Alexion Pharmaceuticals Inc., which began operations in mid-1992, is a renowned biopharmaceutical company based in the US. It was publicly listed in mid-1996. In 2011 it joined the NASDAQ-100 Index. In 2012 it joined the Standard & Poor’s 500 Index. Alexion Pharmaceuticals develops treatments for ultra-rare, as well as severe, diseases. One of the Alexion Pharmaceuticals’ most renowned products is Soliris, which us a terminal complement form of inhibitor, for treating aHUS (atypical Hemolytic Uremic Syndrome) as well as PNH (Paroxysmal Nocturnal Hemoglobinuria). Alexion Pharmaceuticals’ metabolic products include varied enzyme-replacement therapies for treating HPP (hypophosphatasia) as well as LALD (acid lipase deficiency). The pipeline for Alexion Pharmaceuticals includes multiple therapeutic areas’ product candidates. The company’s head office is in New Haven, Connecticut according to Alexion (2016a).
By and large, the therapies developed by Alexion Pharmaceuticals are life-transforming, especially in the case of patients suffering from rare, as well as devastating, diseases. The patients often lack effectual treatment alternatives. They and own families battle the diseases with limited hope if any. The company’s main objective is to develop medical breakthroughs and innovations. Most of the company’s products are specialty drugs for treating unusual diseases (Alexion, 2016a). Notably, the specialty drugs are targeted at a small base of customers and thus they are relatively cheaper, as well as quicker, than the drugs targeted at the mass market. Besides, established pharmaceutical firms have been inclined towards steering clear of specialty drugs markets, giving rise to a niche, which has limited competition, for the company.
Jain (2000) asserts that, by and large, insurers are willing to meet the high costs, or prices, for specialty drugs given that only a few of own clients require the drugs. That means that the drugs’ high prices do not considerably affect the outlays of the insurers. The market for the drugs has considerably grown in recent times owing to the decline of the supply of medicines to mass markets by the traditional pharmaceutical businesses according to Jain (2000). Possibly, that is why the prices of specialty drugs have risen rather fast in recent times.
Soliris is used legally in the US, Japan, the EU, and Canada. Even then, the supply of the drug to Canada remains quite limited. Most Canadians who use the drug access it via private clinics. The drug is arguably the most costly of all drugs globally: only a few persons can afford it. Consequently, Alexion Pharmaceuticals engages public relations companies to assist patients, as well as their families, put in place campaigns to demand that their governments purchase the drug for those requiring it as suggested by Alexion (2016a). The company as well engages governments directly to have them purchase the drug for their subjects in need of it. Specialty drugs’ prices have rather elevated margins over the drugs’ cost prices. The prices that the company charges on its drugs commonly attract ethical questions on the company’s product pricing regime and ethics.
Even then, Alexion Pharmaceuticals with its research efforts geared towards developing other high-margined, as well as high price, drugs. There are those questioning the ethical grounding of the company’s motivation to price its products so highly that the targeted consumers are unable to afford them. Notably, most of those suffering from aHUS or PNH die of the conditions since they cannot afford Soliris. Those with such conditions die owing to organ failure organ damage. Commonly, they die owing to kidney failure or damage stemming from unregulated complement activation according to Jain (2000). The approval of the usage of the drugs by particular governments often attracts positive reactions from medical practitioners who see the approval as a markedly significant advance made for those with the conditions and their families (Alexion, 2016a). Over 50 nation-states have since approved the usage of Soliris by PNH patients. Over 40 nation-states have since approved the usage of Soliris by aHUS patients.
Alexion Pharmaceuticals, a world leader regarding complement inhibition, is bolstering, as well as expanding, own complement inhibitor portfolio, including appraising possible eculizumab indications for other ultra-rare and severe diseases. Its metabolic franchise includes asfotase alfa (Strensiq) along with sebelipase alfa (Kanuma). Kanuma and Strensiq are markedly innovative therapies that entail enzyme replacement. They are targeted at those with HPP or LALD. Strensiq is used legally in the US, Japan, the EU, and Canada. Kanuma is used legally in the US, Japan, and the EU (Alexion, 2016a).
Presently, Alexion Pharmaceuticals is developing a rather robust unusual disease pipeline. The pipeline is the most robust within the entire biotech industry. The pipeline, in addition to the company’s metabolic, as well as complement, clinical programs, includes at least 30 different pre-clinical programs covered diverse therapeutic modalities according to Alexion (2016a).