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

Aimee Tharaldson, PharmD, a senior clinical consultant in Emerging Therapeutics for Express Scripts, provides an update on recently approved speciality medications and which we will likely see approved in the remaining months of 2018.


What specialty medications have recently been approved, and which will we likely see approved in the remaining months of 2018?
So, just a few highlights. We had epidiolex approved this year, and this is a new marijuana type derivative that doesn’t have any psycho effects for patients with very rare and resistant types of seizure disorders. That medication was just recently scheduled as a schedule 5 controlled substance, so that’s the least level of control on the medication, and it is effective in treating these patients. They’re planning on launching it soon. It will be available through specialty pharmacies. So, that’s great. That will actually be the first medication specifically approved for Dravet syndrome, which is this rare epilepsy type.

Other significant approvals, we’ve had 2 drugs approved this year for patients with polyneuropathy due to transthyretin mediated amyloidosis, or TTR amyloidosis. So, we have Onpattro, and that’s an infused therapy that’s given once every 3 weeks for these patients, and then Tegsedi was also approved, and that’s a subcutaneous medication that’s given every week. So, this is a patient population that previously had no treatment options available for them. It’s about 3000 patients in the US that are affected by this. It takes a long time to diagnose. Once they are diagnosed, typically life expectancy is between 3 to 15 years, so there’s definitely a need for treatment options for them. Now they have 2 options. These medications cost about $450,000 per year. Both of them do.

As far as new a cancer medication, we had Libtayo approved. So, that’s a new PD-1 inhibitor, so it’s an infused therapy, and it’s the first medication approved to treat patients with cutaneous squamous cell carcinoma. So, most patients can be just treated with surgery or radiation, but for some patients, this actually spreads and this is the first systemic therapy approved for them. That was just recently approved, I think it was September when it gained that approval. Again, the only drug approved for those patients.

So, those are some recent approvals. A couple of drugs to watch for later this year: larotrectinib is a new cancer medication. It’s an oral medication that targets with TRK fusion cancers. It only represents about 3% of cancers, but it’s associated with hard-to-treat cancers like pancreatic cancer, thyroid cancer, salivary gland cancer. So, patients can actually be tested for these TRK fusion proteins and we’ll have this new option available for them. It’s a breakthrough therapy with approval expected next month in November.

And then we also have brexanalone, which is kind of interesting. This is an infused medication for moderate to severe postpartum depression. We usually don’t think of depression as a specialty condition, but this is infused and it’s given over a 60-hour infusion, and it has demonstrated efficacy in improving depression symptoms in as early as 24 hours after the infusion starts and may last as long as 30 days. So, this would just be for patients with moderate to severe postpartum depression, and they’re looking at home infusions and working with specialty pharmacies to have access to this medication. That’s expected to be approved in December.
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