- Shire's much-anticipated prophylaxis treatment for a rare disease that causes severe and sometimes life-threatening swelling has received approval from the Food and Drug Administration.
- Lanadelumab, now branded as Takhzyro, inhibits an enzyme thought to be one of the root causes of the disease, known as hereditary angioedema or HAE. Phase 3 testing that led to Takhzyro's approval found patients treated with 300 mg of the drug every two weeks experienced an 87% reduction in monthly HAE attacks compared to placebo.
- Shire didn't disclose Takhzyro's annual price in a Thursday statement. However, the Institute for Clinical and Economic Review, a watchdog group focused on the cost-effectiveness of drugs, used a placeholder list price of $537,097 for the drug in a report on HAE therapies also released on Thursday. Analysts expect the new product to reach blockbuster status in the next five years.
Of the six FDA-approved drugs for HAE, Shire owns three. In the second quarter, the trio of Cinryze (C1 esterase inhibitor [human]), Firazyr (icatibant) and Kalbitor (ecallantide) brought in $365 million, a 9% increase from the same period in 2017.
Firazyr was the sole driver of that growth, as the other two brands recorded year-over-year declines. The situation has been particularly dire for Cinryze, which suffered from "historic difficulties" with its contract manufacturer. Shire's determined to avoid the same setbacks with Takhzyro.
"Everything is on track," Shire's Chief Financial Officer Thomas Dittrich said of lanadelumab's regulatory timeline during a recent earnings call, "and what is also on track is our readiness both on the manufacturing side and the commercial side for launch in the U.S. and outside the U.S."
That's surely good news for investors, especially considering lanadelumab is a key near-term growth driver for Shire and its soon-to-be merger partner, Japan's Takeda Pharmaceutical. Thomas Moore, senior pharma analyst at market research firm GlobalData, noted in a July report that the drug is the most valuable asset in either Shire or Takeda's pipeline as measured by peak annual sales forecasts between 2018 and 2023. By that latter year, Takhzyro sales are poised to reach $1.3 billion.
"Compared to Shire's future offerings, Takada's own pipeline is looking a little bare, and so the company will be looking to leverage Shire's pipeline once the merger has completed in order to reignite its stumbling operating profits," Moore said.
Analysts also predict Takhzyro to perform well against rival therapies.
For instance, Cowen & Co.'s Ken Cacciatore called the drug a best-in-class HAE treatment in an April investor note, adding that its market entry "should convert Cinryze, defend against Haegarda, pull more patients from on-demand to prophylaxis, open up the potential to reach into currently underserved international markets, and [provide] extended durabillity to this key franchise."
With an approval secured, Wall Street's attention will surely turn to what wholesale acquisition cost Shire sets for Takhzyro. Knowing HAE therapies, it will be high.
ICER used a $537,097 list price for the drug based on mean monthly cost per cycle of C1 Esterase inhibitors Cinryze, Haegarda and Ruconest. The group couldn't give a direct cost-effectiveness judgment for landelumab as the drug's actual price is not yet known, but did conclude that — under a placeholder net price of $497,259 — the budgetary impact of using lanadelumab versus a mix of Haegarda, Cinryze, and no long-term prophylaxis would be positive.
ICER finds lanadelumab would offer 18.42 QALYs in base-case scenario
|Total Cost — U.S. Health System Perspective||$10,560,000||$17,690,000||$23,800,000||$9,810,000||$12,978,000|
|Prophylaxis Drug Cost||$0||$12,465,000||$19,900,000||$8,282,000||$11,592,000|
|Acute Treatment Costs||$10,560,000||$5,225,000||$3,900,000||$1,528,000||$1,383,000|
|Acute Treatment Costs (Drugs)||$9,725,000||$4,814,000||$3,600,000||$1,467,000||$1,274,000|
|Acute Treatment Costs (Other Services)||$830,000||$412,000||$300,000||$141,000||$109,000|
|Quality Adjusted Life Years||17.15||17.91||18.14||18.43||18.42|
|# of Attacks||1,873||929||700||300||245|
More specifically, ICER determined the average potential budgetary impact of using lanadelumab at the $537,097 placeholder list price was about an additional $7,800 per-patient. Under an assumed net price, landelumab would produce per-patient cost savings of nearly $32,000, ICER said.
ICER did acknowledge, however, that there were other difficulties in assessing lanadelumab.
"Although trials of long-term prophylaxis with [C1 inhibitors] and lanadelumab showed benefits in reducing the frequency of HAE attacks with few harms, the evidence base is limited. We identified only four randomized controlled trials meeting our inclusion criteria, one of each drug of interest in our review, and the study populations were small," ICER wrote in its report, though the group also noted "[t]his is to be expected with an ultra-rare disease."