Top 9 Most Exciting Drugs in the 2019 Pipeline

Company: Immunomedics

PDUFA: 1/18/2019

Indication: Antibody drug conjugate (ADC) for the treatment of patients with metastatic triple-negative breast cancer (mTNBC) who previously received at least two prior therapies for metastatic disease.

Why it’s exciting: According to Arash Sadeghi, PharmD, clinical pharmacist of Pipeline and Drug Surveillance at Optum, if approved, this would be the first treatment for mTNBC-which accounts for 10-20% of breast cancer cases.

Jonathan R. Ptachcinski, PharmD, BCPS, BCOP, clinical pharmacist practitioner, University of North Carolina Medical Center, says that data from clinical studies holds “promise for patients who have progressed through multiple lines of treatment and may not be candidates for other currently available therapies.”

Company: Novartis

PDUFA: 3/2019

Indication: A selective modulator of specific subtypes of the sphingosine-1-phosphate (S1P) receptor for the treatment of secondary progressive multiple sclerosis (SPMS).

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

If the FDA approves siponimod, it would serve as another treatment option as the condition progresses from 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. 

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 single year. About half of all patients with depression do not respond to one therapy, and up to 20% of patients do not respond to multiple treatments. 

The drug is touted as a fast-acting alternative to SSRI and SNRI antidepressants, and if approved would be the first new mechanism to treat depression in 30 years.

Pharma Intelligence predicts a $4.3 billion market for depression in 2019-while also predicting that esketamine could achieve over $2.3 billion in sales by 2024.

Company: Sanofi

PDUFA: 3/22/2019

Indication: SGLT-1 and SGLT-2 inhibitor to improve blood sugar control in adults with type 1 diabetes, in addition to insulin therapy.

Why it’s exciting: If approved, it would be the first oral treatment for type 1 diabetes on the market. According to Datamonitor Healthcare, type 1 diabetes is over a $5 billion market this year alone.

Joshua D. Miller, MD, MPH, assistant professor of endocrinology and metabolism at Stony Brook University in Stony Brook, New York, says that while the potential market effects-and side effects-of this drug remain to be seen, the drug is “newsworthy because all we’ve had is insulin.”

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

Company: Boehringer Ingelheim/AbbVie

PDUFA: 4/25/2019

Indication: An interleukin-23 (IL-23) inhibitor for the treatment of patients with moderate-to-severe plaque psoriasis.

Why it’s exciting: It’s no secret that the psoriasis market is huge-according to Pharma Intelligence, it’s projected to be a nearly $7.5 billion U.S. market this year alone.

One reason for its predicted success is its efficacy. Psoriasis medication 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 Stelara’s 48%.

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

Company: Nektar

PDUFA: 5/28/2019

Indication: A selective mu-opioid agonist for pain relief.

Why it’s exciting: With the opioid crisis in full swing, it seems odd to bring yet 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 NKTR-181 does not produce the same high levels of euphoria present in other opioids-it has a low permeability across the blood/brain barrier.

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

Company: Novartis

PDUFA: 6/18/2019

Indication: Gene therapy for the treatment of spinal muscular atrophy (SMA).

Why it’s exciting: While there is currently a treatment for spinal muscular atrophy, nusinersen (Spinraza), it’s also wildly expensive for the around 20,000 SMA patients in the United States. According to Sadeghi, AVXS-101 would be a direct competitor to Spinraza’s $750,000 per year price tag.

AVXS-101 could shake that up, but being a gene therapy, it too is wildly expensive-Novartis announced that it believes the treatment would be cost-effective with a $4 to $5 million per patient price tag. But, like other gene therapies, if it does indeed work long-term, even that hefty of a price tag could result in savings over time. 

Company: DBV Technologies

PDUFA: 6/22/2019

Indication: Immunotherapy for the 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.

Currently, there is no definitive treatment for peanut allergy-the only actions available are separating patients with allergies from any possible allergens.

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.

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, Evenity could capture around $550 million of that.