Each month, we round up the latest FDA approvals and regulations to give you an expert take on what you need to know.
FDA Approves Expanded Indication for Hemophilia Medication
Hemlibra® (emicizumab-kxwh) injection received approval from the FDA to be used as routine prophylaxis to prevent or reduce frequency of bleeding episode in patients with Hemophilia A with or without factor VIII inhibitors.1 This expanded indication opens up the majority of the Hemophilia A market to be candidates for Hemlibra therapy.
This is an injection that is self-administered weekly, bi-weekly, or monthly depending on patient specific factors. Other therapies require administration by a medical professional either in a clinic, hospital, or home health service. Hemophilia is a $10B a year market even though estimates suggest there are fewer than 20,000 patients in the U.S.1
Data on Comparative Effectiveness of Two Oral Multiple Sclerosis Products Presented
Generally, products are not studied vs. competitors but rather vs. placebo or standard of care. Earlier this month at the European Committee for Treatment and Research in Multiple Sclerosis, data was presented comparing Aubagio (teriflunomide) and Tecfidera (dimethyl fumarate) in treatment of relapsing-remitting MS (RRMS).2
There remain many unknowns, but this data suggests patients on Tecfidera had fewer new brain lesions after two years of treatment. The two treatment arms had a similar proportion of patients experiencing at least one relapse as well as similar impact on patient reported disability scores.2 This is not conclusively stating that Tecfidera performed better than Aubagio, but this is great insight into the real world efficacy of these specialty pharmacy agents.
Just in Time for Flu Season, FDA Approves New Treatment
As patients receive their vaccines as the first line of defense against the flu, the FDA has approved Xofluza™ (baloxavir marboxil) tablets for the treatment of acute uncomplicated influenza in patients 12 years of age and older who have been symptomatic for no more than 48 hours.3
This new product is not only the first drug in its class (endonuclease inhibitors4) but is also a one-time dose. Cost of this product is estimated to be around $150 per treatment. It is still to be determined how PBMs will treat this medication. There will likely be some utilization management edits in place to help ensure appropriate utilization such as quantity and age limits.
Price of Cholesterol Drug to be Cut by 60%
Not a headline one sees often...
However, Amgen has decided to reduce the price of their cholesterol medication Repatha (evolocumab) by nearly 60%. This still does not make this product necessarily inexpensive, considering the new annual cost remains nearly $6,000.5
This final cost is in line with the net price Amgen currently receives for Repatha as a result of discounts and rebates to PBMs.5 This is going to interesting to see how PBMs address this pricing change. With the net price back to Amgen being the same, likely rebates are going away or are going to be drastically reduced. The category of medication of Repatha (PCSK9s) have not lived up to sales expectations. This price reduction could be something PBMs use to try to lower specialty rebate guarantees. With the low utilization of this category, it will be important to leverage a tool and technology capable of accurately identifying the value of any changes to rebate guarantees.