1. Zolgensma (Onasemnogene abeparvovec). The drug is a gene therapy for patients with spinal muscular atrophy, an inherited muscle-wasting disease often fatal to infants before the age of 2. Zolgensma, which recently received FDA approval, offers the potential for infants to be cured in one treatment. The drug has been priced at $2.1 million, making it the most expensive drug in the world.
2. NKTR-181. This is a new opioid-based pain reliever that has a molecular structure that makes it harder to abuse. While this drug provides hope for a safer opioid that may lower the likelihood of dependency, some people are skeptical that its benefits will translate into less abuse.
3. Golodirsen. The drug is a gene therapy to treat Duchenne muscular dystrophy, a fatal disease characterized by muscle deterioration. The drug offers a treatment option for a serious condition with no other treatment options available.
4. Upadacitinib. Is an oral treatment for adults with rheumatoid arthritis, a chronic disease that can cause irreversible joint damage. According to Optum, Upadacitinib is the only product in the class that treats rheumatoid arthritis to show superiority to Humira.
5. Vyndaqel (Tafamidis meglumine). This is a first-in-class oral treatment for transthyretin amyloid cardiomyopathy, a disease characterized by the buildup of abnormal deposits in the heart that lead to heart failure, heart block and sudden death. Vyndaqel is the first and only FDA approved treatment for the rare disease. Vyndaqel costs about $250,000 per year.
Read the full report here.
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