In atypical Hemolytic Uremic Syndrome (aHUS),

uncontrolled complement activation leads to systemic

thrombotic microangiopathy (TMA), causing progressive

damage to vital organs and premature death1-5

IN aHUS, complement-mediated

TMA CAUSES sudden and

progressive ORGAN FAILURE

and premature DEATH

Alexion’s Soliris, a drug which has won awards for medical innova-tion, has earned its commercial

stripes, too. At press time, the biologic was on track to cross a billion dollars in worldwide sales. That’s not so rare

among therapies that, like Soliris, treat extremely obscure ailments and cost $200,000 or more per patient per year, but it’s impres-sive considering the patient pool is just a few thousand.

The drug, Alexion’s first and only commercialized product, has

helped push the company into the biotech big leagues with a market cap just shy of $20 billion, the sixth-largest behind names like Gilead and Amgen. Analysts expect Soliris to become a multi-billion dollar franchise.

How it got to this point is a model for building a profitable ultra-orphan brand. “We’re focused on developing and delivering

therapies that transform the lives

An ultrarare success storyP Small pharma marketing team of the Year soLiris P

In atypical Hemolytic Uremic Syndrome (aHUS),

uncontrolled complement activation leads to systemic

thrombotic microangiopathy (TMA), causing progressive

damage to vital organs and premature death

IN aHUS, complement-mediated

TMA CAUSES sudden and

progressive ORGAN FAILURE

and premature DEATH

Alexion’s Soliris, a drug which has won awards for medical innova-tion, has earned its commercial

stripes, too. At press time, the biologic was on track to cross a billion dollars in worldwide sales. That’s not so rare

among therapies that, like Soliris, treat extremely obscure ailments and cost $200,000 or more per patient per year, but it’s impres-sive considering the patient pool is just a few thousand.

The drug, Alexion’s first and only commercialized product, has

helped push the company into the biotech big leagues with a market cap just shy of $20 billion, the sixth-largest behind names like Gilead and Amgen. Analysts expect Soliris to become a multi-billion dollar franchise.

How it got to this point is a model for building a profitable ultra-orphan brand. “We’re focused on developing and delivering

therapies that transform the lives

An ultrarare success story

L-R: Jeroen van Beek, VP, global nephrology franchise; David Hallal, EVP, chief commercial offi cer; Margaret olinger, VP, global hematology franchise, Alexion

of patients with ultrarare and severe, life-theatening diseases—patients that without our therapies have no other hope,” says Alexion’s David Hallal, EVP, chief commercial officer.

When MM&M caught up with Hal-lal in early December, his team was in the midst of launching Soliris for the second of its two indications, atypical hemolytic uremic syndrome (aHUS), approved in late 2011. Approval for the first, paroxysmal nocturnal hemo-globinuria (PNH), was secured in 2007. “In these settings, Soliris has a 100% response rate,” he says.

The US aHUS launch is progress-ing well, according to analysts, with European reimbursement for aHUS expected in mid-2013. Several other indications are set to follow.

“Our approach to aHUS was simi-lar to that of PNH,” notes Alexion’s Jeroen van Beek, PhD, VP, global neph-rology franchise, “in that the focus is on disease education and establishing the importance and pathway to rapidly

diagnose patients with aHUS so that they can receive the life-transforming benefits of Soliris.”

In addition to a highly educated sales force calling on hematologists, nephrologists and renal transplanters, Alexion supports promotional programs where KOLs help teach physicians in the field. It also uti-lizes medical con-ferences, where its trade show booth focuses on early d iagnos i s and intervention.

While the firm maintains a dis-ease, brand and corporate web presence, its most important patient-facing effort, says Hallal, is its One-Source education

An ultrarare success storyPH

OTO

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T: B

ILL

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STEI

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P large pharma marketing team of the Year novo nordisk P

Alexion education efforts about Soliris (below) include non-branded disease sites for aHUS (above) and for PNH. Below left, a disease awareness program

and reimbursement program, in which Alexion’s staff of registered-nurse case managers works one-on-one with the patient, family and HCP.

“It’s really important to advocate for that individual patient and not necessarily attack all payers with a broader central message,” he says. “Among payers, their experience is usually with a few [PNH or aHUS] patients anyway.”

Two-thirds of its business is ex-US, and the firm has secured public fund-ing for Soliris in such jurisdictions as Australia and Canada—two countries well known for being perhaps even more restrictive than the EU. That’s not a slam dunk given that the drug costs about $440,000 per patient per year (it’s sometimes given away, in cases of hardship).

“Because the benefits of Soliris are transformative and not incremental, governments around the world…are recognizing the benefit,” says Hallal.

For instance, he cited research sug-gesting that PNH patients followed for up to eight years on treatment can expect the same life span as those of a normal population that are age- and gender-matched. If left untreated, PNH has a 35% mortality rate at five years. Says Hallal: “This provides a very strong underpinning for our access strategy.”

Disease awareness and diagnostic programs are set to increase, adds Mar-garet Olinger, VP, global hematology franchise, in order to “positively influ-ence the entire cycle of care.”

PNH and aHUS are forecast to achieve global sales peaks of about $2 billion each, and with compelling evi-dence of Soliris efficacy in other indi-cations, it could reach up to $8 billion in peak sales, according to Barclays Research. Go to mmm-online.com for more creative. —Marc Iskowitz

AGENCY RoStERPRoFESSIoNAL (HEMAtoLoGY)McCann Regan Campbell Ward

PRoFESSIoNAL (NEPHRoLoGY)Sudler & Hennessey

DIGItALBand Digital, McCann RCW

PUBLIC RELAtIoNSSmithSolve

mmm-online.com x JANUARY 2013 x MM&M 49

The Case for Orphan Drugs Drug Discovery and Development By Louise Rozik August 22, 2014 Audience Reach – 28,166

Neglected by the pharmaceutical industry before

the Orphan Drug Act of 1983, rare diseases are

now front and center in pharma

R&D. Approximately one-third of all new

medicines approved by the FDA in the past 5

years were developed for the treatment of

diseases that affect fewer than 200,000

Americans. Now, however, the increasing number and cost of orphan drugs are agrowing

concern to patients, providers and payers. The specter of ballooning drug costs should not,

however, discredit the original purpose of the Orphan Drug Act: to encourage the development

of new treatments for patients with rare diseases who had no hope of a cure or even

symptomatic relief.

A case in point is atypical hemolytic uremic syndrome (aHUS), a rare, life-threatening disease

with an incidence of only 2 per million in the U.S. and 1.5-1.8 per million in Europe. When

Danielle Williams was told that her son Justice, a previously growing and healthy boy, had

aHUS, she was scared. She had never heard of aHUS before and didn’t know what, if any,

treatments were available. It turns out that she had good reason for concern.

Had Justice been diagnosed just a year earlier, his options would have been very limited.

Standard treatment was plasma exchange or infusion, which produced dismal results: within a

year of diagnosis, 65% of all aHUS patients treated with plasma exchange or infusion died,

required dialysis, or had permanent renal damage. Fortunately for the Williams family, and all

the other patients with aHUS, Alexion Pharmaceuticals Inc., Cheshire, Conn., had developed a

treatment for this devastating disease. The product of this commitment, a monoclonal antibody

called Soliris, gave Justice a chance. Its availability was a “miracle,” said Williams.

But critics have been quick to point out that companies who develop orphan drugs may not

purely be motivated by altruism. After all, there is a lot of money to be made in the rare disease

space. Wall Street analysts estimate that at a cost of $400,000 per patient per year, Soliris

could bring Alexion $2.6 billion in annual sales by 2017.

Working in the rare disease space

When asked to explain the cost of orphan drugs, Martin Mackay, EVP, Global Head of R&D,

Alexion, said that companies developing these drugs “continue to invest in R&D programs to

develop more innovative treatments for patients suffering with other severe and rare diseases.

Additionally, we invest a great deal in disease education and other programs to ensure that

patients with these devastating diseases receive optimal care. There are also several unique

challenges when working in the rare disease setting, which are reflected in the cost.”

Mackay sites the design of clinical trials as one such challenge, explaining that these trials are

often without precedent, so there are no roadmaps to follow for the endpoints. Companies

developing treatments for rare diseases have to fill this void. They are often pioneers in the

assessment of the efficacy and safety of orphan drugs and must work closely with regulatory

bodies around the world to ensure consensus.

Patient recruitment in rare diseases is tough as well. Due to the small patient populations and

challenges with diagnosis, patient recruitment requires a large and vast global network to enroll

very small numbers of patients, explains Mackay.

As well, rare diseases are often poorly studied, and the pathophysiology of the disease is not

well understood. This can make the search for the defect difficult, prolonged, and therefore

costly.

Despite these challenges, says Mackay, “We are driven to pursue therapeutic candidates that

bring significant value to patients suffering with devastating diseases rather than incremental

benefits. We aim to develop and deliver treatments that will dramatically transform their lives."

Putting the cost of orphan drugs into perspective

Nobody can deny that orphan drugs can be profitable, and some question whether the costs are

excessive. But many also maintain that providing desperately needed treatments for rare

diseases is the right thing to do, despite the cost…that turning our backs on patients with rare

diseases and their families, especially after the success of orphan drug incentives, is unethical.

As more orphan drugs become available, the trick will be to ensure that the ethical obligation to

treat patients with rare diseases is balanced by sustainable costs.