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The Little Orphan That Could: How Alexion is Going Global


Pharmaceutical Executive

Since the initial FDA approval of its flagship orphan drug Soliris in 2007, Alexion Pharmaceuticals has done well for itself as a small biotech firm that focuses on ultra-rare disease.


David Hallal, Chief Commercial Officer, Alexion Pharmaceuticals

Since the initial FDA approval of its flagship orphan drug Soliris in 2007, Alexion Pharmaceuticals has done well for itself as a small biotech firm that focuses on ultra-rare disease. So well in fact, that instead of partnering, the company has taken global expansion into its own hands. Apart from a pipeline that includes asfotase alfa, a promising treatment for a rare metabolic bone disease, Soliris now has approval in over 50 countries for its premier indication, Paroxysmal Nocturnal Hemoglobinuria (PNH), and is gaining steam with a second indication for atypical hemolytic uremia syndrome (aHUS). In focusing on spreading disease awareness to physicians and patients alike, making a case for regulatory approval and reimbursement for Soliris based on clinical efficacy, and recruiting globally as well, Alexion has put solid feet on the ground in its commitment to a worldwide presence.

Alexion knows that within each market, it may only serve a few hundred patients at best with Soliris and its indications. In terms of stakeholder alliances, Alexion’s Soliris strategy hones in on education of the medical community about the diseases it treats, who is at highest likelihood for having these diseases, and which diagnostic technique can be used to make a definitive diagnosis. “We shaped that strategy utilizing field representatives and seminars that are focused on specialists who might have seen patients affected with PNH or aHUS but didn’t recognize it,” says David Hallal, Alexion’s Chief Commercial Officer. “Low awareness is often the greatest obstacle to identifying patients and providing them with treatment because you can’t treat somebody you don’t diagnose.”

Generally, Alexion has encountered a supportive global landscape for therapies targeting ultra-rare diseases and the patients that depend on them. While the UK dealt a setback to the company in its initial rejection of Soliris for aHUS earlier this year (despite recommendations from NICE’s Advisory Group for National Specialised Services (AGNSS)), regulators at NHS have commissioned the treatment as of this month, pending further confirmation from NICE in spring of next year. Alexion’s strategy in obtaining approval and justifying its narrow pricing band, where in the US treatment per patient costs over $400,000 a year, lies mostly in demonstrating Soliris’ clinical efficacy in treating aHUS and PNH. “The approvals from the regulatory authorities highlight that there’s a 100% objective response rate in our clinical trials for both conditions,” Hallal cites. “Such transformative outcomes highlight what we think clearly support our narrow pricing band.” The company also does well to ask, as Hallal puts it, “Is the country willing to pay what other countries are paying to serve its citizens?”

Alexion already operates throughout Europe and Asia, continues to expand its presence in Latin America, and is looking to establish a foothold in Africa as well. The company’s efforts to improve its operations overall coincide with its continuing expansion. For instance, Alexion recently announced it would begin recruiting for its supply chain and quality assurance, quality control and logistics operations in Dublin, Ireland. This news came just weeks before an announcement of its international recall of 1-2% of a month’s supply of Soliris for patients worldwide, and warnings from the FDA in March over issues pertaining to bacterial contamination at the drug’s Rhode Island-based manufacturing plant. The commitment to both serving the EU drug markets and to assuring safety and quality is certainly a step in the right direction.

For a company that obtained approval for its very first indication just six years ago, Alexion is a good example for how focus in the right avenues in the drug industry today can lead to swift global success. In targeting life threatening ultra-rare diseases with impressive data, educating specialty physicians in recognizing these diseases, and focusing on improving diagnostic approaches to identify patients rapidly, this small biotech, currently at 1,100 employees worldwide, is casting a wide net for its diminutive stature and its brief history.

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