The 5 drugs to watch in 2019

1. A gene therapy for Duchenne muscular dystrophy

Duchenne Muscular Dystrophy (DMD) is a devastating and incurable muscle-wasting disease, associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. DMD is always fatal, usually before age 30, and generally due to respiratory or cardiac failure.

If approved, Golodirsen will offer a new treatment option for DMD patients with a certain genetic makeup. While an existing medication in the class, Exondys-51™, works on the 13% of DMD patients who have a mutation on an area of the Duchenne gene known as exon 51, Golodirsen instead targets the 8% of DMD patients who have a mutation on exon 53.1

As with Exondys-51, expect Golodirsen to be costly. Sarepta Therapeutics Inc., which makes both drugs, charges approximately $300,000 per year for the average patient using Exondys-51.2

2. A new option for multiple sclerosis

Most people with the most common form of multiple sclerosis, relapsing-remitting MS (RRMS), go on to develop secondary progressive multiple sclerosis (SPMS) after 10 to 25 years since disease onset.3 SPMS is a form of MS that leads to progressive, irreversible disability, such as the need for enhanced walking aids and wheelchairs, bladder dysfunction and cognitive decline. Researchers have long sought a drug to delay onset of SPMS.

Novartis Pharmaceuticals claims that siponimod is the first investigational medicine to show a significant delay in disability progression in typical SPMS patients. If approved, siponimod would be the first oral disease-modifying therapy for SPMS patients.4

3. An oral insulin adjunct for type 1 diabetes

Left unchecked, the elevated blood sugar levels associated with type 1 diabetes can lead to a variety of serious health complications. Accordingly, patients with type 1 diabetes are dependent on injections of insulin to control blood sugar. Yet, studies show that most people with type 1 diabetes fail to meet glycemic guidelines.5

If approved, Zynquista™ (sotagliflozin) would be the first oral antidiabetic drug used in combination with insulin to control blood glucose levels in type 1 diabetics.

Sotagliflozin has an FDA action date of March 22, 2019. Some analysts are optimistic about approval, citing strong efficacy and safety results presented at a European conference in early October. Peak sotagliflozin sales are predicted to reach $1.3bn by 2024 in the U.S.6

4. An injectable for psoriasis

There is no shortage of drugs used to treat autoimmune conditions. Indeed, two drugs in this class, Humira® and Enbrel®, are currently first and second in overall spending for all medications in the U.S.7

Risankizumab is currently being evaluated by the FDA for the treatment of plaque psoriasis, an autoimmune disease. Risankizumab has demonstrated greater efficacy in reducing psoriasis symptoms in clinical trials vs. an existing market leader.8 Looking forward, risankizumab is also being studied for other autoimmune conditions, such as Crohn’s disease and ulcerative colitis.9

5. A longer-duration drug for a rare blood disorder

Paroxysmal nocturnal hemoglobinuria (PNH) is a condition that leads to early destruction of red blood cells. The disease also impairs disease fighting white blood cells as well as blood-clotting platelets.10 PNH can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.

Made by Alexion Pharmaceuticals, Ultomiris™ was approved on Dec. 21, 2018. According to Alexion, Ultomiris offers longer intervals between injections for PNH than its current medicine in the class, Soliris®. Instead of the current the two-week interval for Soliris, Ultomiris is intended for dosing intervals of up to 8 weeks.11

The estimated average cost of Ultomiris is approximately $458,000 per year, which Alexion estimates to be about 10% less than Soliris.12