Specialty Drug Approvals Dr Aimee Tharaldson is Looking Out for in 2019

Aimee Tharaldson, PharmD, a senior clinical consultant in Emerging Therapeutics for Express Scripts, outlines specialty drug approvals she's keeping her eye on in the coming year.

Transcript

Are there any drug approvals that you are keeping your eye on in 2019?

So, as far as some drugs to watch for in 2019, there’s actually going to be 4 new oral medications for MS [multiple sclerosis]. I just want to highlight one of them: cladribine. This is a little different mechanism. It helps silence the immune function in the brain in patients with MS. It seems to be effective. What’s interesting about this medication is the dosing of it. It’s given once daily for 5 days one week, then a month later it’s given once daily for 5 days. That regimen’s repeated in year 2, and then that’s it. So, it’s very unique as far as how it’s administered, so that’ll be an interesting new option on the market for patients with relapsing MS.

Another gene therapy to watch for is AVXS-101. So, this is for patients with spinal muscular atrophy type 1. So, that only represents patients from birth to 6 months, and it’s a very severe form of muscular atrophy where these patients, who are babies, aren’t able to crawl or walk. They have difficulty breathing and eating. Only about 8% of babies diagnosed with this survive to 20 months of age, so there’s a need for treatment options for them. We do have spinraza, which is an intrathecal medication. It works differently. This is a once-time infusion gene therapy that has demonstrated strong efficacy in clinical trials. This could cost anywhere from $1 million to $3 million for a one-time infusion.

We also have tafamadis—an interesting drug to watch for in 2019, as well. This is for patients with cardiomyopathy due to transthyretin mediated amyloidosis. So this could be used in patients that have hereditary form of disease and wildtype, which just occurs as people age. When patients finally get this diagnosis—it takes a long time to diagnose these patients—their life expectancy is only about 3 to 5 years because they progress rapidly to atrial fibrillation and heart failure.

So, this is an oral medication. It’s going to approved next year to treat these patients, and it looks like it’s going to be a pretty effective medication in clinical trials. We saw a 30% reduction in mortality, as well as a 32% reduction in cardiovascular-related hospitalizations, as well. So, was for that, probably mid 2019.