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Alexion Pharmaceuticals: The Power Of Soliris

June 1, 2013

Alexion Pharmaceuticals’ only marketed product, orphan drug Soliris (Eculizumab), generates blockbuster profits from multiple indications for ultra-rare diseases. Soliris is one of the world’s most expensive drugs, costing approximately $409,500/year/patient. Soliris treats ultra-rare diseases such as :

•   Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare genetic blood disorder
•   Atypical Hemolytic Uremic Syndrome (aHUS), an ultra-rare genetic disorder.

Per Alexion Pharmaceuticals’ first Quarter 2013 financial results, Soliris net product sales increases 38% from $244.7 million in the first Quarter 2012 to $338.9 million in the first Quarter 2013. EvaluatePharma publishes in April 2013, the “Orphan Drug Report 2013”, which analyzes the orphan drug market between now and 2018. Some interesting statistics are provided by the report on Soliris :

•   Is the 3rd top selling orphan drug in the world in 2018 (Roche’s Rituxan is #1 & Celgene’s Revlimid is #2)
•   2012 worldwide sales is $1.134 billion
•   2018 estimated worldwide sales is $3.404 billion.

Alexion Pharmaceuticals’ Vikas Sinha, CFO and Exective Vice President, presents on May 30, 2013, at the Deutsche Bank 38th dbAccess Annual Health Care Conference in Boston, Massachusetts. Here are a few highlights from the presentation :

•   Soliris is approved for PNH in 40 countries, growing its core operations in US, Western Europe and Japan, and expanding into emerging markets such as Turkey, Brazil, Russia
•   The drug is approved for the treatment of patients with aHUS in the US and the European Union (EU) in late 2011. The US launch is currently in process, and launches in the EU will continue into 2013
•   Expanding the Soliris franchise with 5 additional indications :

  1. STEC-HUS
  2. Neuromyelitis Optica (NMO)
  3. MG Kidney transplant
  4. Delayed transplant graft function
  5. Myasthenia Gravis

•    4 additional therapeutic candidates :

  1. Asfotase Alfa for Hypophosphatasia (HPP – recently receives the 15th FDA Breakthrough Therapy Designation)
  2. cPMP replacement therapy for Molybdenum Cofactor Deficiency Type A
  3. Alexion 1007
  4. Alexion 1102 & 1103

•   1st Quarter 2013 saw $338.9 million in sales, about 38% year-over-year
•   2013 revenue forecast is 24% growth over 2012.

Per SeekingAlpha.com’s transcript of the presentation, when asked about the FDA Breakthrough Therapy Designation for Asfotase Alfa, Irving Adler, Executive Director of Corporate Communications, says :

 … people have asked about exactly what does this mean. It is designed to expedite the development of a drug in a setting such as this one. And importantly, it’s based on preliminary clinical evidence that this candidate therapy would be a substantial improvement over any existing. In the case of HPP, you’re dealing with only palliative care for what’s a life-threatening or a deforming disorder… provides … all the benefits of fast-track, plus the additional discussion. And importantly, it includes advice that might spare patients from being subjected to non-efficacious treatment…

Thus, Alexion Pharmaceuticals has shown a slow, steady, continuous, successful growth from its only marketed product, by expanding the Soliris franchise :

•   Adding other indications
•   Expanding its geographic presence and penetration.

There has been some resistance in the UK towards the pricing of Soliris recently. The UK rejected a recommendation by an advisory group tasked with determining whether to approve Soliris for aHUS in January. Instead, the drug was referred for reimbursement review typically reserved for drugs treating larger patient populations. NICE is taking over the evaluation of the cost effectiveness of expensive drugs for rare diseases from the Advisory Group for National Specialized Services. The first test will be NICE’s evaluation of Soliris. Soliris is approved for the treatment of aHUS, a rare kidney disease. Less than 300 people in the UK have aHUS. According to a recent article in the UK’s The Independent :

“NICE has yet to explain how it is going to assess drugs for rare diseases. A spokesman said an announcement would be made “within weeks” …. Nice’s verdict on Eculizumab …  is not expected before summer 2014. Eculizumab costs £327,600 a year for an adult and £163,800 for a child with aHUS. In the meantime, NHS England has established an interim scheme under which new patients – around 20 a year – will get the drug and existing patients already on it will continue to receive it. But patients not currently receiving Eculizumab, such as Dianne, will have to wait for NICE’s decision.”

Please Note: “Dollar Symbol Gold” by Rugby471 (Own work) [GFDL] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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