Patients in the USA who have LAL-D hopefully won’t have to wait too much longer on regulatory news, as in the U.S. Kanuma has been granted a Breakthrough Therapy designation and Priority Review by the FDA.
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On Tuesday, European health regulators also approved Alexion’s Strensiq to treat pediatric-onset hypophosphatasia (HPP), a potentially fatal disease that can lead to bone deformity, fractures and other skeletal abnormalities. For quite some time, Alexion has been known as a one-drug company. We do expect Alexion’s stock to be weak today given the ongoing uncertainty about the timing for approval in the USA for both Kanuma and Strensiq.
LAL-D is caused by a genetic mutation that results in a loss of LAL enzymes, contributing to risky buildups of cholesterol and triglycerides that can severely damage the liver.
Both are classified ultra-rare diseases as they occur in less than 20 patients per one million in the general population. “We are pleased that the European Union label will allow any patient who had symptoms of HPP prior to the age of 18 to be eligible for treatment”, said David Hallal, Chief Executive Officer of Alexion. He declined to provide a sales forecast for Strensiq.
He sees a slow sales uptake for the drugs as raising awareness of the diseases and identifying patients is one of the biggest challenges facing the company. Spokesman Irving Adler said Alexion will not say whether it has enrolled any patients on the drug in those countries. “When we launch, it’s usually one patient at a time”. Canadian regulators said Soliris, which treats a blood disorder, costs more than $700,000 a year for each of the 180 patients who needed it in the country, before negotiated discounts.
The price Alexion paid for Synageva, which was more than twice its market value, raised eyebrows and drew criticism on Wall Street.
Alexion said the Synageva deal will add to profits beginning in 2018. It had sales of $2.23 billion in 2014. The company’s financial success has been 100% dependent on Soliris, a super-expensive drug approved to treat two rare and life-threatening diseases: paroxysmal nocturnal hemoglobinuria, or PNH, and atypical hemolytic uremic syndrome, or aHUS.
On top of that, “we added another 12 preclinical programs from Synageva to our pipeline”, Hallal said.
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Alexion shares fell $1.32 to close at $170.87 on Nasdaq.
