Dr Aimee Tharaldson on Recently Approved Specialty Drugs, Upcoming Approvals to Watch

Aimee Tharaldson, PharmD, a senior clinical consultant in Emerging Therapeutics for Express Scripts, discusses specialty drugs that have recently been approved, and which we will likely see approved in the remainder of the year.

Transcript

What specialty medications have recently been approved, and which will we likely see approved in the remainder of 2018?

So, a few highlights as far as recent approvals would be 2 new HIV medications. There have been more than 2, but some key ones would be biktarvy. So, this is a new integrase strand transfer inhibitor single dose tablet with 2 other medications, and that would be used as a once daily medication. Really, in the HIV market, they’re moving to these integrase strand transfer inhibitors because of their efficacy and they’re pretty well tolerated, as well.

Then there was another drug, trogarzo, which is an infused biologic drug for HIV. That’s just for patients with multidrug resistant HIV. So, that’s another key medication.

We’ve had some recent orphan medication approved. One that just happened last week is Crysvita. This is for patients with x-linked hypophosphatemia, a very rare condition. It affects about 3000 children and 9000 adults in the United States. Previously, they didn’t really have any treatment options available for them. So, this is a medication that really helps improve their bone strength and increase their phosphorus levels. That was just recently approved. So, that’s really a few key recent approvals.

As far as some to watch for the remainder of this year, there are actually quite a few cancer drugs currently pending approval. So, some of these are oral medications that are for very targeted patient populations. One example is larotrectinib for patients with TRK-fusion proteins, so this is really uncommon in cancer. Less than 3% of cancers actually have these mutations, but they’re the harder to treat cancers. So, there’s going to be a new treatment option available for them.

Other things to watch for: we’re going to have new CGRP inhibitors for migraine, so that’s really an area to keep an eye on. So, these will be used to prevent migraine in patients that have 4 or more migraine days per month. Just in the next 6 months, we can have 3 new CGRP inhibitors. So, they’re subcutaneous medications, and the market is pretty large for the potential use for those medications.

One other area to keep an eye on for this year is he hereditary ATTR amyloidosis. So, this is a rare condition, again. There’s about 10,000 to 20,000 patients in the US that have this, but it’s really hard to identify those patients. There’s nothing available to treat them right now. So, they could have cardiac manifestations of their disease, they can have polyneuropathy, and a whole variety of other symptoms. So, there are going to be to 2 new drugs to treat these patients later this year.

So, it’s kind of interesting and fun to watch how underserved patients are going to have new therapies.