The U.S. Food and Drug Administration (FDA) has granted Shire’s Biologics License Application (BLA) for lanadelumab (SHP643) priority review.
The U.S. Food and Drug Administration (FDA) has granted Shire’s Biologics License Application (BLA) for lanadelumab (SHP643) priority review. The compound is being investigated for the prevention in angioedema attacks in patients 12 years and older that have hereditary angioedema (HAE).
The BLA is based on the results from four clinical trials, including HELP, a pivotal Phase III trial. That trial enrolled 125 patients ages 12 and over with type I/II HAE. The results of that trial showed that subcutaneous administration of 300 mg of lanadelumab once every two weeks showed an 87 percent decrease in the mean frequency of HAE attacks. The company also indicated that an exploratory endpoint that will require confirmation in other studies, “showed that during the steady state stage of the trial (day 70-182) a 91 percent attack reduction was achieved with 8 out of 10 patients reaching an attack free state.”
Lanadelumab is a fully human monoclonal antibody that binds and inhibits plasma kallikrein. HAE is a rare, genetic disorder. It causes debilitating, painful and potentially life-threatening swelling or edema. No treatment-related serious adverse events were reported. The most common side effect was pain at the injection site.
“Every day, patients living with HAE struggle to manage their disease—not knowing when their next attack will occur,” said Andreas Busch, Shire’s executive vice president, Head of Research and Development, in a statement. “Lanadelumab if approved will be the first monoclonal antibody for HAE, a serious and potentially life threatening disease. Lanadelumab provides a new mechanism of action inhibiting plasma kallikrein for the prevention of HAE attacks. Lanadelumab will offer patients a new option to help control this disease with the potential to change the treatment paradigm. The FDA’s decision underscores Shire’s serial innovation in HAE and commitment to improving treatment options for patients.”
With the priority review status, Shire is expected a decision by the FDA by August 26, 2018. Priority review status cuts the completion time from about 12 months to about 8 months.
“Physicians as well as patients in the HAE community are excited to see lanadelumab moving forward for FDA review because there is now the real possibility of having a new way to prevent HAE attacks,” said Aleena Banerji, a clinical trial investigator and physician at Massachusetts General Hospital, Boston, in a statement. “As an investigator, I am appreciative of the HAE patients who have participated in the clinical trial to help advance science in a way that may transform the treatment of HAE.”
The report also coincided with the company’s fourth-quarter results, with better-than-expected fourth quarter results, but what Seeking Alpha refers to as disappointing outlook for 2018. Fourth-quarter sales were $4.07 billion, 2.5 percent over consensus expectations. Seeking Alpha, however, writes, “2018 guidance looks disappointing. In details, Shire guided for a Core EPS per ADS between $14.90 and $15.50, which implies zero organic growth for the earnings in 2018, even if Shire guided for around mid-single digit sales growth. The key reasons behind this weak guidance are related to lower expected royalties and gross margin, mainly as a result of the investments behind the expansion of the plasma manufacturing capacity of the company.”
