. According to Pharma Intelligence, the osteoporosis market will be around $4.1 billion in five years, and Evenity could capture around $550 million of that.

Nicholas Hamm is editor of Managed Healthcare Executive, a sister publication to Drug Topics.